S-1/A 1 forms-1a.htm

 

As filed with the Securities and Exchange Commission on December 14, 2023

 

Registration No. 333-275534

 

 

 

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

FORM S-1

Amendment No. 1

 

REGISTRATION STATEMENT
UNDER
THE SECURITIES ACT OF 1933

 

TELOMIR PHARMACEUTICALS, INC.

(Exact name of registrant as specified in its charter)

 

Florida

2834

87-2606031

(State or other jurisdiction of

incorporation or organization)

 

(Primary Standard Industrial

Classification Code Number)

 

(I.R.S. Employer

Identification No.)

 

855 N Wolfe Street, Suite 601
Baltimore, Maryland 21205
(737) 289-0835

(Address, including zip code, and telephone number, including area code, of registrant’s principal executive offices)

 

Dr. Christopher Chapman, Jr.

Chief Executive Officer
Telomir Pharmaceuticals, Inc.
900 West Platt Street, Suite 200
Tampa, Florida 33606
(737) 289-0835

(Name, address, including zip code, and telephone number including area code, of agent for service)

 

Copies of all communications, including communications sent to agent for service, should be sent to:

 

Curt P. Creely

Neda Sharifi

Foley & Lardner LLP

100 North Tampa Street, Suite 2700

Tampa, Florida 33602

(813) 229-2300

 

Joseph M. Lucosky, Esq.

Lucosky Brookman LLP
101 Wood Avenue South, 5th Floor
Woodbridge, New Jersey 08830
(732) 395-4402

 

Approximate date of commencement of proposed sale to the public: As soon as practicable after this Registration Statement becomes effective.

 

If any of the securities being registered on this Form are to be offered on a delayed or continuous basis pursuant to Rule 415 under the Securities Act of 1933, as amended (the “Securities Act”), check the following box. ☒

 

If this Form is filed to register additional securities for an offering pursuant to Rule 462(b) under the Securities Act, please check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

 

If this Form is a post-effective amendment filed pursuant to Rule 462(c) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

 

If this Form is a post-effective amendment filed pursuant to Rule 462(d) under the Securities Act, check the following box and list the Securities Act registration statement number of the earlier effective registration statement for the same offering. ☐

 

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

 

Large accelerated filer ☐ Accelerated filer ☐
Non-accelerated filer ☒ Smaller reporting company ☒
  Emerging growth company ☒

 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 7(a)(2)(B) of the Securities Act. ☐

 

The Registrant hereby amends this Registration Statement on such date or dates as may be necessary to delay its effective date until the Registrant shall file a further amendment which specifically states that this Registration Statement shall thereafter become effective in accordance with Section 8(a) of the Securities Act of 1933 or until this Registration Statement shall become effective on such date as the Securities and Exchange Commission, acting pursuant to said Section 8(a), may determine.

 

 

 

 
 

 

EXPLANATORY NOTE

 

This Registration Statement contains two prospectuses as set forth below.

 

  Public Offering Prospectus: A prospectus to be used for the initial public offering by the Company of 1,000,000 shares of its common stock through the underwriters named on the cover page, which we refer to as the “Public Offering Prospectus”.
     
  Resale Prospectus: A prospectus to be used for the potential resale by selling stockholders of up to 6,203,696 shares of common stock of the Company, which we refer to as the “Resale Prospectus”.

 

The Resale Prospectus is substantively identical to the Public Offering Prospectus, except for the following principal points:

 

they contain different outside and inside front covers;
   
they contain different Offering sections in the Prospectus Summary section;
   
they contain different Use of Proceeds sections;
   
the Capitalization and Dilution sections are deleted from the Resale Prospectus;
   
a Selling Stockholders section is included in the Resale Prospectus;
   
the Underwriting section from the Public Offering Prospectus is deleted from the Resale Prospectus and a Plan of Distribution is inserted in its place; and
   
the Legal Matters section in the Resale Prospectus deletes the reference to counsel for the underwriters.

 

We have included in this Registration Statement, after the financial statements, a set of alternate pages after the back cover page of the Public Offering Prospectus, which we refer to as the “Alternate Pages”, to reflect the foregoing differences in the Resale Prospectus as compared to the Public Offering Prospectus. The Public Offering Prospectus will exclude the Alternate Pages and will be used for the public offering by the registrant. The Resale Prospectus will be substantively identical to the Public Offering Prospectus except for the addition or substitution of the Alternate Pages and will be used for the resale offering by the selling stockholders.

 

 
 

 

The information in this preliminary prospectus is not complete and may be changed. We may not sell these securities until the registration statement filed with the Securities and Exchange Commission is effective. This preliminary prospectus is not an offer to sell, and it is not soliciting an offer to buy, these securities in any state where the offer or sale is not permitted.

 

Subject to completion, dated December 14, 2023

 

PROSPECTUS

 

1,000,000 Shares

 

Common Stock

 

A blue and black logo

Description automatically generated

 

This is the initial public offering of 1,000,000 shares of common stock, no par value, of Telomir Pharmaceuticals, Inc.

 

Prior to this offering, there has been no public market for shares of our common stock. The assumed initial public offering price is $7.00 per share. We have applied to have shares of our common stock listed on the Nasdaq Capital Market (“Nasdaq”) under the symbol “TELO”. If shares of our common stock are not approved for listing on Nasdaq, we will not consummate this offering. No assurance can be given that our application will be approved.

 

Unless otherwise noted, the share and per share information in this prospectus reflects a 1-for-2.05 reverse stock split of our common stock that became effective as of December 11, 2023.

 

We are an “emerging growth company” as defined in the federal securities laws, and, as such, are subject to reduced public company reporting requirements. See “Prospectus Summary — Implications of Being an Emerging Growth Company”.

 

Investing in shares of our common stock involves risks. See “Risk Factors” beginning on page 11 to read about factors you should consider before buying shares of our common stock.

 

Neither the Securities and Exchange Commission nor any state securities commission or other regulatory body has approved or disapproved of these securities or passed upon the adequacy or accuracy of this prospectus. Any representation to the contrary is a criminal offense.

 

   Per Share   Total 
Initial public offering price  $ 7.00    $ 7,000,000  
Underwriting discounts and commissions(1)  $

0.49

   $

490,000

 
Proceeds, before expenses, to us  $

6.51

   $

6,510,000

 

 

(1) See “Underwriting” for a description of the compensation payable to the underwriters.

 

The underwriters have the option for a period of 45 days from the date of this prospectus to purchase up to 150,000 additional shares of our common stock from us at the initial public offering price, less underwriting discounts and commissions. If the underwriters exercise the option in full, the total underwriting discounts and commissions payable by us will be $563,500, and the total proceeds, before expenses, to us will be $7,486,500.

 

The underwriters expect to deliver the shares to investors on or about , 2023.

 

Kingswood Investments

division of Kingswood Capital Partners, LLC

 

The date of this prospectus is            , 2023

 

 
 

 

TABLE OF CONTENTS

 

  Page
INDUSTRY AND MARKET DATA ii
   
TRADEMARKS AND COPYRIGHTS ii
   
CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS ii
   
GLOSSARY OF CERTAIN SCIENTIFIC TERMS iv
   
PROSPECTUS SUMMARY 1
   
RISK FACTORS 11
   
USE OF PROCEEDS 39
   
DIVIDEND POLICY 40
   
CAPITALIZATION 41
   
DILUTION 42
   
MANAGEMENT’S DISCUSSION AND ANALYSIS OF FINANCIAL CONDITION AND RESULTS OF OPERATIONS 43
   
BUSINESS 48
   
MANAGEMENT 67
   
EXECUTIVE COMPENSATION 74
   
CERTAIN RELATIONSHIPS AND RELATED PARTY TRANSACTIONS 81
   
PRINCIPAL SHAREHOLDERS 82
   
DESCRIPTION OF CAPITAL STOCK 83
   
SHARES ELIGIBLE FOR FUTURE SALE 89
   
MATERIAL U.S. FEDERAL INCOME TAX CONSIDERATIONS FOR NON-U.S. HOLDERS OF OUR COMMON STOCK 91
   
UNDERWRITING 94
   
LEGAL MATTERS 101
   
EXPERTS 101
   
WHERE YOU CAN FIND MORE INFORMATION 101
   
INDEX TO FINANCIAL STATEMENTS F-1

 

Please read this prospectus carefully. It describes our business, financial condition, results of operations and prospects, among other things. We are responsible for the information contained in this prospectus and in any free-writing prospectus we have authorized. Neither we nor the underwriters have authorized anyone to provide you with different information, and neither we nor the underwriters take responsibility for any other information others may give you. Neither we nor the underwriters are making an offer to sell these securities in any jurisdiction where the offer or sale is not permitted. The information contained in this prospectus is accurate only as of the date on the front of this prospectus, regardless of the time of delivery of this prospectus or any sale of securities. You should not assume that the information contained in this prospectus is accurate as of any date other than its date.

 

i
 

 

INDUSTRY AND MARKET DATA

 

We are responsible for the disclosure in this prospectus. However, this prospectus includes industry data that we obtained from internal surveys, market research, publicly available information, and industry publications. We did not fund and are not otherwise affiliated with any of the sources cited in this prospectus. The market research, publicly available information, and industry publications that we use generally state that the information contained therein has been obtained from sources believed to be reliable. The information therein represents the most recently available data from the relevant sources and publications, and we believe remains reliable. However, this data involves a number of assumptions and limitations regarding our industry which are necessarily subject to a high degree of uncertainty and risk due to a variety of factors, including those described in the section titled “Risk Factors.” Forward-looking information obtained from these sources is also subject to the same qualifications and additional uncertainties regarding the other forward-looking statements in this prospectus.

 

TRADEMARKS AND COPYRIGHTS

 

We own or have rights to various trademarks, service marks and trade names that we use in connection with the operation of our business. This prospectus may also contain trademarks, service marks and trade names of third parties, which are the property of their respective owners. Our use or display of third parties’ trademarks, service marks and trade names or products in this prospectus is not intended to, and does not imply a relationship with, or endorsement or sponsorship by us. Solely for convenience, the trademarks, service marks and trade names referred to in this prospectus may appear without the ®, trademark (™) or servicemark (SM) symbols, but the omission of such references is not intended to indicate, in any way, that we will not assert, to the fullest extent under applicable law, our rights or the right of the applicable owner of these trademarks, service marks and trade names.

 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

 

This prospectus contains forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential”, or “continue” or the negative of these terms or other similar expressions. In particular, statements about the markets in which we operate, including growth of our various markets, and our expectations, beliefs, plans, strategies, objectives, prospects, assumptions, or future events or performance contained in this prospectus under the headings “Prospectus Summary,” “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and “Business” are forward-looking statements.

 

We have based these forward-looking statements on our current expectations, assumptions, estimates and projections. While we believe these expectations, assumptions, estimates, and projections are reasonable, such forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond our control. These and other important factors, including those discussed in this prospectus under the headings “Prospectus Summary,” “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and “Business,” may cause our actual results, performance, or achievements to differ materially from any future results, performance or achievements expressed or implied by these forward-looking statements, or could affect our share price. Important factors that could cause actual results or events to differ materially from those expressed in forward-looking statements include, but are not limited to, the following:

 

  our use of the net proceeds from this offering;
     
  our ability to obtain and maintain regulatory approval of our product candidates;
     
  our ability to successfully commercialize and market our product candidates, if approved by the FDA;
     
  our ability to contract with third-party suppliers, manufacturers and other service providers and their ability to perform adequately;
     
  the potential market size, opportunity, and growth potential for our product candidates, if approved by the FDA;

 

ii
 

 

  our ability to obtain additional funding for our operations and development activities;
     
  the accuracy of our estimates regarding expenses, capital requirements and needs for additional financing;
     
  the initiation, timing, progress and results of our pre-clinical studies and clinical trials, and our research and development programs;
     
  the timing of anticipated regulatory filings;
     
  the timing of availability of data from our clinical trials;
     
  our future expenses, capital requirements, need for additional financing, and the period over which we believe that the net proceeds from this offering, together with our existing cash and cash equivalents, will be sufficient to fund our operating expenses and capital expenditure requirements;
     
  our ability to retain the continued service of our key professionals and to identify, hire and retain additional qualified professionals;
     
  our ability to advance product candidates into, and successfully complete, clinical trials;
     
  our ability to recruit and enroll suitable patients in our clinical trials;
     
  the timing or likelihood of the accomplishment of various scientific, clinical, regulatory, and other product development objectives;
     
  the pricing and reimbursement of our product candidates, if approved by the FDA;
     
  the rate and degree of market acceptance of our product candidates, if approved by the FDA;
     
  the implementation of our business model and strategic plans for our business, product candidates, and technology;
     
  the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology;
     
  developments relating to our competitors and our industry;
     
  the development of major public health concerns, including the novel coronavirus outbreak or other pandemics arising globally, and the future impact of it and COVID-19 on our clinical trials, business operations and funding requirements; and
     
  other risks and factors listed under “Risk Factors” and elsewhere in this prospectus.

 

Given the risks and uncertainties set forth in this prospectus, you are cautioned not to place undue reliance on such forward-looking statements. The forward-looking statements contained in this prospectus are not guarantees of future performance and our actual results of operations, financial condition, and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this prospectus. In addition, even if our results of operations, financial condition and liquidity, and events in the industry in which we operate, are consistent with the forward-looking statements contained in this prospectus, they may not be predictive of results or developments in future periods.

 

Any forward-looking statement that we make in this prospectus speaks only as of the date of such statement. Except as required by federal securities laws, we do not undertake any obligation to update or revise, or to publicly announce any update or revision to, any of the forward-looking statements, whether as a result of new information, future events or otherwise, after the date of this prospectus.

 

iii
 

 

GLOSSARY OF CERTAIN SCIENTIFIC TERMS

 

The following medical and scientific terms used in this prospectus have the following meanings:

 

“Antineoplastic drugs” are medications used to treat cancer, also known as chemotherapies.

 

“API” stands for Active Pharmaceutical Ingredient, which is the main ingredient in a medicine that causes the desired effect of the medicine.

 

“AMES test” is a biological assay to assess the mutagenic potential of chemical compounds. It utilizes bacteria to test whether a given chemical can cause mutations in the DNA of the test organism.

 

“C282Y” is a gene mutation associated with hereditary hemochromatosis.

 

“CDMO” stands for Contract Development and Manufacturing Organization, a specialized type of supplier of development and production services to the pharmaceutical industry.

 

“cGMP” is the current Good Manufacturing Practices under the US Food and Drug Administration’s standards. cGMP contains the minimum requirements for the methods, facilities, and controls used in the manufacturing, processing, and packing of a drug product. The regulations make sure that a product is manufactured under conditions and tested to ensure that it meets standards of identity, strength, quality, and purity.

 

“CMC” stands for chemistry, manufacturing, and controls and covers the various procedures used to assess the physical and chemical characteristics of drug products, and to ensure their quality and consistency during manufacturing.

 

“CYP” stands for cytochrome P450 enzymes, which are a complex and important component of drug metabolism. It is the root of many drug interactions due to inhibition, induction, and competition for common enzymatic pathways by different drugs.

 

“Cytokines” are a broad category of small proteins that are important in cell signaling. The release of pro-inflammatory cytokines leads to activation of immune cells and production of and release of additional cytokines.

 

“DNA” is the molecule that carries genetic information for the development and functioning of an organism.

 

“FDA” is the U.S. Food and Drug Administration.

 

“GMP” is good manufacturing practice – a standard that is observed in regulated pharmaceutical-manufacturing facilities.

 

“H63D” is a gene mutation associated with hereditary hemochromatosis.

 

“HFE gene” is a gene that provides instructions for producing a protein that is located on the surface of cells, primarily liver and intestinal cells.

 

“Hereditary Hemochromatosis” a genetic disorder that can cause severe liver disease and other health problems.

 

“IL-17” or Interleukin-17 is a pro-inflammatory cytokine that modulates protective innate immunity to pathogens or contributes to the pathogenesis of inflammatory conditions.

 

“In situ” means in the original or normal place.

 

“Iron Overload” is a condition in which your body stores too much iron.

 

“Maximum tolerated dose” is the highest dose of a drug or treatment that does not cause unacceptable side effects. The maximum tolerated dose is determined in clinical trials by testing increasing doses on different subjects until the highest dose with acceptable side effects is found.

 

“Metabolite” is a substance made or used when the body breaks down food, drugs or chemicals, or its own tissue.

 

“mTOR” stands for mammalian target of rapamycin which regulates cell proliferation, autophagy, and apoptosis by participating in multiple signaling pathways in the body.

 

“Pluripotent stem cells” are stem cells that are able to self-renew by dividing and developing into primary groups of cells that make up the human body.

 

“qPCR” stands for quantitative polymerase chain reaction (qPCR). qPCR is the accumulation of amplification product is measured as the reaction progresses, in real time, with product quantification after each cycle.

 

iv
 

 

 

PROSPECTUS SUMMARY

 

The following summary highlights selected information about our company and this offering that is included elsewhere in this prospectus in greater detail. It does not contain all of the information that you should consider before investing in our common stock. Before investing in our common stock, you should read this entire prospectus carefully, including the information presented under the heading “Risk Factors” and in our financial statements and notes thereto.

 

Unless otherwise noted, the share and per share information in this prospectus reflects a 1-for-2.05 reverse stock split of our common stock that became effective as of December 11, 2023.

 

In this prospectus, unless we indicate otherwise or the context requires, “Telomir,” “the company,” “our company,” “we,” “our,” “ours” and “us” refer to Telomir Pharmaceuticals, Inc.

 

Business Summary

 

Overview

 

We are a pre-clinical-stage pharmaceutical company focused on the development and commercialization of TELOMIR-1, a novel small molecule being developed to function as an oral in situ therapeutic treatment for human stem cells. Based on our pre-clinical studies and if approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 may potentially serve as a metal enzyme inhibitor of essential metals such as zinc and copper. These essential metals play an important role in the production and function of many enzymatic reactions and the modulation of key cellular pathways. In particular, zinc is essential to the function of pro-inflammatory cytokines such as Interleukin-17, or IL-17, that play a role in a host of age-related inflammatory conditions such as hemochromatosis and osteoarthritis as well as in post-chemotherapy health problems. IL-17 is a type of pro-inflammatory cytokine, and while the pro-inflammatory properties of IL-17 are key to its host-protective capacity, unrestrained IL-17 signaling is associated with immunopathology, inflammatory disease and cancer progression. Our initial focus will be on treatments to inhibit the production of pro-inflammatory cytokines, such as IL-17, by oral administration of TELOMIR-1 as a therapeutic treatment for stem cells in situ. To the best of our knowledge, there is no approved oral IL-17 inhibitor. Our goal is to advance the clinical development of TELOMIR-1 in the United States for the treatment of age-related inflammatory conditions such as hemochromatosis and osteoarthritis, as well as in post-chemotherapy recovery, with our initial targeted indications being hemochromatosis and post-chemotherapy recovery.

 

Pluripotent stem cells are a type of stem cells that have the ability to undergo self-renewal and to give rise to various cell types of the tissues of the body. Based on pre-clinical studies, we believe that TELOMIR-1 may have the potential to protect stem cells in situ by reducing the overload of metals such as zinc and copper that accompany age-related inflammatory conditions and certain cancers by modulating pro-inflammatory cytokines such as IL-17. If demonstrated by future clinical trials and approved by the FDA and comparable foreign regulators, we believe TELOMIR-1 may protect the stem cells by elongating and stimulating the telomeres to sustain self-renewal of stem cells. Telomeres are repetitive DNA sequences at the end of chromosomes that protect the chromosomes from becoming frayed or tangled. Each time a cell divides, the telomeres become slightly shorter, and eventually they become so short that the cell can no longer divide, with the result being that the cell dies. Effectively, telomeres protect the ends of our chromosomes by forming a cap, much like the plastic tip on shoelaces, thereby allowing the chromosome to be replaced properly during cell division.

 

 

1

 

 

 

TELOMIR-1 is currently under investigation to provide a potential therapeutic intervention against age-related inflammatory conditions such as hemochromatosis, as well as for post-chemotherapy recovery, by interrupting and preventing the IL-17 induced inflammatory pathways that create the systemic imbalance of cellular metals. Our pre-clinical studies suggest that TELOMIR-1 may inhibit the concentration and accumulation of metals such as zinc and copper in the serum that drive the hyperactivity of pro-inflammatory cytokines such as IL-17. Our studies suggest that TELOMIR-1 may achieve this outcome by selectively binding to metal ions in a dose dependent manner, slowing enzyme reactivity, and protecting and lengthening telomeres in the human chromosome. If demonstrated in clinical trials and approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 has potential as a non-toxic oral enzyme inhibitor that may regulate the overactivity of the enzymes caused by excessive metal reactivity.

 

We were organized as a Florida corporation in August 2021 for the purpose of pursuing the development and commercialization of TELOMIR-1 in the United States in human applications. We were originally incorporated under the name “Metallo Therapies Inc.” and changed our name to “Telomir Pharmaceuticals, Inc.” in October 2022. We are an early-stage company that had net losses of $0.14 million and $0.85 million for the years ended December 31, 2021 and 2022, respectively.

 

In Situ Therapeutic Treatment of Stem Cells

 

Stem cells have the potential to renew themselves. They can develop into many different cell types in the body during early and adult life. Pluripotent stem cells have the ability to differentiate into all of the cells of the adult body. Since pluripotent stem cells are undifferentiated, they do not have any tissue-specific characteristics that allow them to perform specialized functions. Given their unique regenerative abilities and limited quantities in the adult human body, in situ treatment and protection of stem cells may provide an important therapeutic mechanism for treatment of disease.

 

Metal Overload and Telomere Length

 

Research studies have demonstrated that metal overload in stem cells could severely impair the proliferation of pluripotent stem cells through excessive DNA damage. Furthermore, many pro-inflammatory cytokine-induced conditions create an imbalance of cellular metals such as zinc and copper. This metal imbalance may impact the length of telomeres in stem cells and impact their ability to sustain self-renewal.

 

Hemochromatosis

 

According to the CDC, hemochromatosis is a disorder in which the body builds up too much iron in the skin, heart, liver, pancreas, pituitary glands, and joints. This overload of iron is toxic to the body, and over time, the high levels of iron can damage tissues and organs and lead to conditions such as liver damage, liver cancer, heart problems, arthritis, and diabetes. Other conditions associated with high iron levels include inflammatory conditions, chronic kidney disease, and autoimmune disorders. Hemochromatosis is a life-long condition requiring regular treatment to avoid long-term serious effects with poor pharmacologic options. The most used treatment for hemochromatosis is phlebotomy, a procedure to remove some of the patient’s blood. Phlebotomy is relatively inexpensive, well accepted, and well tolerated, but it requires regular visits to health care professionals and blood draws and may not be appropriate for all patients. Additionally, iron chelators, which are molecules that bind to iron to help the body excrete it, are sometimes used, but they have limited use due to gastrointestinal and kidney toxicity issues. Iron chelators can be natural or synthetic and are used to treat iron overload, which can occur after regular blood transfusions. Despite the presence of these potential treatment options, we believe significant unmet need remains such that an innovative therapy achieving reduction in iron overload with a new profile could be subject to widespread use.

 

 

2

 

 

 

We are pursuing TELOMIR-1 as a metal enzyme inhibitor that, if approved by the FDA and comparable foreign regulators, may selectively bind metal ions to inhibit pro-inflammatory cytokines such as IL-17 and protect telomeres and that aims to increase the average telomere length, or ATL, by selectively binding iron and reducing blood iron levels, therefore supporting the ability to treat hemochromatosis. On average, about 750,000 US patients express one or more iron overload symptoms. There are 2 types of hemochromatosis, with the following patient mix: 150,000 primary hemochromatosis and 65,000 secondary hemochromatosis confirmed diagnoses in the United States. Over 150,000 patients have sought treatment since 2018, with most receiving phlebotomy, which is the withdrawal of blood to bring iron to normal levels. At present, 99% of hemochromatosis patients receive phlebotomy, with a portion of patients also receiving iron chelators as a co-treatment. Note that in the last 12 months, there have been 76,000 confirmed diagnoses of hereditary hemochromatosis and approximately 1% of hemochromatosis patients received deferasirox or deferoxamine off-label for 12 weeks (1 year for primary).

 

Certain sub-groups of hemochromatosis patients may benefit from TELOMIR-1, in addition to phlebotomy, which is the current standard of care for the treatment of hemochromatosis, if contraindicated for this treatment method. Lastly, iron chelation is used off-label, poorly tolerated and has toxic long-term effects. In pre-clinical stage testing, TELOMIR-1 may have more effective metal complexing than Doxycycline, an FDA-approved metalloproteinases (“MMP”) inhibitor. However, pre-clinical data may prove inaccurate and is not necessarily indicative of future results. MMP inhibitors act as “brakes” for enzymes in our body. MMP enzymes sometimes become too active and break down compounds and structures they should not, such as the body’s tissues. MMP inhibitors are compounds that typically slow down or stop these overactive enzymes, preventing them from causing damage to our tissues. They act as a control switch to keep the body in balance and protect it from harm.

 

Post-Chemotherapy Recovery

 

Post-chemotherapy recovery from adverse effects of antineoplastic treatments is often important for cancer therapy success. While chemotherapy treatment can be highly effective for cancer, it can also come with many side effects, as chemotherapy drugs destroy both cancerous and healthy cells. We are investigating the use of TELOMIR-1 as a potential complementary treatment for patients receiving chemotherapy in the form of a twice daily, oral regimen to inhibit pro-inflammatory cytokines and to reduce blood iron levels, enabling potentially more effective adherence and improved outcomes. In exploratory early discovery studies, proof of concept was demonstrated, and the animal studies are pending. Post-chemotherapy recovery space contains several treatments that are used symptomatically based on the nature or severity of side effects.

 

We believe that post-chemotherapy recovery presents several potential unmet commercial needs for TELOMIR-1. There is potential to enable a more rapid recovery of patients receiving chemotherapy, improving adherence with chemotherapy regimens. Additionally, as current recovery management is based on severity and treated as needed, we believe that TELOMIR-1 could potentially be used as a preventative measure. As a complementary treatment, side effect reduction in post-chemotherapy is important to minimize patient burden while improving treatment outcomes.

 

Our Strategy

 

Our goal is to develop and commercialize new treatment options for treatment of age-related inflammatory conditions, with hemochromatosis as our initial clinical focus, and to thereafter expand the development of TELOMIR-1 to post-chemotherapy recovery as well as a broader range of other age-related inflammatory diseases and conditions such as osteoarthritis. The key elements of our strategy to achieve this goal include:

 

  Advancing TELOMIR-1 through clinical development and approval for hemochromatosis.
     
  Continuing pre-clinical development of TELOMIR-1 for post-chemotherapy recovery and across a range of inflammatory diseases associated with cellular aging.
     
  Exploring strategic collaborations to maximize the value of TELOMIR-1.

 

 

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Pre-Clinical Studies

To date, we have completed several pre-clinical proof-of-concept studies with respect to TELOMIR-1 that were designed to demonstrate that TELOMIR-1 is not mutagenic and has good biological and metal binding capabilities. These studies included an Ames test for the TELOMIR-1 compound and several Eurofins BioMap studies. Eurofins BioMAP studies are scientific experiments that help with understanding how different chemicals or drugs impact the human body. These in vitro studies use cells and tissues to see how they react when exposed to various substances. BioMAP studies may demonstrate the safety and efficacy of drugs and chemicals in treatment of diseases. They assist researchers in assessing whether a substance, at microscopic level, has a positive or negative impact on health of the subject by studying how it interacts within the cells or with other cells. The Ames test assesses whether a chemical or substance causes DNA mutations. Scientists use this test to figure out if a chemical might be harmful and could potentially cause cancer because DNA mutations are often linked to cancer.

 

The Eurofins in vitro studies showed that TELOMIR-1 was more active against malignant cells, colorectal cancer cells, and lung cancer cells than rapamycin. Rapamycin is a compound that has anti-tumor and immunosuppressive properties, and it was selected to compare against TELOMIR-1 because rapamycin and TELOMIR-1 both inhibit similar cytokines involved in immune responses. Several pro-inflammatory cytokines are common proteins that are affected by TELOMIR-1 and rapamycin. Rapamycin is also known by the brand names Rapamune and Sirolimus. It is a mammalian target of rapamycin (mTOR) inhibitor which works by slowing down cellular multiplication. mTOR inhibitors have been associated with anemia (a condition characterized by a low red blood cell count or low hemoglobin levels) due to a change in iron metabolism. Rapamycin is used to prevent organ transplant rejection and to treat certain types of cancer. mTORs regulates cell proliferation, autophagy, and apoptosis by participating in multiple signaling pathways in the body.

 

Doxycycline possesses anti-inflammatory properties that are in part attributed to its ability to chelate metal ions such as zinc, which are involved in the enzymatic processes related to inflammation. In particular, doxycycline has been shown to inhibit the TNF-α Converting Enzyme (TACE) as this enzyme requires zinc to function. Inhibition of TACE in turn leads to decreased systemic levels of the active TNF-α, which is implicated in inflammation and autoimmune diseases. This action contributes to the anti-inflammatory properties of doxycycline, which are beneficial in diseases such as rheumatoid arthritis and inflammatory skin conditions.

 

Similar to doxycycline, TELOMIR-1 is also a metal ion chelating drug, which underlies its ability to inhibit TACE by competing for the zinc that is required for its enzymatic activity. Both Doxycycline and TELOMIR-1inhibit the enzymatic activity of TACE which plays a key role in both acute and chronic inflammation.

 

Thus, doxycycline is a reasonable choice for comparison with TELOMIR-1 since, similar to doxycycline TELOMIR-1 is believed to exert its anti-inflammatory effects by inhibiting TACE in the same manner as doxycycline.

 

Other studies showed that, in comparison to Doxycycline, TELOMIR-1 was more active at metal complexing. Extensive analysis is being conducted on the potential of TELOMIR-1 to protect and treat stem cells and lower blood ferritin. We have also completed a variety of toxicology studies, and several others are ongoing or planned. Upon completion of the toxicology studies, we plan to submit to the FDA an Investigational New Drug application, IND for TELOMIR-1, focused on investigating TELOMIR-1 for the treatment of hemochromatosis. We may consider a second IND for post-chemotherapy recovery. All pre-clinical studies described in this prospectus were conducted with the assistance of third parties. For a summary of completed, ongoing, and planned pre-clinical studies and development activities, see the section entitled “Business—Pre-Clinical Studies.”

 

 

4

 

 

 

Our Clinical Development Plan

 

Following completion of our pre-clinical development program, we plan to submit to the FDA an IND, focused on investigating TELOMIR-1 for the treatment of hemochromatosis. We may consider a second IND for post-chemotherapy recovery.

 

Our first IND application submission investigating TELOMIR-1 for the treatment of hemochromatosis is currently planned for the third quarter of 2024. If allowed to proceed by the FDA, a Phase I double-blind, randomized, placebo-controlled trial to evaluate the safety, tolerability, and pharmacokinetics of TELOMIR-1 in 40-60 healthy male and female adult subjects will be initiated approximately 30 days post-IND submission. We have contracted with Frontage Laboratories Phase I clinic in New Jersey consisting of 160 beds across three units for normal volunteers. After the Phase I trial is complete, a Phase II trial in the hemochromatosis population is planned to commence after results and guidance from the FDA.

 

Our second IND application will likely focus on investigating TELOMIR-1 for the treatment of post-chemotherapy recovery and is planned for submission with guidance from the FDA. Additionally, osteoarthritis may be considered as a future contender with additional guidance from the FDA.

 

Our clinical development plans will depend on FDA acceptance of our IND applications. As appropriate and pursuant to discussions with the FDA, we may periodically adjust the timeline for certain filings and associated clinical trials. It is important to note that the process for conducting clinical trials is uncertain and there is no assurance that our clinical development activities will meet the planned timelines set forth above.

 

Intellectual Property

 

We license the U.S. patent rights for the use of TELOMIR-1 in human applications from MIRALOGX, LLC (“MIRALOGX”), an intellectual property development and holding company established by Jonnie R. Williams, Sr., the founder of our company and the sole inventor of TELOMIR-1. MIRALOGX has filed a Patent Cooperation Treaty (PCT) application, PCT/US2023/073106 on August 29, 2023. The application designated the U.S. and will enter U.S. national phase. The application, if granted and subject to payment of patent maintenance fees, would offer protection extending through at least August 29, 2043 in the U.S. The patent rights for TELOMIR-1 outside of the United States are not included in our current patent rights. Mr. Williams, our founder, does not hold any formal degrees in chemistry or certifications related to the development of pharmaceuticals; however, he developed extensive knowledge of tobacco alkaloid chemistry in the 1990’s through collaboration with industry experts during his development of techniques for preventing the formation of carcinogenic tobacco-specific nitrosamines (TSNAs) during the tobacco curing process. To better understand the mechanism by which TSNAs are formed, Mr. Williams collaborated closely with a leading expert in tobacco chemistry at the University of Kentucky, where he developed a working knowledge of tobacco alkaloid chemistry, which led to his development of patented curing technologies which would become the industry standard. This work led to him focusing on the therapeutic properties of tobacco alkaloids, and later to his design of novel small molecule therapeutics. He is a named inventor on 31 issued US patents and approximately 30 pending or published US patent applications, all of which are in the life sciences, a field that embraces the study of living organisms, including biology, botany, zoology, and biochemistry. Approximately 20 of his issued patents and 27 of his applications involve therapeutic treatment methods, and 4 issued patents and approximately 18 applications are directed to novel small molecules, principally involving alkaloid and cannabinoid chemistries. His work on TELOMIR-1 began in late 2020.

 

Our license from MIRALOGX is set forth in an Amended and Restated Exclusive License Agreement, dated August 11, 2023, between the Company and MIRALOGX, pursuant to which we obtained the exclusive perpetual right and license under the above-described patent rights to make, have made, use, and sell “Licensed Products” in the U.S. for human uses and pre-clinical studies and activities of any kind conducted in furtherance of obtaining regulatory approval or commercialization for human uses (the “Initial MIRALOGX License Agreement”). On November 10, 2023, we and MIRALOGX entered into the Amendment No. 1 to the Amended and Restated License Agreement, pursuant to which the field of use relating to the license was amended to include therapeutic treatments and other medical or health uses in animals, in addition to humans, and related preclinical studies and activities conducted in furtherance of obtaining regulatory approval for and commercialization of veterinary, in addition to human, therapeutic treatments and uses (together with the “Initial MIRALOGX License Agreement, the “MIRALOGX License Agreement”). “Licensed Product” is defined in the agreement as a drug product containing as an active agent 2,4,6-tris(3,4-dihydro-2H-pyrrol-2-yl)pyridine or a pharmaceutically acceptable salt, ester, or solvate thereof. We also have the right to grant corresponding sublicenses under the licensed patent rights. The MIRALOGX License Agreement provides for the payment to MIRALOGX of an 8% royalty (payable quarterly) on our net sales of Licensed Products by us or our sublicensees and on non-royalty bearing milestone revenue. There are no up-front, execution, or milestone payments in the license agreement. Further, no payments have been made to date under the agreement. For a more detailed description of the MIRALOGX License Agreement, see the section entitled “Business—Intellectual Property.”

 

 

5

 

 

 

Summary Risk Factors

 

There are a number of risks that you should understand before making an investment decision regarding this offering. These risks are discussed more fully in the section entitled “Risk Factors” following this prospectus summary. If any of these risks actually occur, our business, financial condition, or results of operations would likely be materially and adversely affected. In such a case, the trading price of our common stock would likely decline, and you may lose all or part of your investment. These risks include, but are not limited to:

 

  We are an early development-stage company with no revenues.
     
  Management has concluded that factors raise substantial doubt about our ability to continue as a going concern and our auditor has included an explanatory paragraph relating to our ability to continue as a going concern in their audit report for the year ended December 31, 2022.
     
  Because we have a limited operating history, you may not be able to accurately evaluate our operations.
     
  We are dependent on additional financing for the continuation of our operations.
     
  Our operating results may fluctuate, which could have a negative impact on our ability to grow our client base, establish sustainable revenues and succeed overall.
     
  We have yet to achieve a profit and may not achieve a profit in the near future, if at all.
     
  Certain of our executive officers, including our Chief Executive Officer and Chairman of our board of directors, Dr. Christopher Chapman, will not be employed by us on a full-time basis and will working for us on a part-time and as-needed basis.
     
  Certain of our directors and officers may have actual or potential conflicts of interest because of their positions with other companies.
     
  Conflicts of interest may arise between us and MIRALOGX.
     
  Our future success will largely depend on the success of TELOMIR-1 and any future product candidates, which development will require significant capital resources and years of clinical development effort.
     
  We are dependent on the success of our current and future product candidates, some of which may not receive regulatory approval or be successfully commercialized.
     
  Our business may be materially and adversely affected in the future by the evolving effects of the COVID-19 pandemic as a result of the current and potential future impacts on our commercialization efforts, supply chain, regulatory and clinical development activities, and other business operations, in addition to the impact of a global economic slowdown.
     
  Results of pre-clinical studies and earlier clinical trials are not necessarily predictive indicators of future results.
     
  Should we later determine it is in our best interest to develop a sales force we may be unable to effectively train and equip our sales force, therefore our ability to successfully commercialize our products may be harmed.
     
  Our product candidates, if approved, may be unable to achieve the expected market acceptance and, consequently, limit our ability to generate revenue from new products.
     
  If the price for any future approved products decreases or if government and other third-party payers do not provide coverage and adequate reimbursement levels, our revenue and prospects for profitability will suffer.
     
  We expect to face intense competition, often from companies with greater resources and experience than we have.

 

 

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  Business interruptions could delay us in the process of developing our product candidates and could disrupt our product sales.
     
  We have significant and increasing liquidity needs and may require additional funding.
     
  Operating results may vary significantly in future periods.
     
  If product liability lawsuits are successfully brought against us, we will incur substantial liabilities and may be required to limit the commercialization of TELOMIR-1 and our product candidates.
     
  We depend upon our key personnel and our ability to attract and retain employees.
     
  Failure of our information technology systems, including cybersecurity attacks or other data security incidents, could significantly disrupt the operation of our business.
     
  Security breaches, loss of data and other disruptions could compromise sensitive information related to our business, prevent us from accessing critical information or expose us to liability, which could adversely affect our business and our reputation.
     
  Legislative or regulatory reform of the health care system in the U.S. may affect our ability to profitably sell our products, if approved.
     
  We expect additional federal and state legislative proposals for health care reform, which could limit the prices that can be charged for the products we develop and may limit our commercial opportunity.
     
  We may acquire other companies which could divert our management’s attention, result in additional dilution to our shareholders and otherwise disrupt our operations and harm our operating results.
     
  Clinical trials for our product candidates are expensive, time-consuming, uncertain, and susceptible to change, delay or termination. The results of clinical trials are open to differing interpretations.
     
  Any failure by us to comply with existing regulations could harm our reputation and operating results.
     
  The regulatory approval processes with the FDA are lengthy and inherently unpredictable.
     
  There is a high rate of failure for drug candidates proceeding through clinical trials.
     
  Our existing collaboration arrangements and any that we may enter into in the future may not be successful, which could adversely affect our ability to develop and commercialize our product candidates.
     
  We maintain our cash at financial institutions, at times in balances that exceed federally insured limits. The failure of financial institutions could adversely affect our ability to pay operational expenses or make other payments.
     
  We may not be able to adequately protect our product candidates or our proprietary technology in the marketplace.
     
  If third parties claim that the Company’s intellectual property, products, processes, or anything else used by us infringes upon their intellectual property, our operating profits could be adversely affected.
     
  Because of the speculative nature of investment risk, you may lose your entire investment.

 

 

7

 

 

 

Implications of Being an Emerging Growth Company

 

As a company with less than $1.235 billion in annual gross revenue during our last fiscal year, we qualify as an “emerging growth company” as defined in Section 2(a) of the Securities Act of 1933, as amended (the “Securities Act”). An emerging growth company may take advantage of specified reduced reporting and other requirements that are otherwise applicable generally to public companies. These provisions include:

 

  we are required to present only two years of audited financial statements and related management’s discussion and analysis of financial condition and results of operations in the registration statement of which this prospectus is a part;
     
  we are exempt from compliance with the requirement that our independent registered public accounting firm provide an attestation report on the effectiveness of our internal control over financial reporting;
     
  we are exempt from compliance with any requirement that the Public Company Accounting Oversight Board (the “PCAOB”) has adopted regarding communication of critical accounting matters and may adopt regarding mandatory audit firm rotation or a supplement to the auditor’s report providing additional information about the audit and the financial statements;
     
  we are exempt from the “say on pay,” “say when on pay,” and “say on golden parachute” non-binding advisory vote requirements; and
     
  we can provide reduced disclosures about our executive compensation arrangements.

 

We currently intend to take advantage of each of the exemptions described above. It is possible, therefore, that some investors will find our common stock less attractive, which may result in a less active trading market for our common stock and higher volatility in our stock price.

 

We may take advantage of these provisions until the last day of our fiscal year following the fifth anniversary of the completion of this offering or such an earlier time that we are no longer an emerging growth company. We would cease to be an emerging growth company upon the earliest of: (i) the last day of the first fiscal year in which our annual gross revenues are $1.235 billion or more; (ii) the date on which we have, during the previous three-year period, issued more than $1 billion in non-convertible debt securities; or (iii) the date on which we are deemed to be a “large accelerated filer,” which will occur as of the end of any fiscal year in which we (x) have an aggregate market value of our common stock held by non-affiliates of $700 million or more as of the last business day of our most recently completed second fiscal quarter, (y) have been required to file annual and quarterly reports under the Securities Exchange Act of 1934, as amended (the “Exchange Act”), for a period of at least 12 months and (z) have filed at least one annual report pursuant to the Exchange Act.

 

In addition, emerging growth companies may take advantage of the extended transition period provided in Section 7(a)(2)(B) of the Securities Act for complying with new or revised accounting standards. In other words, an emerging growth company can delay the adoption of certain accounting standards until those standards would otherwise apply to private companies. We intend to take advantage of the benefits of this extended transition period. For risks related to our status as an emerging growth company, see “Risk Factors — Risks Related to Ownership of Our Common Stock — Taking advantage of the reduced disclosure requirements applicable to “emerging growth companies” may make our common stock less attractive to investors.”

 

Implications of Being a Smaller Reporting Company

 

We are a “smaller reporting company” as defined in Item 10(f)(1) of Regulation S-K. Smaller reporting companies may take advantage of certain reduced disclosure obligations, including, among other things, providing only two years of audited financial statements. We will remain a smaller reporting company until the last day of any fiscal year for so long as either: (i) the market value of our shares of common stock held by non-affiliates does not equal or exceed $250 million as of the prior June 30th; or (ii) our annual revenues did not equal or exceed $100 million during such completed fiscal year. To the extent we take advantage of such reduced disclosure obligations, it may also make comparison of our financial statements with other public companies difficult or impossible.

 

Reverse Stock Split

 

Effective December 11, 2023, we completed a reverse stock split of our outstanding common stock upon the filing of our Second Amended and Restated Articles of Incorporation with the Florida Secretary of State. No fractional shares were or will be issued in connection with the reverse stock split, and all such fractional shares resulting from the reverse stock split were and will be rounded up to the nearest whole number. Unless otherwise noted, the shares issuable upon the exercise of our outstanding warrants, and the exercise prices of such warrants, have been adjusted to reflect the reverse stock split. Unless otherwise noted, the share and per share information in this prospectus reflects the reverse stock split.

 

Corporate Information

 

Our corporate headquarters is located at 855 N Wolfe Street, Suite 601, Baltimore, Maryland 21205. Our telephone number is (737) 289-0835.

 

Our website address is www.telomirpharma.com. The information contained on, or that can be accessed through, our website is deemed not to be incorporated in this prospectus or to be part of this prospectus. You should not consider information contained on our website to be part of this prospectus.

 

 

8

 

 

 

The Offering

 

Common stock offered by us   1,000,000 shares.
     
Initial public offering price   The assumed initial public offering price is $7.00 per share.
     
Shares of common stock outstanding before this offering (1)   28,609,814 shares.
     
Shares of common stock to be outstanding immediately after this offering   29,609,814 shares (or 29,759,814 shares if the underwriters exercise the option to purchase additional shares from us in full. See “Over-allotment Option” below).
     
Over-allotment Option   We have granted the underwriters an option exercisable for a period of 45 days from the date of this prospectus to purchase from us in whole or in part and at any time or from time to time up to 150,000 additional shares of common stock, solely to cover overallotments, if any, at a purchase price equal to the initial public offering price less underwriting discounts and commissions.
     
Use of proceeds   We estimate that we will receive net proceeds from the sale of shares of our common stock in this offering of approximately $5.9 million, assuming an initial public offering price of $7.00 per share, and after deducting underwriting discounts and commissions and estimated offering expenses payable by us. We intend to use the net proceeds from this offering to advance the clinical development of our programs, to fund our research and development activities, and for working capital and general corporate purposes. In order to advance our clinical development programs, we plan to use an estimated $3.2 million. Our management will have broad discretion in the application of the net proceeds from this offering and investors will be relying on the judgment of our management regarding the application of the proceeds. See “Use of Proceeds.”
     
Lock up   Our directors and officers, our initial stockholders holding an aggregate of 26,332,026 shares of our common stock (the “Initial Stockholders”), and other shareholders who beneficially own 3% or more of the outstanding shares of our common stock have agreed with not to offer for sale, issue, sell, contract to sell, pledge or otherwise dispose of any of our common stock or securities convertible into common stock for a period of 180 days (or 365 days for the Initial Stockholders), commencing on the date of this prospectus, except with the prior written consent of the underwriters.
     
Representative’s warrants   We have agreed to issue to the representative of the underwriters or its designees at the closing of this offering, warrants to purchase the number of shares of our common stock equal to 5.0% of the aggregate number of shares sold in this offering (the “Representative’s Warrants”). The Representative’s Warrants will be exercisable at any time and from time to time, in whole or in part, during the four-and-a-half-year period commencing six months after the commencement of sales in this offering. The exercise price of the Representative’s Warrants will equal 100% of the initial public offering price per share, subject to adjustments. The Representative’s Warrants provide for registration rights (including a one-time demand registration right and piggyback registration rights that expire 5 years from the commencement of sales of the offering) and customary anti-dilution provisions as permitted under FINRA Rule 5110(g)(8). The registration statement of which this prospectus is a part also covers the Representative’s Warrants and the shares of common stock issuable upon the exercise thereof.
     
Proposed Nasdaq Capital Market Symbol   We have applied to list our shares of common stock on Nasdaq under the symbol “TELO”. No assurance can be given that our listing will be approved by Nasdaq or that a trading market will be approved by Nasdaq or that a trading market will develop for the common stock. We will not proceed with this offering in the event the common stock is not approved for listing on Nasdaq.
     
Risk factors   Investing in our common stock involves a high degree of risk. See “Risk Factors” beginning on page 11 of this prospectus for a discussion of factors you should carefully consider before investing in our common stock.

 

  (1) The number of shares of our common stock to be outstanding immediately after this is based on an aggregate of 28,609,814 shares of our common stock outstanding as of December 12, 2023, which excludes:

 

  6,500,000 shares of our common stock reserved for future issuance under the Telomir Pharmaceuticals, Inc. 2023 Omnibus Incentive Plan, as well as any automatic increases in the number of shares of common stock reserved for future issuance under such plan:
     
  268,025 shares of our common stock to be issued upon the exercise of outstanding warrants at an exercise price of $15.42 per share; and
     
  2,506,035 shares of our common stock to be issued upon the exercise of outstanding warrants at an exercise price of $3.73 per share.

 

  Unless the context otherwise requires, the information presented in this prospectus assumes the following:

 

  that the shares of our common stock to be sold in this offering are sold at the assumed initial public offering price of $7.00 per share;
     
  no exercise by the underwriters of their over-allotment option; and
     
  no exercise by the Representative of the Representative’s Warrants.

 

 

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Summary Financial Data

 

The following tables summarize our financial data as of the dates and for the periods presented. We have derived the summary statements of operations data for the years ended December 31, 2022 and 2021, and the balance sheet data as of December 31, 2022 and 2021, from our audited financial statements included elsewhere in this prospectus. We have derived the summary statement of operations data for the three months ended September 30, 2023 and 2022, and the balance sheet data as of September 30, 2023 and 2022, from our unaudited financial statements included elsewhere in this prospectus. Our historical results are not necessarily indicative of the results that may be expected in the future.

 

The following summary financial and other data should be read in conjunction with the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and our financial statements and related notes included elsewhere in this prospectus.

 

Statement of Operations data:

 

   Three Months Ended September 30,   Year Ended December 31 
   2023   2022   2022   2021 
Revenues  $-   $-   $-   $- 
                     
Operating costs:                    
General and administrative expenses   102,191    3,750    20,941    11,639 
Related party travel costs   589,400    -    -    - 
Research and development expenses   262,562    229,747    833,206    126,492 
Total operating costs   954,153    233,497    854,147    138,131 
                     
Interest expense   (757,173)   -    -    - 
                     
Net loss  $(1,711,326)  $(233,497)  $(854,147)  $(138,131)

 

Balance Sheet data:

 

   September 30,   December 31,   December 31, 
   2023   2022   2021 
ASSETS               
Current assets:               
Cash  $2,452   $1,419   $- 
Deferred offering costs   102,894    47,311    - 
Prepaid expenses   963    -    - 
Due from related parties   130,000    -    - 
Total other current assets   236,309    48,730    - 
                
Deferred Financing Costs   5,082,292    -    - 
                
Total assets  $5,318,601   $48,730   $- 
                
LIABILITIES AND STOCKHOLDERS’ EQUITY (DEFICIT)               
Current liabilities:               
Trade accounts payable and accrued liabilities  $496,027   $404,221   $18,735 
Accrued Interest   13,517    -    - 
Due to related parties   1,293,070    581,787    119,396 
Related party line of credit   1,337,914    -    - 
Total current liabilities   3,140,528    986,008    138,131 
                
Total liabilities   3,140,528    986,008    138,131 
                
Stockholders’ Equity (Deficit)               
Preferred Stock, no par value, 100,000,000 shares authorized and none issued or outstanding.   -    -    - 
Common Stock, no par value; 300,000,000 shares authorized, 27,097,294 and 26,829,269 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively.   -    -    - 
Additional paid-in capital   6,915,000    55,000    55,000 
Accumulated deficit   (4,736,927)   (992,278)   (138,131)
Stock subscription receivable   -    -    (55,000)
Total stockholders’ equity (deficit)   2,178,073    (937,278)   (138,131)
Total liabilities and stockholders’ deficit  $5,318,601   $48,730   $- 

 

 

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RISK FACTORS

 

Investing in shares of our common stock involves a high degree of risk. You should carefully consider the risks and uncertainties described below, the section of this prospectus entitled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and our financial statements and related notes included elsewhere in this prospectus before investing in shares of our common stock. The risks and uncertainties described below are not the only ones we face. Additional risks and uncertainties that we are unaware of, or that we currently believe are not material, may also become important factors that affect us. If any of the following risks occur, our business, operating results and prospects could be materially harmed. In that event, the price of our common stock could decline, and you could lose part or all of your investment.

 

Risks Related to Our Operations and Financial Condition

 

We are an early development-stage company with no revenues.

 

As an early development-stage enterprise that is focused on the development of a pre-clinical pharmaceutical product, we have generated no revenue and have an accumulated deficit of $4.7 million through September 30, 2023, and $1.0 million through December 31, 2022. There can be no assurance that sufficient funds required to pursue our development program will be generated from operations or that funds will be available from external sources, such as debt or equity financings or other potential sources. The lack of additional capital resulting from the inability to generate cash flow from operations, or to raise capital from external sources would force us to substantially curtail or cease operations and would, therefore, have a material adverse effect on business. Furthermore, there can be no assurance that any such required funds, if available, will be available on attractive terms or that they will not have a significant dilutive effect on our existing stockholders.

 

We seek to overcome the circumstances that impact our ability to remain a going concern in the future through the growth of revenues with interim cash flow deficiencies being addressed through additional equity and debt financing. We anticipate raising additional funds through public or private financing, strategic relationships, or other arrangements in the near future to support our business operations; however, we may not have commitments from third parties for a sufficient amount of additional capital. We cannot be certain that any such financing will be available on acceptable terms, or at all, and our failure to raise capital when needed could limit our ability to continue operations. Our ability to obtain additional funding will determine our ability to continue as a going concern. Failure to secure additional financing in a timely manner and on favorable terms would have a material adverse effect on our financial performance, results of operations and stock price and require us to curtail or cease operations, sell off our assets, seek protection from our creditors through bankruptcy proceedings, or otherwise. Furthermore, additional equity financing may be dilutive to the holders of our common stock, and debt financing, if available, may involve restrictive covenants, and strategic relationships, if necessary, to raise additional funds, and may require that we relinquish valuable rights.

 

Management has concluded that factors raise substantial doubt about our ability to continue as a going concern and our auditor has included an explanatory paragraph relating to our ability to continue as a going concern in their audit report for the year ended December 31, 2022.

 

We anticipate that we will continue to report losses and negative cash flow for the foreseeable future. We have concluded that our historical recurring losses from operations and negative cash flows from operations as well as our dependence on debt and other financings raise substantial doubt about our ability to continue as a going concern and our auditor has included an explanatory paragraph relating to our ability to continue as a going concern in its audit report for the year ended December 31, 2022.

 

The consolidated financial statements of the Company do not include any adjustments that might result from the outcome of this uncertainty. These adjustments would likely include substantial impairment of the carrying amount of our assets and potential contingent liabilities that may arise if we are unable to fulfill various operational commitments. In addition, the value of our securities, including the subordinate voting shares included in this offering, would be greatly impaired. Our ability to continue as a going concern is dependent upon generating sufficient cash flow from operations and obtaining additional capital and financing. If our ability to generate cash flow from operations is delayed or reduced and we are unable to raise additional funding from other sources, we may be unable to continue in business.

 

11

 

 

Because we have a limited operating history, you may not be able to accurately evaluate our operations.

 

We have had limited operations to date. Therefore, we have a limited operating history upon which to evaluate the merits of investing in our company. Potential investors should be aware of the difficulties normally encountered by new companies and the high rate of failure of such enterprises. The likelihood of success must be considered in light of the problems, expenses, difficulties, complications, and delays encountered in connection with the operations that we plan to undertake. These potential problems include, but are not limited to, unanticipated problems relating to the ability to generate sufficient cash flow to operate our business, and additional costs and expenses that may exceed current estimates. We expect to continue to incur significant losses into the foreseeable future. We recognize that if the effectiveness of our business plan is not forthcoming, we will not be able to continue business operations. There is no history upon which to base any assumption as to the likelihood that we will prove successful, and it is doubtful that we will generate any operating revenues or ever achieve profitable operations. If we are unsuccessful in addressing these risks, our business will most likely fail.

 

We are dependent on additional financing for the continuation of our operations.

 

Because we have generated no revenues and currently operate at a loss, we are completely dependent on the continued availability of financing in order to continue our business operations. There can be no assurance that financing sufficient to enable us to continue our operations will be available to us in the future.

 

We will need additional funds to complete further development of our business plan to achieve a sustainable level where ongoing operations can be funded out of revenues. We expect that the proceeds from this Offering will provide adequate resources to fund our operations and initial clinical development programs through January 2025. We will require further funding to fully implement our business plan to its fullest potential and achieve our growth plans. There is no assurance that any additional financing will be available or if available, on terms that will be acceptable to us.

 

Our failure to obtain future financing or to produce levels of revenue to meet our financial needs could result in our inability to continue as a going concern in the future and, as a result, our investors could lose their entire investment.

 

Our operating results may fluctuate, which could have a negative impact on our ability to grow our client base, establish sustainable revenues and succeed overall.

 

Our results of operations may fluctuate as a result of a number of factors, some of which are beyond our control including but not limited to:

 

  general economic conditions in the geographies and industries where we sell our services and conduct operations; legislative policies where we sell our services and conduct operations;
     
  the budgetary constraints of our customers; seasonality;
     
  success of our strategic growth initiatives;
     
  costs associated with the launching or integration of new or acquired businesses; timing of new product introductions by us, our suppliers and our competitors; product and service mix, availability, utilization and pricing;
     
  the mix, by state and country, of our revenues, personnel, and assets; movements in interest rates or tax rates;
     
  changes in, and application of, accounting rules; changes in the regulations applicable to us; and litigation matters.

 

As a result of these factors, we may not succeed in our business, and we could go out of business.

 

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We have yet to achieve a profit and may not achieve a profit in the near future, if at all.

 

We have not yet produced any revenues or profit and may not in the near future, if at all. We cannot be certain that we will be able to realize sufficient revenue to achieve profitability. Further, many of our competitors have a significantly larger industry presence and revenue stream but have yet to achieve profitability. Our ability to continue as a going concern in the future is dependent upon raising capital from financing transactions, increasing revenue and keeping operating expenses below our revenue levels in order to achieve positive cash flows, none of which can be assured.

 

Certain of our executive officers, including our Chief Executive Officer and Chairman of our board of directors, Dr. Christopher Chapman, will not be employed by us on a full-time basis and will working for us on a part-time and as-needed basis.

 

Dr. Christopher Chapman, our Chief Executive Officer and Chairman of our board of directors, will not be employed by our company on a full-time basis. As intended to be provided in his employment agreement with our company, he is expected to work on a part-time and as-needed basis. Because he will not work full time for our company, instances may occur where he may not be immediately available to provide solutions to problems or address concerns that arise in the course of us conducting our business and thus adversely affect our business. In addition, he can become subject to conflicts of interest because he devotes part of his working time to other business endeavors and may have responsibilities to other entities. Although Dr. Chapman is aware of his duties and accountability to our company and to applicable laws and policies relating to corporate opportunity and conflicts of interest, such conflicts of interest may include deciding how much time to devote to our affairs, as well as what business opportunities should be presented to us.

 

Certain of our directors and officers may have actual or potential conflicts of interest because of their positions with other companies.

 

Following this offering, Dr. Christopher Chapman, our Chief Executive Officer and Chairman of our board of directors, will continue to serve as the Executive Chairman of MIRA Pharmaceuticals, Inc. (“MIRA”) and as a director, President, and Chief Medical Officer of MyMD Pharmaceuticals, Inc. (“MyMD”). Further, each of Christos Nicholoudis, Brad Kroenig, Talhia Tuck, and Hugh McColl serve as members of the board of directors of MIRA and will continue to serve on our board of directors following this offering, and Mr. Nicholoudis also serves as our General Counsel and the General Counsel of MIRA. Although these persons are not full-time employees of MyMD or MIRA, it is possible that the amount of time that they expend on their work for other companies may adversely impact the amount of time that they can spend on their work for our company. These persons may also own or acquire shares of MyMD or MIRA common stock and options to purchase such common stock. Their respective positions at MyMD or MIRA, as applicable, and the ownership of any equity or equity awards of MyMD or MIRA, as applicable, creates, or may create the appearance of, conflicts of interest when these individuals are faced with decisions that could have different implications for MyMD or MIRA than the decisions have for us.

 

Conflicts of interest may arise between us and MIRALOGX.

 

MIRALOGX holds the patent rights to TELOMIR-1. MIRALOGX is a separate intellectual property development company owned by the Bay Shore Trust, which is an irrevocable trust established by the Company’s founder, Jonnie R. Williams, Sr., and in which Brian McNulty is the trustee. The Bay Shore Trust is also our largest stockholder. Additionally, our General Counsel and one of our directors, Christos Nicholoudis, performs certain consulting work for MIRALOGX through his law firm, The Law Firm of Christos Nicholoudis PLLC, on an as-needed basis. We have an exclusive license from MIRALOGX to develop and commercialize TELOMIR-1 in the U.S. for human and non-human applications. Although the interests of MIRALOGX are 100% owned by the Bay Shore Trust, and neither Mr. Williams nor Mr. Nicholoudis is an officer or director of MIRALOGX and Mr. Williams does not have voting or dispositive power over the shares of the Company held by Bay Shore Trust, our relationship with the Bay Shore Trust, Mr. Williams, or Mr. Nicholoudis may create, or may create the appearance of, conflicts of interest when we are faced with decisions that could have different implications for MIRALOGX than the decisions have for us. Furthermore, in light of the license agreement that we have with MIRALOGX, if a dispute were to arise between MIRALOGX and us relating to our past or future relationship with MIRALOGX or with respect to intellectual property matters, these potential conflicts of interest may make it more difficult for us to favorably resolve such disputes.

 

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Risks Relating to Our Business and Our Industry

 

Our future success will largely depend on the success of TELOMIR-1 and any future product candidates, which development will require significant capital resources and years of clinical development effort.

 

We currently have no drug products on the market, and all of our drug development projects are in a pre-clinical stage of development. Our business depends almost entirely on the successful pre-clinical and clinical development, FDA regulatory approval, and commercialization of our product candidates, principally TELOMIR-1. Investors need to be aware that substantial additional investments including pre-clinical and clinical development and FDA regulatory submission and approval efforts will be required before we are permitted to undertake clinical studies and market and commercialize our product candidates, if ever. It may be several years before we can commence clinical trials, if ever. Any clinical trial will be subject to extensive and rigorous review and regulation by numerous government authorities in the United States and other jurisdictions where we intend, if approved, to market our product candidates. Before obtaining regulatory approvals for any of our product candidates, we must demonstrate through pre-clinical testing and clinical trials that the product candidate is safe and effective for its specific application. This process can take many years and may include post- marketing studies and surveillance, which would require the expenditure of substantial resources. Of the large number of drugs in development for approval in the United States (and the rest of the world), only a small percentage will successfully complete the FDA regulatory approval financing to fund our planned research, development, and clinical programs, we cannot assure you that any of our product candidates will be successfully developed or commercialized.

 

We may be unable to formulate or scale up any or all of our product candidates. There is no guarantee that any of the product candidates will be or are able to be manufactured or produced in a manner to meet the FDA’s criteria for product stability, content uniformity and all other criteria necessary for product approval in the United States and other markets. Any of our product candidates may fail to achieve their specified endpoints in clinical trials.

 

Furthermore, product candidates may not be approved even if they achieve their specified endpoints in clinical trials. The FDA may disagree with our trial design and our interpretation of data from clinical trials or may change the requirements for approval even after it has reviewed and commented on the design for our clinical trials. The FDA may also approve a drug for fewer or more limited indications than we request or may grant approval contingent on the performance of costly post-approval clinical trials (i.e., Phase IV trials). In addition, the FDA may not approve the labeling claims that we believe are necessary or desirable for the successful commercialization of our product candidates.

 

14

 

 

If we are unable to expand our pipeline and obtain regulatory approval for our product candidates within the timelines we anticipate, we will not be able to execute our business strategy effectively and our ability to substantially grow our revenues will be limited, which would have a material adverse impact on our long-term business, results of operations, financial condition, and prospects.

 

We are dependent on the success of our current and future product candidates, some of which may not receive regulatory approval or be successfully commercialized.

 

Our success will depend on our ability to successfully commercialize our product candidates. Our ability to successfully commercialize our product candidates will depend on, among other things, our ability to:

 

  successfully complete pre-clinical and other nonclinical studies and clinical trials;
     
  receive regulatory approvals from the FDA;
     
  produce, through a validated process, in manufacturing facilities inspected and approved by regulatory authorities, including the FDA, sufficiently large quantities of product candidates to permit successful commercialization;
     
  obtain reimbursement from payers such as government health care programs and insurance companies and achieve commercially attractive levels of pricing;
     
  secure acceptance of our product candidates from physicians, health care payers, patients, and the medical community;
     
  create positive publicity surrounding our product candidates;
     
  manage our spending as costs and expenses increase due to clinical trials and commercialization; and
     
  obtain and enforce sufficient intellectual property for our product candidates.

 

Our failure or delay with respect to any of the factors above could have a material adverse effect on our business, results of operations and financial condition.

 

Our business may be materially and adversely affected in the future by the evolving effects of the COVID-19 pandemic as a result of the current and potential future impacts on our commercialization efforts, supply chain, regulatory and clinical development activities, and other business operations, in addition to the impact of a global economic slowdown.

 

Our business could be materially and adversely affected in the future by the evolving effects of the COVID-19 pandemic. If we are unable to obtain adequate supplies of personal protective equipment due to shortages or encounter other challenges related to the evolving COVID-19 pandemic, we may have to place or may experience additional limitations on our in-person activities. In addition, our increased reliance on personnel working from home may negatively impact productivity or disrupt, delay or otherwise adversely impact our business. This could also increase our cybersecurity risk, create data accessibility concerns, and make us more susceptible to communication disruptions, any of which could adversely impact our business operations. Impacts related to the COVID-19 pandemic could materially and adversely affect our business, our ability to generate sales of and revenues from our approved products, and our ability to advance the development of our products and product candidates, as described elsewhere in this “Risk Factors” section. The magnitude of such impacts will depend, in large part, on the ultimate duration and severity of the evolving effects of the COVID-19 pandemic.

 

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The effects of the COVID-19 pandemic continue to rapidly evolve. These effects have increased market volatility and could result in a significant long-term disruption of global financial markets, reducing our ability to access capital, which could in the future negatively affect our liquidity. In addition, market corrections resulting from the effects of the COVID-19 pandemic could materially affect our business and the value of our common stock. The extent to which the evolving effects of the COVID-19 pandemic impact our business, our ability to generate sales of and revenues from our approved products, and our clinical development and regulatory efforts will depend on future developments that are highly uncertain and cannot be predicted with confidence, such as the ultimate duration and severity of the pandemic, government actions, such as travel restrictions, quarantines and social distancing requirements in the U.S. and in other countries, business closures or business disruptions and the effectiveness of actions taken in the U.S. and in other countries to contain and treat the disease. Accordingly, we do not yet know the full extent of potential delays or impacts on our business, sales of our products, our clinical and regulatory activities, our research programs, healthcare systems or the global economy as a whole. However, these effects could materially and adversely affect our business, financial condition, results of operations and growth prospects. In addition, to the extent the evolving effects of the COVID-19 pandemic adversely affect our business, financial condition, results of operations and growth prospects, they may also have the effect of heightening many of the other risks and uncertainties described elsewhere in this “Risk Factors” section. It is also possible that future global pandemics could also occur and materially and adversely affect our business, financial condition, results of operations and growth prospects.

 

Results of pre-clinical studies and earlier clinical trials are not necessarily predictive indicators of future results.

 

Any positive results from future pre-clinical testing of our product candidates and potential future clinical trials may not necessarily be predictive of the results from Phase I, Phase II or Phase III clinical trials. In addition, our interpretation of results derived from clinical data or our conclusions based on our pre-clinical data may prove inaccurate. Frequently, pharmaceutical and biotechnology companies have suffered significant setbacks in clinical trials after achieving positive results in pre-clinical testing and early phase clinical trials, and we cannot be certain that we will not face similar setbacks. These setbacks may be caused by the fact that pre-clinical and clinical data can be susceptible to varying interpretations and analyses. Furthermore, certain product candidates may perform satisfactorily in pre-clinical studies and clinical trials, but nonetheless fail to obtain FDA approval or appropriate approvals by the appropriate regulatory authorities in other countries. If we fail to produce positive results in our clinical trials for our product candidates, the development timeline and regulatory approval and commercialization prospects for them and as a result our business and financial prospects, would be materially adversely affected.

 

We have limited marketing experience, and we do not anticipate at this time establishing a sales force or distribution and reimbursement capabilities, and we may not be able to successfully commercialize any of our product candidates if they are approved in the future.

 

Our ability to generate revenues ultimately depends on our ability to sell our approved products and secure adequate third-party reimbursement. We currently have limited experience in marketing and selling our products. We currently do not have any products approved for sale in the United States or in any other country.

 

The commercial success of our product candidates will depend on a number of factors beyond our control, including the willingness of physicians to prescribe our products to patients, payers’ willingness and ability to pay for the drugs, the level of pricing achieved, patients’ response to our drugs and the ability of our marketing partners to generate sales. There can be no guarantee that we will be able to establish or maintain the personnel, systems, arrangements and capabilities necessary to successfully commercialize TELOMIR-1 or any product candidate approved by the FDA in the future. If we fail to establish or maintain successful marketing, sales and reimbursement capabilities or fail to enter into successful marketing arrangements with third parties, our product revenues may suffer.

 

Should we later determine it is in our best interest to develop a sales force we may be unable to effectively train and equip our sales force, therefore our ability to successfully commercialize our products may be harmed.

 

We will be required to expend significant time and resources to train our sales force to be credible, persuasive, and compliant with applicable laws in marketing TELOMIR-1 or our other product candidates to physicians for their approved uses. In addition, we must continue to train our sales force to ensure that a consistent and appropriate message about TELOMIR-1 or our other product candidates is being delivered to our potential customers. If we are unable to effectively train our sales force and equip them with effective materials, including medical and sales literature to help them inform and educate potential customers about the benefits of TELOMIR-1 and our product candidates and its proper administration, our efforts to successfully commercialize TELOMIR-1 and our product candidates could be jeopardized, which would negatively impact our ability to generate product revenues.

 

16

 

 

We will need to further increase the size and complexity of our organization in the future, and we may experience difficulties in managing our growth and executing our growth strategy.

 

Our management and personnel, systems, and facilities currently in place may not be adequate to support our business plan and future growth. As a result, we may need to further expand certain areas of our organization.

 

Our need to effectively manage our operations, growth and various projects requires that we:

 

  continue to improve our operational, financial, management and regulatory compliance controls and reporting systems and procedures;
     
  attract and retain enough talented employees;
     
  manage our clinical trials effectively;

 

  manage our external manufacturing operations with contract research organizations effectively and in a cost-effective manner;
     
  manage our development efforts effectively while carrying out our contractual obligations to contractors and other third parties; and

 

In addition, we may utilize the services of part-time outside consultants and contractors to perform several tasks for us, including tasks related to compliance programs, clinical trial management, regulatory affairs, formulation development and other drug development functions. Our growth strategy may entail expanding our use of consultants and contractors to implement these and other tasks going forward. If we are not able to effectively expand our organization by hiring new employees and expanding our use of consultants and contractors, we may be unable to successfully implement the tasks necessary to effectively execute on our planned research, development, manufacturing, and commercialization activities and, accordingly, may not achieve our research, development and commercialization goals.

 

Our product candidates, if approved, may be unable to achieve the expected market acceptance and, consequently, limit our ability to generate revenue from new products.

 

Even when product development is successful and regulatory approval has been obtained, our ability to generate sufficient revenue depends on the acceptance of our products by physicians and patients. We cannot assure you that our product candidates will achieve the expected level of market acceptance and revenue if and when they obtain the requisite regulatory approvals. The market acceptance of any product depends on a number of factors, including the indication statement and warnings required by regulatory authorities in the product label. Market acceptance can also be influenced by continued demonstration of efficacy and safety in commercial use, physicians’ willingness to prescribe the product, reimbursement from third-party payers such as government health care programs and private third-party payers, the price of the product, the nature of any post-approval risk, management activities mandated by regulatory authorities, competition, and marketing and distribution support. Further, an ineffective or inefficient distribution model at launch may lead to the inability to fulfill demand, and consequently a loss of revenue. Any factors preventing or limiting the market acceptance of our products could have a material adverse effect on our business, results of operations and financial condition.

 

17

 

 

If the price for any future approved products decreases or if government and other third-party payers do not provide coverage and adequate reimbursement levels, our revenue and prospects for profitability will suffer.

 

Patients who are prescribed medicine for the treatment of their conditions generally rely on third-party payers to reimburse all or part of the costs associated with their prescription drugs. Reimbursement systems in international markets vary significantly by country and by region, and reimbursement approvals generally must be obtained on a country-by-country basis. Coverage and adequate reimbursement from governmental healthcare programs, such as Medicare and Medicaid, and commercial payers is critical to new product acceptance. Coverage decisions may depend upon clinical and economic standards that disfavor new drug products when more established or lower-cost therapeutic alternatives are already available or subsequently become available. Even if we obtain coverage for products we may market, the resulting reimbursement payment rates may require co-payments that patients find unacceptably high. Patients may not use our products if coverage is not provided, or reimbursement is inadequate to cover a significant portion of its cost.

 

In addition, the market for our products will depend significantly on access to third-party payers’ drug formularies or lists of medications for which third-party payers provide coverage and reimbursement. The industry competition to be included in such formularies often leads to downward pricing pressures on pharmaceutical companies. Also, third-party payers may refuse to include a particular branded drug in their formularies or otherwise restrict patient access to a branded drug when a less costly generic equivalent or other alternative is available, even if not approved for the indications for which our products are approved.

 

Third-party payers or governmental or commercial entities are developing increasingly sophisticated methods of controlling healthcare costs. The current environment is putting pressure on companies to price products below what they may feel is appropriate. Selling our products at less than an optimized price could impact our revenues and overall success as a company. It will be difficult to determine the optimized price for our products. In addition, in the U.S., no uniform policy of coverage and reimbursement for drug products exists among third-party payers. Therefore, coverage and reimbursement for our products may differ significantly from payer to payer. As a result, the coverage determination process is often a time-consuming and costly process that will require us to provide scientific and clinical support for the use of our products to each payer separately, with no assurance that coverage will be obtained. If we are unable to obtain coverage of, and adequate payment levels for, products we may market to third-party payers, physicians may limit how much or under what circumstances they will prescribe or administer them, and patients may decline to purchase them. This in turn could affect our ability to successfully commercialize products we may market, and thereby adversely impact our profitability, results of operations, financial condition, and future success.

 

In addition, where we have chosen to collaborate with a third party on product candidate development and commercialization, our partner may elect to reduce the price of our products in order to increase the likelihood of obtaining reimbursement approvals. In many countries, products cannot be commercially launched until reimbursement is approved and the negotiation process in some countries can exceed 12 months. In addition, pricing and reimbursement decisions in certain countries can be affected by decisions taken in other countries, which can lead to mandatory price reductions and/or additional reimbursement restrictions across a number of other countries, which may thereby adversely affect our sales and profitability. If countries impose prices that are not sufficient to allow us or our partners to generate a profit, our partners may refuse to launch the product in such countries or withdraw the product from the market, which would adversely affect sales and profitability. Events, such as price decreases, government mandated rebates or unfavorable reimbursement decisions, could affect the pricing and reimbursement of TELOMIR-1 and our other product candidates and could have a material adverse effect on our business, reputation, results of operations and financial condition.

 

We expect to face intense competition, often from companies with greater resources and experience than we have.

 

The development and commercialization of drugs and medicines is highly competitive. We compete with a variety of multinational pharmaceutical companies and specialized biotechnology companies, as well as products and processes being developed by universities and other research institutions. Many of our competitors have developed, are developing, or will develop drugs and processes which may be competitive with our drug candidates. Competitive products include those that have already been approved by medicines regulators and accepted by the medical community and any new products that may enter the market. For some of our drug development programs / areas of interest, other treatment options or products are currently available, under development, and may become commercially available in the future. If any of our product candidates are approved for the diseases and conditions we are currently pursuing, they may compete with a range of medicines or therapeutic treatments that are either in development, will be developed in the future or currently marketed.

 

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Established companies may have a competitive advantage over us due to their size and experiences, financial resources, and institutional networks. Many of our competitors may have significantly greater financial, technical, and human resources than we do. Due to these factors, our competitors may have an advantage in marketing their approved drugs and may obtain regulatory approval of their drug candidates before we are able to, which may limit our ability to develop or commercialize our drug candidates. Our competitors may also develop drugs or medicines that are safer, more effective, more widely used and less expensive than ours. These advantages could materially impact our ability to develop and, if approved, commercialize our product candidates successfully. Furthermore, some of these competitors may make acquisitions or establish collaborative relationships among themselves or with third parties to increase their ability to rapidly gain market share.

 

Moreover, as generic versions of drug products enter the market, the price for such medicines may be expected to decline rapidly and substantially. Even if we are the first to obtain FDA approval of one of our product candidates, the future potential approval of generics could adversely affect the price we are able to charge, and the profitability of our product(s) will likely decline.

 

Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in more resources being concentrated among a smaller number of our competitors. Smaller and other early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and established companies.

 

These companies may compete with us in recruiting and retaining qualified scientific, management and commercial personnel, utilizing contract manufacturing facilities or contract research organizations (CROs), or establishing clinical trial sites and subject registration for clinical trials, as well as in acquiring technologies complementary to our research projects.

 

Problems in our manufacturing process, failure to comply with manufacturing regulations or unexpected increases in our manufacturing costs could harm our business, results of operations and financial condition.

 

The manufacturing of our product candidates necessitates compliance with cGMP and other regulatory requirements in jurisdictions internationally. We must ensure chemical consistency among our batches of products, including clinical batches and, if approved, marketing batches. Demonstrating such consistency may require typical manufacturing controls as well as clinical data. We must also ensure that our batches conform to complex release specifications. If we are unable to manufacture our product candidates in accordance with regulatory specifications, including cGMP, or if there are disruptions in our manufacturing process due to damage, loss or otherwise, or failure to pass regulatory inspections of our manufacturing facilities, we may not be able to meet current demand or supply sufficient product for use in clinical trials, and this may also harm our ability to commercialize our product candidates on a timely or cost-competitive basis, if at all.

 

We may fail to expand our manufacturing capability in time to meet market demand for our products and product candidates, and the FDA may refuse to accept our facilities or those of our contract manufacturers as being suitable for the production of our products and product candidates. Any problems in our manufacturing process could have a material adverse effect on our business, results of operations and financial condition.

 

In addition, before we can begin commercial manufacture of any product candidates for sale in the U.S., we must obtain FDA regulatory approval for the product, which requires a successful FDA inspection of our manufacturing facilities and those of our contract manufacturers, processes, and quality systems in addition to other product-related approvals. Although we may successfully navigate this pre-approval inspection process as it relates in the U.S., pharmaceutical manufacturing facilities are continuously subject to post-approval inspection by the FDA and foreign regulatory authorities. Due to the complexity of the processes used to manufacture our product candidates, we may be unable to initially or continue to pass federal, state or international regulatory inspections in a cost-effective manner. If we are unable to comply with manufacturing regulations, we may be subject to fines, unanticipated compliance expenses, recall or seizure of any approved products, total or partial suspension of production and/or enforcement actions, including injunctions, and criminal or civil prosecution. These possible sanctions would adversely affect our business, results of operations and financial condition.

 

19

 

 

Business interruptions could delay us in the process of developing our product candidates and could disrupt our product sales.

 

Our research and development activities are conducted through outside contractors and manufacturers. Loss of our contracted manufacturing facilities, stored inventory or laboratory facilities through fire, theft or other causes, or loss of our raw material, could have an adverse effect on our ability to continue product development activities and to conduct our business. Failure to supply our partners with commercial product may lead to adverse consequences, including the right of partners to take over responsibility for product supply. We currently do not have insurance coverage to compensate us for such business interruptions. Our contract manufacturers and suppliers provide that in their separate operations; however, such coverage may prove insufficient to fully compensate us for the damage to our business resulting from any significant property or casualty loss to those facilities.

 

We have significant and increasing liquidity needs and may require additional funding.

 

Our operations have consumed substantial amounts of cash since inception. For the nine months ended September 30, 2023, we reported a net operating cash outflow of $2.8 million and a net cash inflow from financing activities of $2.8 million. For the year ended December 31, 2022, we reported a net operating cash outflow of $0.5 million and a net cash inflow from investing activities of $0.5 million. For the year ended December 31, 2021, we reported a net operating cash outflow of $0.1 million and a net cash inflow from investing activities of $0.1 million.

 

Research and development, and general and administrative expenses, and cash used for operations will continue to be significant and may increase substantially in the future in connection with new research and development initiatives and continued product commercialization efforts. We may need to raise additional capital to fund our operations, continue to conduct clinical trials to support potential regulatory approval of marketing applications and to fund commercialization of our products.

 

The amount and timing of our future funding requirements will depend on many factors, including, but not limited to:

 

  the timing of FDA approval, if any, and approvals in international markets of our product candidates, if at all;
  the timing and amount of revenue from sales of our products, or revenue from grants or other sources;
  the rate of progress and cost of our clinical trials and other product development programs;
  costs of establishing or outsourcing sales, marketing, and distribution capabilities;
  costs and timing of completion of expanded in-house manufacturing facilities as well as any outsourced commercial manufacturing supply arrangements for our product candidates;
  costs of filing, prosecuting, defending, and enforcing any patent claims and other intellectual property rights associated with our product candidates;
  the effect of competing technological and market developments;
  personnel, facilities, and equipment requirements; and
  the terms and timing of any additional collaborative, licensing, co-promotion, or other arrangements that we may establish.

 

While we expect to fund our future capital requirements from several sources including existing cash balances, future cash flows from operations and the proceeds from equity offerings, we cannot assure you that any of these funding sources will be available to us on favorable terms, or at all. Further, even if we can raise funds from all of the above sources, the amounts raised may not be sufficient to meet our future capital requirements.

 

Operating results may vary significantly in future periods.

 

Our expenses and operating results have fluctuated in the past and our revenues, expenses, and operating results are likely to fluctuate significantly in the future. Our financial results are unpredictable and may fluctuate, for among other reasons, due to:

 

  commercial sales of our products;

 

  our achievement of product development objectives and milestones;
     
  clinical trial enrollment and expenses;
     
  research and development expenses; and
     
  the timing and nature of contract manufacturing and contract research payments.

 

20

 

 

A high portion of our costs are predetermined on an annual basis, due in part to our significant research and development costs. Thus, small declines in revenue could disproportionately affect financial results in a quarter. Because of these factors, our financial results in one or more future quarters may fail to meet the expectations of securities analysts or investors, which could cause our share price to decline.

 

If product liability lawsuits are successfully brought against us, we will incur substantial liabilities and may be required to limit the commercialization of TELOMIR-1 and our product candidates.

 

Although we have never had any product liability claims or lawsuits brought against us, we face potential product liability exposure related to the testing of our product candidates in human clinical trials. We may face exposure to claims by an even greater number of persons when we begin to market and distribute our products commercially in the U.S., Europe and elsewhere. Now, and in the future, an individual may bring a liability claim against us alleging that TELOMIR-1 or one of our product candidates caused an injury. While we continue to take what we believe are appropriate precautions, we may be unable to avoid significant liability if any product liability lawsuit is brought against us. Large judgments have been awarded in class action or individual lawsuits based on drugs that had unanticipated side effects. If we cannot successfully defend ourselves against product liability claims, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:

 

  decreased demand for TELOMIR-1 and our product candidates if such product candidates are approved;
  injury to our reputation;
  withdrawal of clinical trial participants;
  costs of related litigation;
  substantial monetary awards to patients and others;
  increased cost of liability insurance;
  loss of revenue; and
  the inability to successfully commercialize our products.

 

Counterfeit versions of our products could harm our business.

 

Counterfeiting activities and the presence of counterfeit products in a number of markets and over the Internet continue to be a challenge for maintaining a safe drug supply for the pharmaceutical industry. Counterfeit products are frequently unsafe or ineffective and can be life-threatening. To distributors and users, counterfeit products may be visually indistinguishable from the authentic version. Reports of adverse reactions to counterfeit drugs along with increased levels of counterfeiting could be mistakenly attributed to the authentic product, affect patient confidence in the authentic product and harm the business of companies such as ours. If our products were to be the subject of counterfeits, we could incur reputational and financial harm.

 

We depend upon our key personnel and our ability to attract and retain employees.

 

Our future growth and success depend on our ability to recruit, retain, manage, and motivate our employees. The inability to hire or retain experienced management personnel could adversely affect our ability to execute our business plan and harm our operating results. Due to the specialized scientific and managerial nature of our business, we rely heavily on our ability to attract and retain qualified scientific, technical, and managerial personnel. The competition for qualified personnel in the pharmaceutical field is intense. Due to this intense competition, we may be unable to continue to attract and retain the qualified personnel necessary for the development of our business or to recruit suitable replacement personnel.

 

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Our employees may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.

 

We are exposed to the risk of employee fraud or other misconduct. Misconduct by employees could include intentional failures to comply with FDA or foreign regulations, provide accurate information to FDA or other regulatory authorities, comply with applicable manufacturing standards, comply with other foreign, federal, and state laws and regulations, report information or data accurately or disclose unauthorized activities to us. Employee misconduct could also involve the improper use of information, including information obtained during clinical trials, or illegal appropriation of drug products, which could result in government investigations and serious harm to our reputation. The precautions we take to detect and prevent these prohibited activities may not be effective in controlling unknown or unmanaged risks or losses or in protecting us from governmental investigations or other actions or lawsuits stemming from a failure to comply with such laws or regulations. If any such actions are instituted against us, and we are not successful in defending ourselves or asserting our rights, those actions could have a significant impact on our business, including the imposition of significant fines or other sanctions.

 

We are subject to the U.S. Foreign Corrupt Practices Act and other anti-corruption laws, as well as export control laws, customs laws, sanctions laws and other laws governing our operations. If we fail to comply with these laws, we could be subject to civil or criminal penalties, other remedial measures, and legal expenses, which could adversely affect our business, results of operations and financial condition.

 

Our operations are subject to anti-corruption laws, including the U.S. Foreign Corrupt Practices Act of 1977, as amended (the “FCPA”), and other anti-corruption laws that apply in countries where we do business. The FCPA and these other laws generally prohibit us and our employees and intermediaries from bribing, being bribed or making other prohibited payments to government officials or other persons to obtain or retain business or gain some other business advantage. We and our commercial partners operate in a number of jurisdictions that pose a high risk of potential FCPA violations, and we participate in collaborations and relationships with third parties whose actions could potentially subject us to liability under the FCPA or local anti-corruption laws. In addition, we cannot predict the nature, scope, or effect of future regulatory requirements to which our international operations might be subject or the manner in which existing laws might be administered or interpreted.

 

We are also subject to other laws and regulations governing our international operations, including regulations administered by the government of the U.S. and other countries in which we operate or plan to operate, including applicable export control regulations, economic sanctions on countries and persons, customs requirements, and currency exchange regulations, (collectively referred to as the “Trade Control laws”).

 

However, there is no assurance that we will be completely effective in ensuring our compliance with all applicable anti-corruption laws, including the FCPA or other legal requirements, including Trade Control laws. If we are not in compliance with the FCPA and other anti-corruption laws or Trade Control laws, we may be subject to criminal and civil penalties, disgorgement and other sanctions and remedial measures, and legal expenses, which could have an adverse impact on our business, financial condition, results of operations and liquidity, as well as our reputation. Likewise, any investigation of any potential violations of the FCPA, other anti-corruption laws or Trade Control laws by the U.S. or other authorities could also have an adverse impact on our reputation, our business, results of operations and financial condition.

 

Our proprietary information, or that of our customers, suppliers, and business partners, may be lost or we may suffer security breaches.

 

In the ordinary course of our business, we will collect and store sensitive data, including valuable and commercially sensitive intellectual property, clinical trial data, our proprietary business information and that of our customers, suppliers and business partners, and personally identifiable information of our customers, clinical trial subjects and employees, and patients, on our networks, and with our third-party cloud service providers. The secure processing, maintenance and transmission of this information is critical to our operations. Despite our security measures, our information technology and infrastructure, and that of our third parties, may be vulnerable to attacks by hackers or breached due to employee error, malfeasance, or other disruptions. Any breach could compromise our networks and the information stored there could be accessed, publicly disclosed, lost, or stolen. Any such access, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, regulatory penalties, disrupt our operations, damage our reputation, and cause a loss of confidence in our products and our ability to conduct clinical trials, which could adversely affect our business and reputation and lead to delays in gaining regulatory approvals for TELOMIR-1 or other product candidates.

 

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Failure of our information technology systems, including cybersecurity attacks or other data security incidents, could significantly disrupt the operation of our business.

 

Our business is increasingly dependent on critical, complex, and interdependent information technology (“IT”) systems, including internet-based systems, some of which are managed or hosted by third parties, to support business processes as well as internal and external communications. The size and complexity of our IT systems make us potentially vulnerable to IT system breakdowns, malicious intrusion, and computer viruses, which may result in the impairment of our ability to operate our business effectively.

 

We are continuously evaluating and, where appropriate, enhancing our IT systems to address our planned growth, including to support our planned manufacturing operations. There are inherent costs and risks associated with implementing the enhancements to our IT systems, including potential delays in access to, or errors in, critical business and financial information, substantial capital expenditures, additional administrative time and operating expenses, retention of sufficiently skilled personnel to implement and operate the enhanced systems, demands on management time, and costs of delays or difficulties in transitioning to the enhanced systems, any of which could harm our business and results of operations. In addition, the implementation of enhancements to our IT systems may not result in productivity improvements at a level that outweighs the costs of implementation, or at all. In addition, our systems and the systems of our third-party providers and collaborators are potentially vulnerable to data security breaches which may expose sensitive data to unauthorized persons or to the public. Such data security breaches could lead to the loss of confidential information, trade secrets or other intellectual property, could lead to the public exposure of personal information (including personally identifiable information or individually identifiable health information) of our employees, clinical trial patients, customers, business partners, and others, could lead to potential identity theft, or could lead to reputational harm. Data security breaches could also result in loss of clinical trial data or damage to the integrity of that data. In addition, the increased use of social media by our employees and contractors could result in inadvertent disclosure of sensitive data or personal information, including but not limited to, confidential information, trade secrets and other intellectual property.

 

Any such disruption or security breach, as well as any action by us or our employees or contractors that might be inconsistent with the rapidly evolving data privacy and security laws and regulations applicable within the United States and elsewhere where we conduct business, could result in enforcement actions by U.S. states, the U.S. federal government or foreign governments, liability or sanctions under data privacy laws, including healthcare laws such as HIPAA, that protect certain types of sensitive information, regulatory penalties, other legal proceedings such as but not limited to private litigation, the incurrence of significant remediation costs, disruptions to our development programs, business operations and collaborations, diversion of management efforts and damage to our reputation, which could harm our business and operations. Because of the rapidly moving nature of technology and the increasing sophistication of cybersecurity threats, our measures to prevent, respond to and minimize such risks may be unsuccessful.

 

Security breaches, loss of data and other disruptions could compromise sensitive information related to our business, prevent us from accessing critical information or expose us to liability, which could adversely affect our business and our reputation.

 

In the ordinary course of our business, we, our vendors, and our third-party cloud service providers may collect and store sensitive data, including legally protected patient health information, credit card information, personally identifiable information about our employees and patients, intellectual property, and proprietary business information. We manage and maintain our applications and data utilizing cloud-based and on-site systems. These applications and data encompass a wide variety of business-critical information including research and development information, commercial information and business and financial information.

 

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The secure processing, storage, maintenance, and transmission of this critical information is vital to our operations and business strategy, and we devote significant resources to protecting such information. Although we take measures to protect sensitive information from unauthorized access or disclosure, our information technology and infrastructure may be vulnerable to attacks by hackers, or viruses, breaches, or interruptions due to employee error, malfeasance or other disruptions, or lapses in compliance with privacy and security mandates. Any such virus, breach or interruption could compromise our networks and the information stored there could be accessed by unauthorized parties, publicly disclosed, lost or stolen. We have measures in place that are designed to prevent, and if necessary to detect and respond to such security incidents, breaches of privacy, and security mandates. However, in the future, any such access, disclosure or other loss of information could result in legal claims or proceedings, liability under laws that protect the privacy of personal information, such as HIPAA in the United States and the General Data Protection Regulation in the European Union, or GDPR, government enforcement actions and regulatory penalties. Unauthorized access, loss or dissemination could also disrupt our operations, including our ability to process samples, provide test results, share and monitor safety data, bill payers or patients, provide customer support services, conduct research and development activities, process and prepare company financial information, manage various general and administrative aspects of our business and may damage our reputation, any of which could adversely affect our business, financial condition and results of operations.

 

Legislative or regulatory reform of the health care system in the U.S. may affect our ability to profitably sell our products, if approved.

 

Our ability to commercialize our future products successfully, alone or with collaborators, will depend in part on the extent to which coverage and reimbursement for the products will be available from government and health administration authorities, private health insurers and other third-party payers. The continuing efforts of the U.S., European, and foreign governments, insurance companies, managed care organizations and other payers for health care services to contain or reduce health care costs may adversely affect our ability to set prices for our products which we believe are fair, and our ability to generate revenues and achieve and maintain profitability.

 

Specifically, in both the U.S. and some foreign jurisdictions, there have been a number of legislative and regulatory proposals to change the health care system in ways that could affect our ability to sell our products profitably.

 

We expect additional federal and state legislative proposals for health care reform, which could limit the prices that can be charged for the products we develop and may limit our commercial opportunity.

 

The continuing efforts of government and other third-party payers to contain or reduce the costs of health care through various means may limit our commercial opportunity. It will be time-consuming and expensive for us to go through the process of seeking coverage and reimbursement from Medicare, Medicaid, and other governmental health programs and from private payers. Our products may not be considered cost-effective, and government and third-party private health insurance coverage and reimbursement may not be available to patients for any of our future products or sufficient to allow us to sell our products on a competitive and profitable basis. Our results of operations could be adversely affected by the Patient Protection and Affordable Care Act (the “ACA”), changes to the ACA, and by other health care reforms that may be enacted or adopted in the future. In addition, increasing emphasis on managed care in the U.S. will continue to put downward pressure on the pricing of pharmaceutical products. Cost-control initiatives could decrease the price that we or any potential collaborators could receive for any of our future products and could adversely affect our ability to generate revenue in the U.S. market and maintain profitability.

 

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We may acquire other companies which could divert our management’s attention, result in additional dilution to our shareholders and otherwise disrupt our operations and harm our operating results.

 

We may in the future seek to acquire businesses, products, or technologies that we believe could complement or expand our product offerings, enhance our technical capabilities or otherwise offer growth opportunities. The pursuit of potential acquisitions may divert the attention of management and cause us to incur various expenses in identifying, investigating, and pursuing suitable acquisitions, whether or not they are consummated. If we acquire additional businesses, we may not be able to integrate the acquired personnel, operations and technologies successfully, effectively manage the combined business following the acquisition or realize anticipated cost savings or synergies. We also may not achieve the anticipated benefits from the acquired business due to a number of factors, including:

 

  incurrence of acquisition-related costs;
     
  diversion of management’s attention from other business concerns;
     
  unanticipated costs or liabilities associated with the acquisition;
     
  harm to our existing business relationships with collaboration partners as a result of the acquisition;
     
  harm to our brand and reputation;
     
  the potential loss of key employees;
     
  use of resources that are needed in other parts of our business; and
     
  use of substantial portions of our available cash to consummate the acquisition.

 

In the future, if our acquisitions do not yield expected returns, we may be required to take charges to our operating results arising from the impairment assessment process. Acquisitions may also result in dilutive issuances of equity securities or the incurrence of debt, which could adversely affect our operating results. In addition, if an acquired business fails to meet our expectations, our business, results of operations and financial condition may be adversely affected.

 

Risks Related to Development and Regulatory Approval of Our Product Candidates

 

Clinical trials for our product candidates are expensive, time-consuming, uncertain, and susceptible to change, delay or termination. The results of clinical trials are open to differing interpretations.

 

Clinical trials are expensive, time consuming and difficult to design and implement. Regulatory agencies may analyze or interpret the results differently than us. Even if the results of our clinical trials are favorable, the clinical trials for a number of our product candidates are expected to continue for several years and may take significantly longer to complete. In addition, we, the FDA, or other regulatory authorities, including state and local authorities, or an Institutional Review Board, or IRB, with respect to a trial at its institution, may suspend, delay or terminate our clinical trials at any time, require us to conduct additional clinical trials, require a particular clinical trial to continue for a longer duration than originally planned, require a change to our development plans such that we conduct clinical trials for a product candidate in a different order, e.g., in a step-wise fashion rather than running two trials of the same product candidate in parallel, or the DEA could suspend or terminate the registrations and quota allotments we require in order to procure and handle controlled substances, for various reasons, including:

 

  lack of effectiveness of any product candidate during clinical trials;
     
  discovery of serious or unexpected toxicities or side effects experienced by trial participants or other safety issues, such as drug interactions, including those which cause confounding changes to the levels of other concomitant medications;
     
  slower than expected rates of subject recruitment and enrollment rates in clinical trials;
     
  difficulty in retaining subjects who have initiated a clinical trial but may withdraw at any time due to adverse side effects from the therapy, insufficient efficacy, fatigue with the clinical trial process or for any other reason;
     
  the evolving effects of the COVID-19 pandemic;
     
  delays or inability in manufacturing or obtaining sufficient quantities of materials for use in clinical trials due to regulatory and manufacturing constraints;

 

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  inadequacy of or changes in our manufacturing process or product formulation;
     
  delays in obtaining regulatory authorization to commence a trial, including “clinical holds” or delays requiring suspension or termination of a trial by a regulatory agency, such as the FDA, before or after a trial is commenced;
     
  changes in applicable regulatory policies and regulation, including changes to requirements imposed on the extent, nature, or timing of studies;
     
  delays or failure in reaching agreement on acceptable terms in clinical trial contracts or protocols with prospective clinical trial sites;
     
  uncertainty regarding proper dosing;
     
  delay or failure to supply product for use in clinical trials which conforms to regulatory specification;
     
  unfavorable results from ongoing pre-clinical studies and clinical trials;
     
  failure of our contract research organizations, or CROs, or other third-party contractors to comply with all contractual requirements or to perform their services in a timely or acceptable manner;

 

  failure by us, our employees, our CROs or their employees to comply with all applicable FDA or other regulatory requirements relating to the conduct of clinical trials or the handling, storage, security, and recordkeeping;
     
  scheduling conflicts with participating clinicians and clinical institutions;

 

  failure to design appropriate clinical trial protocols;
     
  insufficient data to support regulatory approval;
     
  inability or unwillingness of medical investigators to follow our clinical protocols; or
     
  difficulty in maintaining contact with patients during or after treatment, which may result in incomplete data.

 

Any of the foregoing could have a material adverse effect on our business, results of operations and financial condition.

 

Any failure by us to comply with existing regulations could harm our reputation and operating results.

 

We are subject to extensive regulation by U.S. federal and state governments in each of the markets where we have product candidates progressing through the approval process.

 

We must also adhere to all regulatory requirements including FDA’s Good Laboratory Practice, Good Clinical Practice, and current Good Manufacturing Practices requirements (“cGMP”) pharmacovigilance requirements, advertising, and promotion restrictions, reporting and recordkeeping requirements. If we or our suppliers fail to comply with applicable regulations, including FDA pre-or post-approval cGMP requirements, then FDA could sanction us. Even if a drug is FDA-approved, regulatory authorities may impose significant restrictions on a product’s indicated uses or marketing or impose ongoing requirements for potentially costly post-marketing trials. TELOMIR-1, and any of our product candidates that may be approved in the U.S. in the future, will be subject to ongoing regulatory requirements for manufacturing, labeling, packaging, storage, distribution, import, export, advertising, promotion, sampling, recordkeeping and submission of safety and other post-market information, including both federal and state requirements in the U.S. In addition, manufacturers and manufacturers’ facilities are required to comply with extensive FDA requirements, including ensuring that quality control and manufacturing procedures conform to GMP. As such, we, and our contract manufacturers (in the event contract manufacturers are appointed in the future) are subject to continual review and periodic inspections to assess compliance with GMP. Accordingly, we and others with whom we work must continue to spend time, money, and effort in all areas of regulatory compliance, including manufacturing, production, quality control and quality assurance. We will also be required to report certain adverse reactions and production problems, if any, to the FDA, and to comply with requirements concerning advertising and promotion for our products. Promotional communications with respect to prescription drugs are subject to a variety of legal and regulatory restrictions and must be consistent with the information in the product’s approved label.

 

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If a regulatory agency discovers previously unknown problems with a product, such as adverse events of unanticipated severity or frequency, or problems with the facility where the product is manufactured, or disagrees with the promotion, marketing or labeling of the product, it may impose restrictions on that product or us, including requiring withdrawal of the product from the market. If we fail to comply with applicable regulatory requirements, a regulatory agency or enforcement authority may:

 

  issue untitled or warning letters;
     
  seek to enjoin our activities;
     
  impose civil or criminal penalties;
     
  suspend regulatory approval;
     
  suspend any of our ongoing clinical trials;
     
  refuse to approve pending applications or supplements to approved applications submitted by us;
     
  impose restrictions on our operations, including by requiring us to enter into a Corporate Integrity Agreement or closing our contract manufacturers’ facilities, if any; or
     
  seize or detain products or require a product recall.

 

In addition, any government investigation of alleged violations of law could require us to expend significant time and resources in response and could generate negative publicity. Any failure to comply with ongoing regulatory requirements may significantly and adversely affect our ability to commercialize and generate revenue from our product candidates. If regulatory sanctions are applied or if regulatory approval is withdrawn, the value of our business and our operating results may be adversely affected.

 

Any action against us for violation of these laws, even if we successfully defend against it, could cause us to incur significant legal expenses, divert our management’s attention from the operation of our business and damage our reputation. We expend significant resources on compliance efforts and such expenses are unpredictable and might adversely affect our results. Changing laws, regulations and standards might also create uncertainty, higher expenses and increase insurance costs. As a result, we intend to invest all reasonably necessary resources to comply with evolving standards, and this investment might result in increased management and administrative expenses and a diversion of management time and attention from revenue-generating activities to compliance activities.

 

We are subject to federal and state healthcare laws and regulations and implementation of or changes to such healthcare laws and regulations could adversely affect our business and results of operations.

 

In the United States, there have been a number of legislative and regulatory proposals to change the healthcare system in ways that could impact our ability to sell our product candidates. If we are found to be in violation of any of these laws or any other federal or state regulations, we may be subject to administrative, civil and/or criminal penalties, damages, fines, individual imprisonment, exclusion from federal health care programs and the restructuring of our operations. Any of these could have a material adverse effect on our business and financial results. Since many of these laws have not been fully interpreted by the courts, there is an increased risk that we may be found in violation of one or more of their provisions. Any action against us for violation of these laws, even if we ultimately are successful in our defense, will cause us to incur significant legal expenses and divert our management’s attention away from the operation of our business.

 

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We expect that the ACA, as well as other healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria and in additional downward pressure on the price that we may receive for any approved product. There have been judicial challenges to certain aspects of the ACA and numerous legislative attempts to repeal and/or replace the ACA in whole or in part, and we expect there will be additional challenges and amendments to the ACA in the future. At this time, the full effect that the ACA will have on our business in the future remains unclear. An expansion in the government’s role in the U.S. healthcare industry may cause general downward pressure on the prices of prescription drug products, lower reimbursements, or any other product for which we obtain regulatory approval, reduce product utilization, and adversely affect our business and results of operations. Any reduction in reimbursement from Medicare or other government programs may result in a similar reduction in payments from private payers. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability, or commercialize products for which we may receive regulatory approval.

 

The regulatory approval processes with the FDA are lengthy and inherently unpredictable.

 

We are not permitted to market our drug candidates as medicines in the United States or other countries until we receive approval of a New Drug Application (“NDA”) from the FDA or in any foreign countries until we receive the approval from the regulatory authorities of such countries. Prior to submitting an NDA to the FDA for approval of our drug candidates we will need to have completed our pre-clinical studies and clinical trials and demonstrate that our products meet all applicable standards of identity, strength, quality, and purity throughout their expiration date. Successfully completing any clinical program and obtaining approval of an NDA is a complex, lengthy, expensive, and uncertain process, and the FDA (or other country medicines regulatory body) may delay, limit, or deny approval of product candidates for many reasons, including, among others, because:

 

  an inability to demonstrate that our product candidates are safe and effective in treating patients to the satisfaction of the FDA;
     
  results of clinical trials that may not meet the level of statistical or clinical significance required by the FDA;
     
  disagreements with the FDA with respect to the number, design, size, conduct or implementation of clinical trials;
     
  requirements by the FDA to conduct additional clinical trials;

 

  disapproval by the FDA of certain formulations, labeling or specifications of product candidates;
     
  findings by the FDA that the data from pre-clinical studies and clinical trials are insufficient;

 

  findings by the FDA that our API or finished products do not meet all applicable standards of identity, strength, quality, and purity;
     
  the FDA may disagree with the interpretation of data from pre-clinical studies and clinical trials; and
     
  the FDA may change their approval policies or adopt new regulations.

 

Any of these factors, many of which are beyond our control, could increase development time and / or costs or jeopardize our ability to obtain regulatory approval for our drug candidates.

 

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There is a high rate of failure for drug candidates proceeding through clinical trials.

 

Generally, there is a high rate of failure for drug candidates proceeding through clinical trials. We may suffer significant setbacks in our clinical trials similar to the experience of a number of other companies in the pharmaceutical and biotechnology industries, even after receiving promising results in earlier trials. Further, even if we view the results of a clinical trial to be positive, FDA may disagree with our interpretation of the data. In the event that we obtain negative results from clinical trials for product candidates or other problems related to potential chemistry, manufacturing and control issues or other hurdles occur and our product candidates are not approved, we may not be able to generate sufficient revenue or obtain financing to continue our operations, our ability to execute on our current business plan may be materially impaired, our reputation in the industry and in the investment community might be significantly damaged and the price of our common stock could decrease significantly. In addition, our inability to properly design, commence and complete clinical trials may negatively impact the timing and results of our clinical trials and ability to seek approvals for our drug candidates.

 

If we are found in violation of federal or state “fraud and abuse” laws, we may be required to pay a penalty and/or be suspended from participation in federal or state health care programs, which may adversely affect our business, financial condition, and results of operations.

 

In the United States, we are subject to various federal and state health care “fraud and abuse” laws, including anti-kickback laws, false claims laws and other laws intended to reduce fraud and abuse in federal and state health care programs, which could affect us particularly upon successful commercialization of our products in the U.S. The Medicare and Medicaid Patient Protection Act of 1987, or federal Anti-Kickback Statute, makes it illegal for any person, including a prescription drug manufacturer (or a party acting on its behalf), to knowingly and willfully solicit, receive, offer or pay any remuneration that is intended to induce the referral of business, including the purchase, order or prescription of a particular drug for which payment may be made under a federal health care program, such as Medicare or Medicaid. Under federal law, some arrangements, known as safe harbors, are deemed not to violate the federal Anti-Kickback Statute. Although we seek to structure our business arrangements in compliance with all applicable requirements, it is often difficult to determine precisely how the law will be applied in specific circumstances. Accordingly, it is possible that our practices may be challenged under the federal Anti-Kickback Statute and Federal False Claims Act. Violations of fraud and abuse laws may be punishable by criminal and/or civil sanctions, including fines and/or exclusion or suspension from federal and state health care programs such as Medicare and Medicaid and debarment from contracting with the U.S. government. In addition, private individuals have the ability to bring actions on behalf of the government under the federal False Claims Act as well as under the false claims laws of several states.

 

Many states have adopted laws similar to the federal anti-kickback statute, some of which apply to the referral of patients for health care services reimbursed by any source, not just governmental payers. There are ambiguities as to what is required to comply with these state requirements and if we fail to comply with an applicable state law requirement, we could be subject to penalties.

 

Neither the government nor the courts have provided definitive guidance on the application of fraud and abuse laws to our business. Law enforcement authorities are increasingly focused on enforcing these laws, and it is possible that some of our practices may be challenged under these laws. While we believe we have structured our business arrangements to comply with these laws, it is possible that the government could allege violations of, or convict us of violating, these laws. If we are found in violation of one of these laws, we could be required to pay a penalty and could be suspended or excluded from participation in federal or state health care programs, and our business, results of operations and financial condition may be adversely affected.

 

Serious adverse events or other safety risks could require us to abandon development and preclude, delay or limit approval of our product candidates, limit the scope of any approved label or market acceptance, or cause the recall or loss of marketing approval of products that are already marketed.

 

If any of our product candidates prior to or after any approval for commercial sale, cause serious or unexpected side effects, or are associated with other safety risks such as misuse, abuse or diversion, a number of potentially significant negative consequences could result, including:

 

  regulatory authorities may interrupt, delay or halt clinical trials;

 

  regulatory authorities may deny regulatory approval of our product candidates;
     
  regulatory authorities may require certain labeling statements, such as warnings or contraindications or limitations on the indications for use, and/or impose restrictions on distribution in the form of a Risk Evaluation and Mitigation Strategy (“REMS”) in connection with approval or post-approval;
     
  regulatory authorities may withdraw their approval, require more onerous labeling statements, impose a more restrictive REMS, or require us to recall any product that is approved;
     
  we may be required to change the way the product is administered or conduct additional clinical trials;
     
  our relationships with our collaboration partners may suffer;
     
  we could be sued and held liable for harm caused to patients; or
     
  our reputation may suffer. The reputational risk is heightened with respect to those of our product candidates that are being developed for pediatric indications.

 

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We may voluntarily suspend or terminate our clinical trials if at any time we believe that they present an unacceptable risk to participants or if preliminary data demonstrate that our product candidates are unlikely to receive regulatory approval or unlikely to be successfully commercialized. Following receipt of approval for commercial sale of a product we may voluntarily withdraw or recall that product from the market if at any time we believe that its use, or a person’s exposure to it, may cause adverse health consequences or death. To date we have not withdrawn, recalled, or taken any other action, voluntary or mandatory, to remove an approved product from the market. In addition, regulatory agencies, IRBs, or data safety monitoring boards may at any time recommend the temporary or permanent discontinuation of our clinical trials or request that we cease using investigators in the clinical trials if they believe that the clinical trials are not being conducted in accordance with applicable regulatory requirements, or that they present an unacceptable safety risk to participants. Although we have never been asked by a regulatory agency, IRB, or data safety monitoring board to discontinue a clinical trial temporarily or permanently, if we elect or are forced to suspend or terminate a clinical trial of any of our product candidates, the commercial prospects for that product will be harmed and our ability to generate product revenue from that product may be delayed or eliminated. Furthermore, any of these events may result in labeling statements such as warnings or contraindications. In addition, such events or labeling could prevent us or our partners from achieving or maintaining market acceptance of the affected product and could substantially increase the costs of commercializing our product candidates and impair our ability to generate revenue from the commercialization of these products either by us or by our collaboration partners.

 

Risks Related to Our Reliance Upon Third Parties

 

Our existing collaboration arrangements and any that we may enter into in the future may not be successful, which could adversely affect our ability to develop and commercialize our product candidates.

 

We may seek additional collaboration arrangements with pharmaceutical or biotechnology companies for the development or commercialization of our product candidates. We may, with respect to our product candidates, enter into new arrangements on a selective basis depending on the merits of retaining commercialization rights for ourselves as compared to entering into selective collaboration arrangements with leading pharmaceutical or biotechnology companies for each product candidate, both in the U.S. and internationally. To the extent that we decide to enter into collaboration agreements, we will face significant competition in seeking appropriate collaborators and the terms of any collaboration or other arrangements that we may establish may not be favorable to us.

 

Any existing or future collaboration that we enter may not be successful. The success of our collaboration arrangements will depend heavily on the efforts and activities of our collaborators. Collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaborations. Disagreements between parties to a collaboration arrangement regarding development, intellectual property, regulatory or commercialization matters can lead to delays in the development process or commercialization of the applicable product candidate and, in some cases, termination of the collaboration arrangement. These disagreements can be difficult to resolve if neither of the parties has final decision-making authority. Any such termination or expiration could harm our business reputation and may adversely affect us financially.

 

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We depend on a limited number of suppliers for materials and components required to manufacture our product candidates. The loss of these suppliers, or their failure to supply us on a timely basis, could cause delays in our current and future capacity and adversely affect our business.

 

We depend on a limited number of suppliers for the materials and components required to manufacture our product candidates. As a result, we may not be able to obtain sufficient quantities of critical materials and components in the future. A delay or interruption by our suppliers may also harm our business, results of operations and financial condition. In addition, the lead time needed to establish a relationship with a new supplier can be lengthy, and we may experience delays in meeting demand in the event we must switch to a new supplier. The time and effort to qualify for and, in some cases, obtain regulatory approval for a new supplier could result in additional costs, diversion of resources or reduced manufacturing yields, any of which would negatively impact our operating results. Our dependence on single-source suppliers exposes us to numerous risks, including the following: our suppliers may cease or reduce production or deliveries, raise prices or renegotiate terms; our suppliers may become insolvent or cease trading; we may be unable to locate a suitable replacement supplier on acceptable terms or on a timely basis, or at all; and delays caused by supply issues may harm our reputation, frustrate our customers and cause them to turn to our competitors for future needs.

 

We maintain our cash at financial institutions, at times in balances that exceed federally insured limits. The failure of financial institutions could adversely affect our ability to pay operational expenses or make other payments.

 

Our cash held in non-interest-bearing and interest-bearing accounts can at times exceed the Federal Deposit Insurance Corporation (“FDIC”) insurance limits. If such banking institutions were to fail, we could lose all or a portion of those amounts held in excess of such insurance limitations. In addition, even if account holders are ultimately made whole with respect to a future bank failure, account holders’ access to their accounts and assets held in their accounts may be substantially delayed. Any material loss that we may experience in the future or inability for a material time period to access our cash and cash equivalents could have an adverse effect on our ability to pay our operational expenses or make other payments, which could adversely affect our business.

 

Risks Related to Our Intellectual Property

 

We depend on rights to TELOMIR-1 that are or will be licensed to us. We do not own the intellectual property rights to TELOMIR-1 and any loss of our rights to it could prevent us from selling our product.

 

Within our present and future pipeline of treatments, TELOMIR-1 is in-licensed from another company. We do not currently own any intellectual property rights, including the patent application that underlies this license. Our rights to use TELOMIR-1 is subject to the negotiation of, continuation of and compliance with the terms of this license. Thus, the non-provisional patent application is not written by us or our attorneys, and we did not have control over the drafting and prosecution. The patent owner and our licensor might not have given the same attention to the drafting and prosecution of these patents and applications as we would have if we had been the owner of the patent application and had control over the drafting. We cannot be certain that drafting of the licensed patent application, or patent prosecution, by the licensor have been or will be conducted in compliance with applicable laws and regulations or will result in valid and enforceable patents and other intellectual property rights.

 

Significant additional research and development activity, pre-clinical testing, and/or clinical testing TELOMIR-1 is required before we will have a chance to achieve a viable product for licensing or commercialization. Our business currently depends entirely on the successful development, regulatory approval, and licensing or commercialization of our product candidate, which may never occur.

 

Enforcement of our licensed patent application or defense of any claims asserting invalidity of these patents is often subject to the control or cooperation of our licensor. Legal action could be initiated against the owners of the intellectual property that we license and an adverse outcome in such legal action could harm our business because it might prevent such companies or institutions from continuing to license intellectual property that we may need to operate our business. In addition, such licensor may resolve such litigation in a way that benefits it but adversely affects our ability to have freedom to operate to develop and commercialize TELOMIR-1.

 

We may not be able to adequately protect our product candidates or our proprietary technology in the marketplace.

 

Our success will depend, in part, on our ability to obtain patents, protect our trade secrets and operate without infringing on the proprietary rights of others. We may rely upon a combination of patents, trade secret protection (i.e., know-how), trademarks, licenses, and confidentiality agreements to protect the intellectual property of our product candidates. The strengths of patents in the pharmaceutical field involve complex legal and scientific questions and can be uncertain. Where appropriate, we seek patent protection for certain aspects of our products and technology. However, patent protection for naturally occurring compounds is exceedingly difficult to obtain, defend and enforce. Filing, prosecuting and defending patents throughout the world would be prohibitively expensive, so our policy is to look to patent technologies with commercial potential in jurisdictions with significant commercial opportunities. However, patent protection may not be available for some of the products or technology we are developing. If we must spend significant time and money protecting, defending, or enforcing our patents, designing around patents held by others or licensing, potentially for large fees, patents or other proprietary rights held by others, our business, results of operations and financial condition may be harmed. We may not develop additional proprietary products that are patentable.

 

The patent positions of pharmaceutical products are complex and uncertain. Although we have sought and expect to continue to seek patent protection for our product candidates, their methods of use, and methods of manufacture, any, or all of them may not be subject to effective patent protection. If any of our products are approved and marketed for an indication for which we do not have an issued patent, our ability to use our patents to prevent a competitor from commercializing a non-branded version of our commercial products for that non-patented indication could be significantly impaired or even eliminated.

 

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Publication of information related to our product candidates by us, or others may prevent us from obtaining or enforcing patents relating to these products and product candidates. Furthermore, others may independently develop similar products, may duplicate our products, or may design around our patent rights. In addition, any of our issued patents may be opposed and/or declared invalid or unenforceable. If we fail to adequately protect our intellectual property, we may face competition from companies who attempt to create a generic product to compete with our product candidates. We may also face competition from companies who develop a substantially similar product to one of our product candidates that is not covered by any of our patents.

 

Many companies have encountered significant problems in protecting, defending and enforcing intellectual property rights in foreign jurisdictions. The legal systems of certain countries, particularly certain developing countries, do not favor the enforcement of patents and other intellectual property rights, particularly those relating to pharmaceuticals, which could make it difficult for us to stop the infringement of our patents or marketing of competing products in violation of our proprietary rights generally. Proceedings to enforce our patent rights in foreign jurisdictions could result in substantial cost and divert our efforts and attention from other aspects of our business.

 

Currently, we do not own the rights to the intellectual property and technology that will be used to commercially develop our initial product candidate, TELOMIR-1. MIRALOGX, which is a separate intellectual property development company owned by a trust established by the Company’s founder, holds the patent rights to TELOMIR-1, which are currently comprised of a pending non-provisional patent application. We have an exclusive, license from MIRALOGX to develop and commercialize TELOMIR-1 in the U.S. for human and non-human applications. The term of the license will continue through the date of the expiration of the last-to-expire licensed patent or, if later, the date of the expiration of the last strategic partnership/sublicensing agreement covering the licensed products. The licensed patent rights are expected to extend through 2043. We expect additional patent terms may be awarded, including additional patent terms based on the time for regulatory review of drug products. There are no up-front, execution, or milestone payments required under the license agreement. Further, no payments have been made to date under the agreement. We are also required to pay an 8% royalty on net sales or revenue in exchange for an exclusive, worldwide license to patent rights, and we may bring suit in our own name to enforce our patent rights under the license agreement. In the event we are unable to enforce our rights under the agreement or are unable to detect unauthorized use of our intellectual property, we may lose the benefit of the licensed rights used to commercially develop TELOMIR-1. MIRALOGX will control the prosecution of the patent applications for TELOMIR-1.

 

If third parties claim that the Company’s intellectual property, products, processes, or anything else used by us infringes upon their intellectual property, our operating profits could be adversely affected.

 

There is a substantial amount of litigation, both within and outside the U.S., involving patent and other intellectual property rights in the pharmaceutical industry. We may, from time to time, be notified of claims that we are infringing upon patents, trademarks, copyrights, or other intellectual property rights owned by third parties, and we cannot provide assurances that other companies will not, in the future, pursue such infringement claims against us, our commercial partners or any third-party proprietary technologies we have licensed. If we were found to infringe upon a patent or other intellectual property right, or if we failed to obtain or renew a license under a patent or other intellectual property right from a third party, or if a third party that we were licensing technologies from was found to infringe upon a patent or other intellectual property rights of another third party, we may be required to pay damages, including damages of up to three times the damages found or assessed, if the infringement is found to be willful, suspend the manufacture of certain products or reengineer or rebrand our products, if feasible, or we may be unable to enter certain new product markets. Any such claims could also be expensive and time consuming to defend and divert management’s attention and resources. Our competitive position could suffer as a result. In addition, if we have declined or failed to enter into a valid non-disclosure or assignment agreement for any reason, we may not own the invention or our intellectual property, and our products may not be adequately protected. Thus, we cannot guarantee that our product candidates, or our commercialization thereof, does not and will not infringe any third party’s intellectual property.

 

We have been granted a license to the right to develop TELOMIR-1 in the United States in human and non-human application, but we have not been granted a license to the rights to patents covering TELOMIR-1 in foreign jurisdictions.

 

We have been granted a license to the right to develop TELOMIR-1 in the United States but not in countries outside the United States, as MIRALOGX has retained all rights outside the United States and may license such rights to other parties. Accordingly, MIRALOGX potentially could develop a competing product for such jurisdictions outside of the United States.

 

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Risks Relating to our Common Stock

 

Because of the speculative nature of investment risk, you may lose your entire investment.

 

An investment in our securities carries a high degree of risk and should be considered as a speculative investment. We have a limited operating history, no revenues, have not paid dividends, and are unlikely to pay dividends in the immediate or near future. The likelihood of our success must be considered in light of the problems, expenses, difficulties, complications and delays frequently encountered in connection with the establishment of any business. An investment in our securities may result in the loss of an investor’s entire investment. Only potential investors who are experienced in high-risk investments and who can afford to lose their entire investment should consider an investment in our securities.

 

The requirements of being a public company may strain our resources, divert management’s attention and affect our ability to attract and retain executive management and qualified board members.

 

As a reporting issuer, we will be subject to the reporting requirements of applicable securities legislation of the jurisdiction in which it is a reporting issuer, the listing requirements of Nasdaq and other applicable securities rules and regulations. Compliance with these rules and regulations will increase our legal and financial compliance costs, make some activities more difficult, time-consuming or costly and increase demand on its systems and resources. Applicable securities laws will require us to, among other things, file certain annual and quarterly reports with respect to its business and results of operations. In addition, applicable securities laws require us to, among other things, maintain effective disclosure controls and procedures and internal control over financial reporting.

 

In order to maintain and, if required, improve its disclosure controls and procedures and internal control over financial reporting to meet this standard, significant resources and management oversight are required. Specifically, due to the increasing complexity of its transactions, it is anticipated that we will improve our disclosure controls and procedures and internal control over financial reporting primarily through the continued development and implementation of formal policies, improved processes and documentation procedures, as well as the continued sourcing of additional finance resources. As a result, management’s attention may be diverted from other business concerns, which could harm our business and results of operations. To comply with these requirements, we may need to hire more employees in the future or engage outside consultants, which will increase its costs and expenses.

 

In addition, changing laws, regulations and standards relating to corporate governance and public disclosure are creating uncertainty for public companies, increasing legal and financial compliance costs and making some activities more time consuming. These laws, regulations and standards are subject to varying interpretations, in many cases due to their lack of specificity, and, as a result, their application in practice may evolve over time as new guidance is provided by regulatory and governing bodies. This could result in continuing uncertainty regarding compliance matters and higher costs necessitated by ongoing revisions to disclosure and governance practices. We intend to continue to invest resources to comply with evolving laws, regulations and standards, and this investment may result in increased general and administrative expenses and a diversion of management’s time and attention from revenue-generating activities to compliance activities. If our efforts to comply with new laws, regulations and standards differ from the activities intended by regulatory or governing bodies due to ambiguities related to their application and practice, regulatory authorities may initiate legal proceedings against us, which could adversely affect our business and financial results.

 

As a public company subject to these rules and regulations, we may find it more expensive for it to obtain director and officer liability insurance, and it may be required to accept reduced coverage or incur substantially higher costs to obtain coverage. These factors could also make it more difficult for us to attract and retain qualified members of our board of directors, particularly to serve on its Audit Committee and Compensation Committee, and qualified executive officers.

 

As a result of disclosure of information in filings required of a public company, our business and financial condition will become more visible, which may result in threatened or actual litigation, including by competitors and other third parties. If such claims are successful, our business and results of operations could be harmed, and even if the claims do not result in litigation or are resolved in its favor, these claims, and the time and resources necessary to resolve them, could divert the resources of our management and harm its business and results of operations.

 

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We are an “emerging growth company” and any decision on our part to comply only with certain reduced reporting and disclosure requirements applicable to emerging growth companies could make shares of our common stock less attractive to investors.

 

We are an “emerging growth company,” as defined in Section 2(a) of the Securities Act. For as long as we continue to be an emerging growth company, we may choose to take advantage of exemptions from various reporting requirements applicable to other public companies that are not emerging growth companies, including, but not limited to, not being required to have our independent registered public accounting firm audit our internal control over financial reporting under Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in our periodic reports and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval of any golden parachute payments not previously approved. We could be an emerging growth company until the fifth anniversary of the fiscal year end date following the completion of this offering, however, our status would change more quickly if we have more than US$1.235 billion in annual revenue, if the market value of our shares of common stock held by non-affiliates equals or exceeds US$700 million as of June 30 of any year, or we issue more than US$1.0 billion of non-convertible debt over a three-year period before the end of that period.

 

Investors could find our shares less attractive if we choose to rely on these exemptions. If some investors find shares less attractive as a result of any choice to reduce future disclosure, there may be a less active trading market for our shares and our share price may be more volatile.

 

For as long as we are an “emerging growth company”, our independent registered public accounting firm will not be required to attest to the effectiveness of our internal controls over financial reporting pursuant to Section 404. We could be an “emerging growth company” until the fifth anniversary of the fiscal year end date following the completion of this offering. An independent assessment of the effectiveness of our internal controls could detect problems that our management’s assessment might not. Undetected material weaknesses in our internal controls could lead to financial statement restatements and require us to incur the expense of remediation.

 

If we identify material weaknesses in our internal control over financial reporting, or if we are unable to comply with the requirements of Section 404 in a timely manner or assert that our internal control over financial reporting is effective, or if our independent registered public accounting firm is unable to express an opinion as to the effectiveness of our internal control over financial reporting when required, investors may lose confidence in the accuracy and completeness of our financial reports and the market price of our securities could be negatively affected, and we could become subject to investigations by the stock exchange on which our securities are listed, the SEC, or other regulatory authorities, which could require additional financial and management resources.

 

We are a “smaller reporting company” and, even if we no longer qualify as an emerging growth company, we may still be subject to reduced reporting requirements.

 

Additionally, we are a “smaller reporting company” as defined in Item 10(f)(1) of Regulation S-K. Smaller reporting companies may take advantage of certain reduced disclosure obligations, including, among other things, providing only two years of audited financial statements. We will remain a smaller reporting company until the last day of any fiscal year for so long as either: (i) the market value of our shares of common stock held by non-affiliates does not equal or exceed $250 million as of the prior June 30th; or (ii) our annual revenues did not equal or exceed $100 million during such completed fiscal year. To the extent we take advantage of such reduced disclosure obligations, it may also make the comparison of our financial statements with other public companies difficult or impossible.

 

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Our management will have broad discretion over the use of the proceeds we receive in this offering and might not apply the proceeds in ways that increase the value of your investment.

 

Our management will have broad discretion over the use of our net proceeds from this offering and you will be relying on the judgment of our management regarding the application of these proceeds. Our management might not use our net proceeds in ways that ultimately increase the value of your investment. We expect to use the net proceeds from this offering to advance the clinical development of our programs, to fund our research and development activities and to fund working capital and for general corporate purposes. Our management might not be able to yield a significant return, if any, on any investment or use of these net proceeds. You will not have the opportunity to influence the decision on how to use the net proceeds from this offering.

 

If we fail to maintain compliance with Nasdaq Listing Rules, our shares may be delisted from Nasdaq, which would result in a limited trading market for our shares and make obtaining future debt or equity financing more difficult for the Company.

 

We have applied to have shares of our common stock sold in this offering listed on the Nasdaq under the symbol “TELO”. However, there is no assurance that we will be able to continue to maintain our compliance with the Nasdaq continued listing requirements. If we fail to do so, our securities may be de-listed and cease trading on Nasdaq. As a result, selling our securities could be more difficult because smaller quantities of shares or warrants would likely be bought and sold, transactions could be delayed, and security analysts’ coverage of us may be reduced. In addition, in the event our securities are delisted, broker-dealers would face certain regulatory requirements which may discourage them from effecting transactions in the securities and further limit the liquidity of the securities. These factors could result in lower prices and larger spreads in the bid and ask prices for the securities. Such delisting from Nasdaq and continued or further declines in the share price of the securities could also greatly impair our ability to raise additional necessary capital through equity or debt financing and could significantly increase the ownership dilution to shareholders caused by our issuing equity in financing or other transactions.

 

If our shares were to be delisted from Nasdaq, they may become subject to the SEC’s “penny stock” rules.

 

Delisting from Nasdaq may cause the securities of the Company to become subject to the SEC’s “penny stock” rules. The SEC generally defines a penny stock as an equity security that has a market price of less than $5.00 per share or an exercise price of less than $5.00 per share, and that is not listed on a national securities exchange, such as Nasdaq subject to certain exemptions. Therefore, if shares of our common stock were to be delisted from Nasdaq, the securities of the Company could become subject to the SEC’s “penny stock” rules. These rules require, among other things, that any broker engaging in a purchase or sale of our securities provide its customers with: (i) a risk disclosure document, (ii) disclosure of market quotations, if any, (iii) disclosure of the compensation of the broker and its salespersons in the transaction, and (iv) monthly account statements showing the market values of our securities held in the customer’s accounts. A broker would be required to provide the bid and offer quotations and compensation information before effecting the transaction. This information must be contained in the customer’s confirmation. Generally, brokers are less willing to effect transactions in penny stocks due to these additional delivery requirements. These requirements may make it more difficult for shareholders to purchase or sell the shares of our common stock. Since the broker, not us, prepares this information, we would not be able to assure that such information is accurate, complete or current.

 

You will experience immediate and substantial dilution as a result of this offering and may experience additional dilution in the future.

 

If you purchase shares in this offering, the value of your shares based on our actual book value will immediately be less than the price you paid. This reduction in the value of your equity is known as dilution. This dilution occurs in large part because our existing stockholders paid less than the assumed public offering price when they acquired their shares of common stock. Based upon the issuance and sale of 1,000,000 shares of common stock by us in this offering at an assumed public offering price of $7.00 per share, you will incur immediate dilution of in the net tangible book value per share. If the underwriters exercise their over-allotment option, or if outstanding warrants to purchase shares of our common stock are exercised, investors will experience additional dilution. For more information, see “Dilution”.

 

There is no existing market for our securities and we do not know if one will develop to provide you with adequate liquidity.

 

Prior to this offering, there has not been a public market for our securities. We cannot assure you that an active trading market for shares of our common stock will develop following this offering, or if it does develop, it may not be maintained. You may not be able to sell your shares of our common stock quickly or at the market price if trading in our securities is not active. The initial public offering price for the shares of common stock offered hereby will be determined by negotiations between us and the underwriter and may not be indicative of prices that will prevail in the trading market.

 

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Some provisions of Florida law and our amended and restated articles of incorporation and amended and restated bylaws that will be in effect immediately prior to the completion of this offering may have anti-takeover effects that could discourage an acquisition of us by others, even if an acquisition would be beneficial to our shareholders, and may prevent attempts by our shareholders to replace or remove our current management.

 

Upon the closing of this offering, our status as a Florida corporation and the anti-takeover provisions of the Florida Business Corporation Act, which we sometimes refer to as the FBCA, may discourage, delay or prevent a change in control even if a change in control would be beneficial to our shareholders.

 

The control share acquisition statute, Section 607.0902 of the FBCA, generally provides that in the event a person acquires voting shares of the company in excess of 20% of the voting power of all of our issued and outstanding shares, such acquired shares will not have any voting rights unless such rights are restored by the holders of a majority of the votes of each class or series entitled to vote separately, excluding shares held by the person acquiring the control shares or any of our officers or employees who are also directors of the company. Certain acquisitions of shares are exempt from these rules, such as shares acquired pursuant to the laws of intestate succession or pursuant to a gift or testamentary transfer, pursuant to a merger or share exchange effected in compliance with the FBCA if we are a party to the agreement, or pursuant to an acquisition of our shares if the acquisition has been approved by our board of directors before the acquisition. The control share acquisition statute generally applies to any “issuing public corporation,” which means a Florida corporation which has:

 

One hundred or more shareholders;

 

Its principal place of business, its principal office, or substantial assets within Florida; and

 

Either (i) more than 10% of its shareholders are resident in Florida; (ii) more than 10% of its shares are owned by residents of Florida; or (iii) one thousand shareholders are resident in Florida.

 

The affiliated transaction (or so-called “business combination”) statute, Section 607.0901 of the FBCA, provides that we may not engage in certain mergers, consolidations, sales of assets, issuances of stock, reclassifications, recapitalizations, and other affiliated transactions with any “interested shareholder” for a period of three years following the time that such shareholder became an interested shareholder, unless:

 

Prior to the time that such shareholder became an interested shareholder, our board of directors approved either the affiliated transaction or the transaction which resulted in the shareholder becoming an interested shareholder; or

 

Upon consummation of the transaction that resulted in the shareholder becoming an interested shareholder, the interested shareholder owned at least 85% of our voting shares outstanding at the time the transaction commenced; or

 

At or subsequent to the time that such shareholder became an interested shareholder, the affiliated transaction is approved by our board of directors and authorized at an annual or special meeting of shareholders, and not by written consent, by the affirmative vote of at least two-thirds of the outstanding voting shares which are not owned by the interested shareholder.

 

An “interested shareholder” is generally defined as any person who is the beneficial owner of more than 15% of our outstanding voting shares.

 

The voting requirements set forth above do not apply to a particular affiliated transaction if one or more conditions are met, including, but not limited to, the following: if the affiliated transaction has been approved by a majority of our disinterested directors; if we have not had more than 300 shareholders of record at any time during the three years preceding the date the affiliated transaction is announced; if the interested shareholder has been the beneficial owner of at least 80% of our outstanding voting shares for at least three years preceding the date the affiliated transaction is announced; or if the consideration to be paid to the holders of each class or series of voting shares in the affiliated transaction meets certain requirements of the statute with respect to form and amount, among other things.

 

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Both the control share acquisition statute and the affiliated transactions statute may have the effect of discouraging or preventing certain change of control or takeover transactions involving us.

 

In addition, our amended and restated articles of incorporation and amended and restated bylaws that will be in effect immediately prior to the completion of this offering contain provisions that may make it more difficult for a third party to acquire us or increase the cost of acquiring us, even if doing so would benefit our shareholders, including transactions in which shareholders might otherwise receive a premium for their shares. These provisions include:

 

nothing in our amended and restated articles of incorporation precludes future issuances without shareholder approval of the authorized but unissued shares of our common stock;

 

advance notice procedures apply for shareholders to nominate candidates for election as directors or to bring matters before an annual meeting of shareholders;

 

a special meeting of shareholders can only be called by our chairman of the board of directors, our chief executive officer, our president (in the absence of a chief executive officer), a majority of our board of directors or the holders of 10% or more of all of our votes entitled to be cast on any issue proposed to be considered at the special meeting of shareholders;

 

no provision in our amended and restated articles of incorporation or amended and restated bylaws provides for cumulative voting, which limits the ability of minority shareholders to elect director candidates;

 

directors will only be able to be removed for cause;
   
our amended and restated articles of incorporation authorizes undesignated preferred stock, the terms of which may be established and shares of which may be issued, without the approval of the holders of our capital stock; and

 

certain litigation against us can only be brought in Florida.

 

These provisions could discourage, delay or prevent a transaction involving a change in control of our company. These provisions could also discourage proxy contests and make it more difficult for you and other shareholders to elect directors of your choosing and cause us to take corporate actions other than those you desire. See “Description of Capital Stock.”

 

Our amended and restated bylaws that will be in effect immediately prior to the completion of this offering designates the state courts located within the state of Florida as the exclusive forum for substantially all disputes between us and our shareholders and the federal district courts as the exclusive forum for Securities Act claims, which could limit our shareholders’ ability to obtain a favorable judicial forum for disputes with us.

 

Our amended and restated bylaws that will be in effect immediately prior to the completion of this offering provide that, unless we consent in writing to the selection of an alternative forum, the sole and exclusive forum for (i) any derivative action or proceeding brought on our behalf, (ii) any action asserting a claim of breach of a fiduciary duty owed by any of our current or former directors, officers or other employees to us or our shareholders, (iii) any action arising pursuant to any provision of the FBCA, our amended and restated articles of incorporation or our amended and restated bylaws, or (iv) any other action asserting a claim that is governed by the internal affairs doctrine shall be a state court located within the state of Florida (or, if a state court located within the state of Florida does not have jurisdiction, the federal district court for the Middle District of Florida); provided that, the exclusive forum provision will not apply to suits brought to enforce any liability or duty created by the Exchange Act, or to any claim for which the federal courts have exclusive jurisdiction. Our amended and restated bylaws that will be in effect immediately prior to the completion of this offering also provide that, unless we consent in writing to the selection of an alternative forum, the U.S. federal district courts shall be the exclusive forum for the resolution of any claims arising under the Securities Act. Under the Securities Act, federal and state courts have concurrent jurisdiction over all suits brought to enforce any duty or liability created by the Securities Act, and investors cannot waive compliance with the federal securities laws and the rules and regulations thereunder. Accordingly, there is uncertainty as to whether a court would enforce such a forum selection provision as written in connection with claims arising under the Securities Act.

 

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By becoming a shareholder in our company, you will be deemed to have notice of and have consented to the provisions of our amended and restated bylaws related to choice of forum. The choice of forum provisions in our amended and restated bylaws may limit our shareholders’ ability to obtain a favorable judicial forum for disputes with us. Additionally, the enforceability of choice of forum provisions in other companies’ governing documents has been challenged in legal proceedings, and it is possible that, in connection with any applicable action brought against us, a court could find the choice of forum provisions contained in our amended and restated bylaws to be inapplicable or unenforceable in such action. If so, we may incur additional costs associated with resolving such action in other jurisdictions, which could harm our business, results of operations, and financial condition.

 

Securities or industry analysts may not regularly publish reports on us, which could cause the price of our securities or trading volumes to decline.

 

The trading market for our securities could be influenced by research and reports that industry and/or securities analysts may publish us, our business, the market or our competitors. We do not have any control over these analysts and cannot be assured that such analysts will cover us or provide favorable coverage. If any of the analysts who may cover our business change their recommendation regarding our securities adversely, or provide more favorable relative recommendations about our competitors, the price of our securities would likely decline. If any analysts who may cover our business were to cease coverage or fail to regularly publish reports on us, we could lose visibility in the financial markets, which in turn could cause the price of our securities or trading volumes to decline.

 

We will likely conduct further offerings of our equity securities in the future, in which case your proportionate interest may become diluted.

 

We will likely be required to conduct equity offerings in the future to finance our current projects or to finance subsequent projects that we decide to undertake. If our common stock shares are issued in return for additional funds, the price per share could be lower than that paid by our current shareholders. We anticipate continuing to rely on equity sales of our common stock shares in order to fund our business operations. If we issue additional common stock shares or securities convertible into shares of our common stock, your percentage interest in us could become diluted.

 

We may issue shares of preferred stock in the future, which could make it difficult for another company to acquire us or could otherwise adversely affect holders of our common stock, which could depress the price of our common stock.

 

Our certificate of incorporation authorizes us to issue one or more series of preferred stock. Our board of directors will have the authority to determine the preferences, limitations and relative rights of the shares of preferred stock and to fix the number of shares constituting any series and the designation of such series, without any further vote or action by our shareholders. Our preferred stock could be issued with voting, liquidation, dividend and other rights superior to the rights of our common stock. The potential issuance of preferred stock may delay or prevent a change in control of us, discouraging bids for our common stock at a premium to the market price, and materially adversely affect the market price and the voting and other rights of the holders of our common stock.

 

We have never declared or paid any cash dividends or distributions on our capital stock. We do not anticipate paying any cash dividends on our common stock in the foreseeable future.

 

We have never declared or paid any cash dividends or distributions on our capital stock. We currently intend to retain our future earnings, if any, to support operations and to finance expansion and therefore we do not anticipate paying any cash dividends on our common stock in the foreseeable future.

 

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The declaration, payment and amount of any future dividends will be made at the discretion of the board of directors, and will depend upon, among other things, the results of our operations, cash flows and financial condition, operating and capital requirements, and other factors as the board of directors considers relevant. There is no assurance that future dividends will be paid, and, if dividends are paid, there is no assurance with respect to the amount of any such dividend.

 

USE OF PROCEEDS

 

The net proceeds to us from the sale of shares of common stock by us in this offering will be approximately $5.9 million, assuming an initial public offering price of $7.00 per share, and after deducting underwriting discounts and commissions and estimated offering expenses.

 

We intend to use the net proceeds from this offering as follows:

 

  approximately $3.0 million to advance our clinical development programs, including:
       
    approximately $2.0 million to fund our pre-clinical animal toxicology studies and CMC activities,
       
    approximately $0.3 million for expenses associated with our initial IND application, and
       
    approximately $0.7 million for expenses relating to our Phase I clinical trials for TELOMIR-1; and
       
  the remaining amounts to fund working capital and general corporate purposes.

 

Based on our current operating plan, we believe that the net proceeds of this offering, together with our existing cash and cash equivalents, will be sufficient to fund our operations and our planned development and research activities through at least January 2025.

 

We cannot specify with certainty the particular uses of the net proceeds that we will receive from this offering or the amounts we actually spend on the uses set forth above. Pending the use of proceeds from this offering as described above, we plan to invest the net proceeds that we receive in this offering in short-term and intermediate-term interest-bearing obligations, investment-grade investments, certificates of deposit or direct or guaranteed obligations of the U.S. government. Our management will have broad discretion in the application of the net proceeds from this offering and investors will be relying on the judgment of our management regarding the application of the proceeds.

 

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DIVIDEND POLICY

 

We have never declared or paid dividends on our capital stock. We currently intend to retain all available funds and future earnings, if any, for use in the operation of our business and do not anticipate paying any dividends on our common stock in the foreseeable future. Investors should not purchase our common stock with the expectation of receiving dividends.

 

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CAPITALIZATION

 

The following table sets forth our cash and cash equivalents, as well as our total capitalization, as of September 30, 2023 (after giving effect to the 1-for-2.05 reverse stock split that occurred on December 11, 2023):

 

  on an actual basis;
     
  on a pro forma basis giving effect to the following: (i) the incurrence of, in the aggregate, an additional $0.5 million in debt from September 30, 2023 through November 30, 2023, and (ii) the conversion of an aggregate of $3.1 million of outstanding debt into 1,512,478 shares of common stock, as described below, on November 30, 2023; and
     
  on a pro forma as adjusted basis to give effect to our issuance and sale of 1,000,000 shares of our common stock in this offering (assuming no exercise of the underwriters’ overallotment option) at an assumed initial public offering price of $7.00 per share, and after deducting underwriting discounts and commissions and estimated offering expenses payable by us.

 

You should read this table together with our financial statements and related notes and the section titled “Management’s Discussion and Analysis of Financial Condition and Results of Operations” that are included elsewhere in this prospectus.

 

   Actual    Pro Forma(1)   

Pro Forma

As Adjusted (2)

 
   (unaudited)    (unaudited)    (unaudited)  
             
Cash   $ 2,452    $ 2,452    $ 5,875,666  
Debt                
Related party line of credit   $ 1,337,914    $ -    $ -  
Due to related parties     1,293,070      525      525  
Stockholders’ deficit:                
Preferred Stock, no par value, 100,000,000 shares authorized and none issued or outstanding, actual, pro forma, and pro forma as adjusted     -      -      -  
Common Stock, no par value; 300,000,000 shares authorized, 28,609,814 issued and outstanding, actual and pro forma; 300,000,000 shares authorized and 29,609,814 shares issued and outstanding pro forma as adjusted     -      -      -  
Additional paid-in capital     6,915,000      10,015,579      15,888,793  
Accumulated deficit     (4,736,927 )     (4,736,927 )     (4,736,927 )
Total stockholders’ deficit     2,178,073      5,278,652      11,151,866  
Total capitalization   $ 4,809,057    $ 5,279,177    $ 11,152,391  

 

(1) Includes the conversion on November 30, 2023 of (i) $1.7 million of outstanding debt due to MIRALOGX LLC into 837,841 shares of common stock, and (ii) $1.4 million of outstanding debt due to the Bay Shore Trust into 674,637 shares of common stock, each such conversion made at a conversion price of $2.05 per share (after giving effect to the 1-for-2.05 reverse stock split that occurred on December 11, 2023). See “Management’s Discussion and Analysis of Financial Condition and Results of Operations—Liquidity and Capital Resources.”
(2) A $1.00 increase or decrease in the assumed initial public offering price per share of our common stock would increase or decrease each of cash, additional paid-in-capital and total capitalization on a pro forma as adjusted basis by approximately $0.92 million, assuming the number of shares of our common stock offered by us remains the same and after deducting the estimated underwriting discounts and commissions and offering expenses payable by us.

 

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DILUTION

 

If you invest in our common stock in this offering, your ownership interest will be immediately diluted to the extent of the difference between the public offering price per share of our common stock and the pro forma net tangible book value per share of our common stock immediately after this offering. Dilution in pro forma net tangible book value per share to new investors represents the difference between the amount per share paid by purchasers of shares of our common stock in this offering and the pro forma net tangible book value per share of our common stock immediately after completion of this offering.

 

Net tangible book value per share is determined by dividing our total tangible assets less our total liabilities and common stock in stockholders’ equity (deficit) by the number of shares of our common stock outstanding. Our historical net tangible book value (deficit) as of September 30, 2023, was approximately $2.1 million, or $0.04 per share. Our pro forma net tangible book value as of September 30, 2023 was approximately $2.0 million, or $0.03 per share, adjusted to give effect to the following: (i) the incurrence of, in the aggregate, an additional $0.5 million in debt from September 30, 2023 through November 30, 2023, and (ii) the conversion of an aggregate of $3.1 million of outstanding debt into 1,512,478 shares of common stock on November 30, 2023. After giving effect to the sale by us of shares of our common stock in this offering at the assumed public offering price of $7.00 per share, and after deducting underwriting discounts and commissions, and estimated offering expenses payable by us, our pro forma as adjusted net tangible book value as of September 30, 2023, would have been approximately $8.0 million, or $0.27 per share. This represents an immediate increase in pro forma net tangible book value of $0.23 per share to our existing stockholders and an immediate dilution in pro forma net tangible book value of $6.73 per share to investors purchasing shares of our common stock in this offering. The following table illustrates this dilution:

 

Public offering price per share of common stock       $ 7.00  
Pro forma net tangible book value per share as of September 30, 2023   $ 0.03       
Increase per share attributable to new investors purchasing shares of common stock in this offering    0.23       
Pro forma as adjusted net tangible book value per share immediately after this offering    0.27       
Dilution in pro forma as adjusted net tangible book value per share to new common stock investors in this offering       $ 6.73  

 

The following table presents, on a pro forma as adjusted basis as of September 30, 2023, after giving effect to (i) the incurrence of, in the aggregate, an additional $0.5 million in debt from September 30, 2023 through November 30, 2023, and (ii) the conversion of an aggregate of $3.1 million of outstanding debt into 1,512,478 shares of common stock on November 30, 2023, and (iii) the sale by us of 1,000,000 shares of our common stock in this offering at the assumed offering price of $7.00 per share, the difference between the directors, officers and their affiliates and the new investors purchasing shares of our common stock in this offering with respect to the number of shares of our common stock purchased from us, the total consideration paid or to be paid to us, and the average price per share paid or to be paid to us by such persons during the last five years and new investors, before deducting underwriting discounts and commissions and estimated offering expenses payable by us:

 

   Shares Purchased   Total Consideration   Average Price 
   Number   Percent   Amount   Percent   Per Share 
   ($ in millions) 
Directors, officers and their affiliates                 %  $                %  $        
New investors         %         %     
Total         %  $      %     

 

If the underwriters exercise in full their option to purchase 150,000 additional shares of our common stock from us in this offering, the pro forma as adjusted net tangible book value (deficit) per share after this offering would be $0.30 per share and the dilution to new investors in this offering would be $6.70 per share. If the underwriters exercise such option in full, the number of shares held by new investors will increase to approximately 1,150,000 shares of our common stock, or approximately 3.74% of the total number of shares of our common stock outstanding after this offering.

 

A $1.00 increase (decrease) in the assumed public offering price of $7.00 per share would increase (decrease) the pro forma as-adjusted net tangible book value per share by $0.03, and the dilution per share to new investors in this offering by $0.97, assuming the number of shares offered by us, as set forth on the cover page of this prospectus, remains the same and after deducting the underwriting discounts and commissions and estimated offering expenses payable by us.

 

In addition, we may choose to raise additional capital due to market conditions or strategic considerations, even if we believe we have sufficient funds for our current or future operating plans. To the extent that we raise additional capital through the sale of equity, as common stock, or other securities that are convertible into our common stock, such as convertible debt securities, the issuance of these securities could result in further dilution to our stockholders.

 

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MANAGEMENT’S DISCUSSION AND ANALYSIS OF

FINANCIAL CONDITION AND RESULTS OF OPERATIONS

 

The following discussion and analysis provide information which our management believes is relevant to an assessment and understanding of our results of operations and financial condition. You should read the following discussion and analysis of our results of operations and financial condition together with our financial statements and related notes and other information included elsewhere in this prospectus.

 

In addition to historical financial information, this discussion contains forward-looking statements based upon our current expectations that involve risks and uncertainties. Our actual results could differ materially from such forward-looking statements as a result of various factors, including those set forth under “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” included elsewhere in this prospectus. Additionally, our historical results are not necessarily indicative of the results that may be expected for any period in the future.

 

Overview

 

We are a pre-clinical-stage pharmaceutical company focused on the development and commercialization of TELOMIR-1, a novel small molecule being developed to function as an oral in situ therapeutic treatment for human stem cells. Our initial focus will be on treatments to inhibit the production of pro-inflammatory cytokines, such as IL-17, by oral administration of TELOMIR-1 as a therapeutic treatment for stem cells in situ. Our goal is to advance the clinical development of TELOMIR-1 in the United States for the treatment of age-related inflammatory conditions such as hemochromatosis and osteoarthritis, as well as in post-chemotherapy recovery, with our initial targeted indications being hemochromatosis and post-chemotherapy recovery.

 

We had net losses of $3.7 million and $0.4 million for the nine months ended September 30, 2023 and 2022, respectively. We had net losses of $0.9 million and $0.1 million for the years ended December 31, 2022 and December 31, 2021, respectively.

 

Reverse Stock Split

 

Effective December 12, 2023, we completed a reverse stock split of our outstanding common stock upon the filing of our Second Amended and Restated Articles of Incorporation with the Florida Secretary of State. No fractional shares were or will be issued in connection with the reverse stock split, and all such fractional shares resulting from the reverse stock split were and will be rounded up to the nearest whole number. The shares issuable upon the exercise of our outstanding warrants, and the exercise prices of such warrants, have been adjusted to reflect the reverse stock split. Unless otherwise noted, the share and per share information in this prospectus reflects the reverse stock split.

 

Supply Chain Disruption / COVID-19 Business Update

 

Due to the residual impact of the global COVID-19 pandemic, we have taken measures to secure our research and development activities, while work in laboratories and facilities has been organized to reduce the risk of COVID-19 transmission. The extent of the impact of the COVID-19 pandemic on our business, operations and clinical development timelines and plans remains uncertain, and will depend on certain developments, including the duration and spread of the outbreak and its impact on our clinical trials, CROs, manufacturing process, supply chain, and other third parties with whom we do business, as well as its impact on regulatory authorities and our key scientific and management personnel. While we are experiencing limited financial impacts at this time, given the global economic slowdown, the overall disruption of global supply chains and the other risks and uncertainties associated with the pandemic, our business, financial condition, and results of operations ultimately could be materially adversely affected. Some of our suppliers have experienced delays in securing critical raw materials; while this has not materially impacted their services, we have observed delays in certain activities. Therefore, we continue to closely monitor the COVID-19 pandemic as we evolve our business continuity plans, clinical development plans and response strategy.

 

Components of our Results of Operations

 

Research and Development Expenses

 

Research and development expenses represent costs incurred to conduct research and development of our product candidate. We recognize all research and development costs as they are incurred. Research and development expenses consist primarily of the following:

 

  contracted research and manufacturing;
     
  consulting arrangements; and
     
  other expenses incurred to advance the Company’s research and development activities.

 

Our operating expenses have historically been the costs associated with our initial investment in pre-clinical research and development activities. We expect research and development expenses will increase in the future as we advance TELOMIR-1 into and through clinical trials and pursue regulatory approvals, which will require a significant investment in costs of clinical trials, regulatory support, and contract manufacturing. In addition, we will evaluate opportunities to acquire or in-license additional product candidates and technologies, which may result in higher research and development expenses due to license fee and/or milestone payments, as well as added clinical development costs.

 

The process of conducting clinical trials necessary to obtain regulatory approval is costly and time consuming. We may never succeed in timely development and achieving regulatory approval for our product candidates. The probability of success of our product candidates may be affected by numerous factors, including clinical data, competition, manufacturing capability and commercial viability. As a result, we are unable to determine the duration and completion costs of our development projects or when and to what extent we will generate revenue from the commercialization and sale of our product candidates.

 

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General and Administrative Expenses

 

General and administrative expenses consist of administrative functions, as well as fees paid for legal, consulting fees and facilities costs not otherwise included in research and development expense. Legal costs include general corporate legal fees and patent costs. We expect to incur additional expenses as a result of becoming a public company, including expenses related to compliance with the rules and regulations of the SEC and Nasdaq, additional insurance, investor relations and other administrative expenses and professional services.

 

Results of Operations for nine months ended September 30, 2023 and 2022

 

   Nine Months Ended September 30, 
   2023   2022 
Revenues  $-   $- 
           
Operating costs:          
General and administrative expenses   204,902    11,150 
Related Party Travel costs   1,288,000    - 
Research and development expenses   1,370,522    355,901 
Total operating costs   2,863,424    367,051 
           
Interest Expense   (881,225)   - 
           
Net loss  $(3,744,649)  $(367,051)

 

General and Administrative Expenses. We incurred general and administrative expenses of $0.2 million and $0.01 million during the nine months September 30, 2023 and September 30, 2022, respectively. General and administrative expenses consisted of consulting fees, office and rent expenses.

 

Related Party Travel Costs. We incurred $1.3 million in related party travel costs during the nine months ended September 30, 2023. There was no such expense incurred during the same period ended September 30, 2022. Related party travel costs consisted of a shared lease and use of an airplane with an entity under common control. The increase in related party travel costs is due to CRO and vendor site visits, plus IPO related efforts.

 

Research and Development Expenses. We incurred research and development expenses of $1.4 million and $0.4 million during the nine months ended September 30, 2023 and September 2022, respectively. The increase in research and development expenses during 2023 compared to 2022 is due to the expansion of pre-clinical programs during 2023.

 

Major components of research and development expenses during 2023 is as follows:

 

R&D Category  Expense 
Toxicology  $0.5 million 
Pre-clinical research  $0.5 million 
R&D consultants  $0.4 million 

 

Interest expense. We recognized $0.9 million in interest expense during the nine months ended September 30, 2023. There was no such expense during the same period ended September 30, 2022. Interest expense during 2023 was composed of debt issuance costs related to a line of credit financing.

 

Results of Operations for years ended December 31, 2022 and 2021

 

   Year ended December 31, 
   2022   2021 
Revenues  $-   $- 
           
Operating costs:          
General and administrative expenses   20,941    11,639 
Research and development expenses   833,206    126,492 
Total operating costs   854,147    138,131 
           
Net loss  $(854,147)  $(138,131)

 

General and Administrative Expenses. We incurred general and administrative expenses of $0.02 million and $0.01 million during the year ended December 31, 2022 and December 31, 2021, respectively. General and administrative expenses consisted of consulting fees, office and rent expenses.

 

Research and Development Expenses. We incurred research and development expenses of $0.8 million and $0.1 million during the year ended December 31, 2022 and December 31, 2021, respectively. The increase in research and development expenses during 2022 compared to 2021 is due to the expansion of pre-clinical programs during 2022 as our contract research organizations (“CROs”) began substantive pre-clinical efforts on TELOMIR-1.

 

Major components of research and development expenses during 2022 is as follows:

 

R&D Category  Expense 
Toxicology  $0.2 million 
Pre-clinical research  $0.4 million 
R&D consultants  $0.2 million 

 

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Liquidity and Capital Resources

 

Sources of Liquidity

 

Since the Company’s inception in August 2021, we have financed our operations primarily through an unsecured line of credit with a major shareholder and an affiliated company and through a private placement of shares of our common stock that occurred during the first quarter 2023. We intend to finance our clinical development programs and working capital needs from existing cash, potential new sources of debt and equity financing, including the proceeds from our anticipated initial public offering. We may enter into new licensing and commercial partnership agreements.

 

On June 15, 2023, we entered into a Promissory Note and Loan Agreement with the Bay Shore Trust, a trust established by our founder, Jonnie R. Williams, Sr., and under which various of his family members are beneficiaries. Under this Promissory Note and Loan Agreement (the “Bay Shore Note”), we have the right to borrow up to an aggregate of $5 million from the Bay Shore Trust at any time up to the second anniversary of the issuance of the Bay Shore Note or, if earlier, upon the completion of our initial public offering (“IPO”). Our right to borrow funds under the Bay Shore Note is subject to the absence of a material adverse change in its assets, operations, or prospects. The Bay Share Note, together with accrued interest, will become due and payable on the second anniversary of the issuance of the note, provided that it may be prepaid at any time without penalty. The Bay Shore Note will accrue interest at a rate equal to 7% per annum, simple interest, during the first year that the note is outstanding and 10% per annum, simple interest, thereafter. The Bay Shore Note is unsecured. As of November 30, 2023, the total amount outstanding under the Bay Shore Note was $1.4 million. The total amount outstanding was converted into 674,637 shares of our common stock on November 30, 2023 at a conversion rate of $2.05 per share (after giving effect to our 1-for-2.05 reverse stock split that occurred on December 11, 2023) pursuant to a conversion agreement.

 

Since January 1, 2023, MIRALOGX, an intellectual property development and holding company owned by Bay Shore Trust, and The Starwood Trust, a separate trust established by our founder, have advanced funds on behalf of Bayshore Trust to our company in order to fund operating activities. The total amount advanced and outstanding as of November 30, 2023, was $1.7 million. These advances were converted into 837,841 shares of our common stock on November 30, 2023 at a conversion rate of $2.05 per share (after giving effect to our 1-for-2.05 reverse stock split that occurred on December 11, 2023) pursuant to a conversion agreement.

 

In consideration of the loan facility provided by the Bay Shore Trust, we issued to the Bay Shore Trust a common stock purchase warrant on June 15, 2023 giving the Bay Shore Trust the right to purchase up to 2,439,024 shares of common stock at an exercise price of $3.73 per share (after giving effect to our 1-for-2.05 reverse stock split that occurred on December 11, 2023), which warrant will expire five years after the date of grant

 

As of September 30, 2023 and December 31, 2022, we had cash of $0.002 million and $0.001 million, respectively. In total, we have raised $1.0 million in 2023 through the issuance of equity. Substantially all our equity capital has been raised at $3.73 per share (after giving effect to our 1-for-2.05 reverse stock split that occurred on December 11, 2023). We used $0.5 million in operating activities during the year ended December 31, 2022, compared to $0.1 million in operating activities during the year ended December 31, 2021. We expect that our existing cash and available line of credit, before our anticipated initial public offering, will be sufficient to finance our planned level of operations through April 2024.

 

Prior to commercializing or monetizing our product candidates, we anticipate that additional capital will be required to support ongoing activities and further phases of development. Should that be required, our available capital may be consumed more rapidly than currently anticipated, resulting in the need for additional funding. In addition, there can be no assurance that additional funding, when and if required, will be available at commercially favorable terms, if at all.

 

Accordingly, we may need to raise additional capital, which may be available to us through a variety of sources, including:

 

  public equity markets;
     
  private equity financings;
     
  commercialization agreements and collaborative arrangements;

 

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  sale of product royalty;
     
  grants and new license revenues;
     
  bank loans; and
     
  public or private debt.

 

Additional funding, capital, or loans (including, without limitation, milestone, or other payments from potential commercialization agreements) may be unavailable on favorable terms, if at all. If adequate funds are not available, we may be required to significantly reduce or refocus our operations or to obtain funds through arrangements that may require us to relinquish rights to certain technologies and drug formulations or potential markets, any of which could have a material adverse effect on us, our financial condition, and our results of operations. To the extent that additional capital is raised through the sale of equity or convertible debt securities or exercise of warrants and options, the issuance of such securities would result in ownership dilution to existing stockholders.

 

If we are unable to attract additional funds on commercially acceptable terms, it may adversely affect our ability to achieve our development and commercialization goals, which could have a material and adverse effect on our business, results of operations and financial condition.

 

Cash Flows

 

The following table provides information regarding our cash flows for the periods presented:

 

   Nine Months Ended
September 30, 2023
   Year Ended
December 31, 2022
   Period from August 2021 (Inception) to December 31, 2021 
Net cash provided by (used in):               
Operating activities  $(2,772,581)  $(468,661)  $(119,396)
Financing activities   2,773,614    470,080    119,396 
Net change in cash  $1,033   $1,419   $- 

 

Net Cash Used in Operating Activities

 

The cash used in operating activities resulted primarily from our net losses and changes in components of accounts payable and accrued liabilities.

 

From August 2021 (inception) to December 31, 2021, operating activities used $0.12 million of cash, primarily due to a net loss of $0.14 million, offset by a $0.02 million in accounts payable and accrued liabilities. Accounts payable and accrued liabilities was composed of research and development payables and accrued rent expense.

 

For year ended December 31, 2022, operating activities used $0.47 million of cash, primarily due to a net loss of $0.85 million, offset by a $0.38 million change in accounts payable. Accounts payable was composed of research and development payables and rent expenses.

 

For the nine months ended September 30, 2023, operating activities used $2.3 million of cash, primarily due to a net loss of $3.8 million, offset by a $0.1 million change in accounts payable and $0.9 million in amortization of debt issuance costs. Accounts payable was composed of research and development payables, rent and legal expenses.

 

Net Cash Provided by Financing Activities

 

From August 2021 (inception) to December 31, 2021, financing activities provided $0.12 million of cash, resulting from $0.12 million in amounts due to a related party. Related party advances were used to pay research and development payables.

 

For year ended December 31, 2022, financing activities provided $0.47 million of cash, resulting from $0.46 million in amounts due to a related party, $0.06 million in collection of stock subscription receivable, offset by a $0.05 million in deferred offering costs.

 

For the nine months ended September 30, 2023, financing activities provided $2.8 million of cash, resulting from $0.9 million in issuance of warrants, net of deferred offering costs, offset by $1.9 million in advances to affiliates.

 

Future Funding Requirements

 

To date, we have not generated any revenue from product sales. We do not expect to generate revenue from product sales unless and until we successfully complete pre-clinical and clinical development of, receive regulatory approval for, and commercialize a program and we do not know when, or if at all, that will occur. We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we advance the pre-clinical activities and studies and initiate clinical trials. In addition, if we obtain regulatory approval for any programs, we expect to incur significant expenses related to product sales, marketing, and distribution to the extent that such sales, marketing and distribution are not the responsibility of potential collaborators. Further, upon the completion of this offering, we expect to incur additional costs associated with operating as a public company. The timing and amount of our operating expenditure will depend largely on the factors set out above. For more information, see the section titled “Risk Factors-Risks Related to Our Operations and Financial Condition.”

 

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Our funding requirements and timing and amount of our operating expenditures will depend on many factors, including, but not limited to:

 

■ the rate of progress in the development of our TELOMIR-1 program and other development programs;

 

■ the scope, progress, results and costs of pre-clinical studies and clinical trials for any other current and future programs;

 

■ the number and characteristics of programs and technologies that we develop or may in-license;

 

■ the costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our programs for which we receive marketing approval;

 

■ the costs necessary to obtain regulatory approvals, if any, for any approved products in the United States and other jurisdictions, and the costs of post-marketing studies that could be required by regulatory authorities in jurisdictions where approval is obtained;

 

■ the costs and timing of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defending any intellectual property-related claims;

 

■ the continuation of our existing licensing arrangements and entry into new collaborations and licensing arrangements;

 

■ the costs we incur in maintaining business operations;

 

■ the costs of hiring additional clinical, quality control, manufacturing and other scientific personnel;

 

■ the costs adding operational, financial and management information systems and personnel;

 

■ the costs associated with being a public company;

 

■ the revenue, if any, received from commercial sales of our programs for which we receive marketing approval;

 

■ the effect of competing technological and market developments; and

 

■ the extent to which we acquire or invest in businesses, products and technologies, including entering into licensing or collaboration arrangements for programs.

 

Identifying potential programs, product candidates, conducting pre-clinical studies and clinical trials is a time consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. In addition, our programs, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for many years, if ever. Accordingly, we will need to obtain substantial additional funds to achieve our business objectives.

 

Recently Issued and Adopted Accounting Pronouncements

 

On January 1, 2022, we adopted Accounting Standards Codification (“ASC”) Topic 842 ASC Topic 842, which is intended to improve financial reporting about leasing transactions. Under the standard, organizations that lease assets, referred to as “Lessees” shall recognize on the balance sheet the assets and liabilities for the rights and obligations created by those leases. In addition, the standard requires disclosures including financial statements to assess the amount, timing and uncertainty of cash flows arising from leases. We do not expect that the adoption of Topic 842 will materially impact our Company’s operations.

 

Off-Balance Sheet Arrangements

 

During the periods presented, we did not have, nor do we currently have, any off-balance sheet arrangements as defined under Generally Accepted Accounting Principles (GAAP) and SEC rules.

 

Quantitative and Qualitative Disclosures about Market Risk

 

We are exposed to market risks in the ordinary course of our business. These risks primarily include interest rate risks and inflation risks. Periodically, we maintain deposits in accredited financial institutions in excess of the FDIC federally insured limits. We deposit our cash in financial institutions that we believe have high credit quality and have not experienced any losses on such accounts and do not believe we are exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships.

 

Interest Rate Risk

 

Our cash consists of cash in readily available checking accounts. We may also invest in short-term money market fund investments. Such interest-earning instruments carry a degree of interest rate risk; however, historical fluctuations in interest income have not been significant.

 

Inflation Risk

 

Inflation generally affects us by increasing our cost of labor and research and development contract costs. We do not believe inflation has had a material effect on our results of operations during the periods presented.

 

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BUSINESS

 

Overview

 

We are a pre-clinical-stage pharmaceutical company focused on the development and commercialization of TELOMIR-1, a novel small molecule being developed to function as an oral in situ therapeutic treatment for human stem cells. Based on our pre-clinical studies and if approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 may effectively serve as a metal enzyme inhibitor of essential metals such as zinc and copper. These essential metals play an important role in the production and function of many enzymatic reactions and the modulation of key cellular pathways. In particular, zinc is essential to the function of pro-inflammatory cytokines such as Interleukin-17, or IL-17, that play a role in a host of age-related inflammatory conditions such as hemochromatosis and osteoarthritis as well as in post-chemotherapy health problems. IL-17 is a type of pro-inflammatory cytokine, and while the pro-inflammatory properties of IL-17 are key to its host-protective capacity, unrestrained IL-17 signaling is associated with immunopathology, inflammatory disease and cancer progression. Our initial focus will be on treatments to inhibit the production of pro-inflammatory cytokines, such as IL-17, by oral administration of TELOMIR-1 as a therapeutic treatment for stem cells in situ. To the best of our knowledge, there is no approved oral IL-17 inhibitor. Our goal is to advance the clinical development of TELOMIR-1 in the United States for the treatment of age-related inflammatory conditions such as hemochromatosis and osteoarthritis, as well as in post-chemotherapy recovery, with our initial targeted indications being hemochromatosis and post-chemotherapy recovery.

 

Pluripotent stem cells are a type of stem cells that have the ability to undergo self-renewal and to give rise to various cell types of the tissues of the body. Based on pre-clinical studies, we believe that TELOMIR-1 may have the potential to protect stem cells in situ by reducing the overload of metals such as zinc and copper that accompany age-related inflammatory conditions and certain cancers by modulating pro-inflammatory cytokines such as IL-17. If demonstrated in clinical trials and approved by the FDA and comparable foreign regulators, we believe that TELOMIR-1 may protect the stem cells by elongating and stimulating the telomeres to sustain self-renewal of stem cells. Telomeres are repetitive DNA sequences at the end of chromosomes that protect the chromosomes from becoming frayed or tangled. Each time a cell divides, the telomeres become slightly shorter, and eventually they become so short that the cell can no longer divide, with the result being that the cell dies. Effectively, telomeres protect the ends of our chromosomes by forming a cap, much like the plastic tip on shoelaces, thereby allowing the chromosome to be replaced properly during cell division.

 

TELOMIR-1 is currently under investigation to provide a potential therapeutic intervention against age-related inflammatory conditions such as hemochromatosis, as well as for post-chemotherapy recovery, by interrupting and preventing the IL-17 induced inflammatory pathways that create the systemic imbalance of cellular metals. Our pre-clinical studies suggest that TELOMIR-1 may inhibit the concentration and accumulation of metals such as zinc and copper in the serum that drive the hyperactivity of pro-inflammatory cytokines such as IL-17. Our studies suggest that TELOMIR-1 may achieve this outcome by selectively binding to metal ions in a dose dependent manner, slowing enzyme reactivity, and protecting and lengthening telomeres in the human chromosome. We believe that TELOMIR-1 has potential as a non-toxic oral enzyme inhibitor that may regulate the overactivity of the enzymes caused by excessive metal reactivity.

 

To date, we have completed several pre-clinical proof-of-concept studies with respect to TELOMIR-1 that were designed to demonstrate that TELOMIR-1 is not mutagenic and has good biological and metal binding capabilities. These studies included an Ames test for the TELOMIR-1 compound and several Eurofins BioMap studies.

 

We were organized as a Florida corporation in August 2021 for the purpose of pursuing the development and commercialization of TELOMIR-1 in the United States in human applications. We were originally incorporated under the name “Metallo Therapies Inc.” and changed our name to “Telomir Pharmaceuticals, Inc.” in October 2022. Our rights to develop and commercialize TELOMIR-1 are licensed to us under a license agreement with MIRALOGX under which we have the exclusive right to develop and commercialize TELOMIR-1 for human and non-human applications in the United States. See “—Intellectual Property.”

 

We are an early-stage company that had net losses of $3.9 million for the nine months ended September 30, 2023, $0.85 million for the year ended December 31, 2022, and $0.14 million for the year ended December 31, 2021.

 

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In Situ Therapeutic Treatment of Stem Cells

 

Stem cells have the potential to renew themselves. They can develop into many different cell types in the body during early and adult life. Pluripotent stem cells have the ability to differentiate into all of the cells of the adult body. Since pluripotent stem cells are undifferentiated, they do not have any tissue-specific characteristics that allow them to perform specialized functions. Given their unique regenerative abilities and limited quantities in the adult human body, in situ treatment and protection of stem cells may provide an important therapeutic mechanism for treatment of disease.

 

Metal Overload and Telomere Length

 

Research studies have demonstrated that metal overload in stem cells could severely impair the proliferation of pluripotent stem cells through excessive DNA damage. Furthermore, many pro-inflammatory cytokine-induced conditions create an imbalance of cellular metals such as zinc and copper. This metal imbalance may impact the length of telomeres in stem cells and impact their viability and survival.

 

Hemochromatosis

 

According to the Centers for Disease Control (“CDC”), hemochromatosis is a disorder in which the body builds up too much iron in the skin, heart, liver, pancreas, pituitary glands, and joints. This overload of iron is toxic to the body, and over time, the high levels of iron can damage tissues and organs and lead to conditions such as liver damage, liver cancer, heart problems, arthritis, and diabetes. Hemochromatosis is most commonly hereditary and caused by certain variants in the HFE gene. In the U.S., about 1 in 300 non-Hispanic white persons have hereditary hemochromatosis, with lower rates among other races and ethnicities. Hemochromatosis can also result in persons who need multiple blood transfusions, which can lead to excess iron build up in a condition also referred to as “iron overload.” Other conditions associated with high iron levels include inflammatory conditions, chronic kidney disease, and autoimmune disorders.

 

Hemochromatosis is a life-long condition requiring regular treatment to avoid long-term serious effects with poor pharmacologic options. The most used treatment for hemochromatosis is phlebotomy, a procedure to remove some of the patient’s blood. Phlebotomy is relatively inexpensive, well accepted, and well tolerated, but it requires regular visits to health care professionals and blood draws and may not be appropriate for all patients. Additionally, iron chelators are sometimes used, but they have limited use due to gastrointestinal and kidney toxicity issues. Despite the presence of these potential treatment options, we believe significant unmet need remains such that an innovative therapy achieving reduction in iron overload with a new profile could be subject to widespread use.

 

If approved by the FDA and comparable foreign regulators, we are pursuing TELOMIR-1 as a metal enzyme inhibitor that may selectively bind metal ions to inhibit pro-inflammatory cytokines such as IL-17 and protect telomeres and that aims to increase the average telomere length, or ATL, by selectively binding iron and reducing blood iron levels, therefore supporting the ability to treat hemochromatosis. On average, about 750,000 US patients express one or more iron overload symptoms. There are 2 types of hemochromatosis, with the following patient mix: 150,000 primary hemochromatosis and 65,000 secondary hemochromatosis confirmed diagnoses in the United States. Over 150,000 patients have sought treatment since 2018, with most receiving phlebotomy, which is the withdrawal of blood to bring iron to normal levels. At present, 99% of hemochromatosis patients receive phlebotomy, with a portion of patients also receiving iron chelators as a co-treatment. Note that in the last 12 months, there have been 76,000 confirmed diagnoses of hereditary hemochromatosis and approximately 1% of hemochromatosis patients received deferasirox or deferoxamine off-label for 12 weeks (1 year for primary). Certain sub-groups of hemochromatosis patient may benefit from TELOMIR-1 in addition to phlebotomy, which is the current standard of care for the treatment of hemochromatosis if contraindicated for this treatment method. Lastly, iron chelation is used off-label, poorly tolerated and has toxic long-term effects. In pre-clinical stage testing, TELOMIR-1 is potentially has more effective metal complexing than Doxycycline, an FDA-approved MMP inhibitor. However, pre-clinical data may prove inaccurate and is not necessarily indicative of future results.

 

Post-Chemotherapy Recovery

 

Post-chemotherapy recovery from adverse effects of antineoplastic treatments is often important for cancer therapy success. While chemotherapy treatment can be highly effective for cancer, it can also come with many side effects, as chemotherapy drugs destroy both cancerous and healthy cells. According to a study published in the British Journal of Clinical Pharmacology1, of approximately 650,000 patients who use chemotherapy each year, only a fraction use drugs for side effects. Side effects and toxicity of chemotherapy lead to reduced compliance, and therefore treating chemotherapy side effects can increase compliance. Common barriers center around the complexity of post-chemotherapy side effects, obtaining reimbursement without significant outcomes, avoiding pauses, and/or delays in treatment due to side effects.

 

We are investigating the use of TELOMIR-1 as a potential complementary treatment for patients receiving chemotherapy in the form of a twice daily, oral regimen to inhibit pro-inflammatory cytokines and to reduce blood iron levels, potentially enabling more effective adherence and improved outcomes. In exploratory early discovery studies, proof of concept was demonstrated, and the animal studies are pending. Post-chemotherapy recovery space contains several treatments that are used symptomatically based on the nature or severity of side effects.

 

Post-chemotherapy recovery may present several potential unmet commercial needs for TELOMIR-1. There is potential to enable a more rapid recovery of patients receiving chemotherapy, improving adherence with chemotherapy regimens. Additionally, as current recovery management is based on severity and treated as needed, we believe that TELOMIR-1 could potentially be used as a preventative measure. As a complementary treatment, side effect reduction in post-chemotherapy is important to minimize patient burden while improving treatment outcomes.

 

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Our Strategy

 

Our goal is to develop and commercialize new treatment options for treatment of age-related inflammatory conditions such as hemochromatosis as our initial clinical focus and to thereafter expand the development of TELOMIR-1 to post-chemotherapy recovery as well as broader range of other age-related inflammatory diseases and conditions such as osteoarthritis. The key elements of our strategy to achieve this goal include:

 

  Advancing TELOMIR-1 through clinical development and approval for hemochromatosis. Our product candidate, TELOMIR-1, is in pre-clinical studies. Existing treatment options for hemochromatosis have significant limitations, and, if approved, we believe TELOMIR-1 may represent a major therapeutic advancement for patients.
     
  Explore the potential of TELOMIR-1 for Post-Chemotherapy Recovery. TELOMIR-1 may represent a major therapeutic advancement for post-chemotherapy patients. Our product candidate, TELOMIR-1, may have the potential in post-chemotherapy recovery.
     
  Continuing pre-clinical development of TELOMIR-1 across a range of inflammatory diseases associated with cellular aging. Given the impact of telomere cap regrowth on a variety of age-related inflammatory diseases, we intend to explore TELOMIR-1’s broader potential, as we believe TELOMIR-1 may have potential in several inflammatory diseases, such as osteoarthritis, associated with a range of metabolic and geriatric related conditions.
     
  Exploring strategic collaborations to maximize the value of TELOMIR-1. We plan to explore collaborations opportunistically to maximize the value of TELOMIR-1. We intend to retain significant economic and commercial rights to our programs.

 

Completed Proof-of-Concept and Pre-Clinical Studies

 

The Ames Test - Computational Mutagenicity

 

The use of in silico (computational) tools to predict toxicity has increased significantly and has become well-established not only in the pharmaceutical industry, but also in the chemical and cosmetic industries. This is specifically reflected in the area of potential genotoxic impurities and finalization in 2015 of the ICH M7 guidance, which was the first regulatory document that supports use of in silico tools as an initial surrogate for conducting in vitro or in vivo testing (ICH M7_Sept 5, 2015). The purpose of the M7 guidance is to aid in the identification and characterization of impurities with mutagenic risk and to outline the control strategy for the various classes (Class 1-5) of compounds in order to limit potential carcinogenic risk to humans. According to ICH M7, “the absence of structural alerts from two complementary quantitative structure activity relationship (“(Q)SAR”) methodologies (expert rule-based and statistical) is sufficient to conclude that the impurity is of no mutagenic concern, and no further testing is recommended.

 

The primary endpoint of concern in the ICH M7 guidance is DNA reactive mutagenicity, with the Ames bacterial mutagenicity assay being the preferred assay for this endpoint. The structure based in silico evaluations demonstrate reasonable ability to differentiate mutagens from non-mutagens with generally high concordance when compared with Ames assay results (Sutter et al, 2013).

 

The primary purpose of this study was to evaluate the bacterial mutagenicity of three potential drug candidates by testing the structures in at least two complementary in silico software programs, followed by expert review of the in silico data. The emphasis of the overall computational assessment of toxicity was placed on the potential for DNA reactive mutagenicity, in accordance with the ICH M7 guidance.

 

In rule-based systems, TELOMIR-1 was predicted non-reactive (no mutagenic concern. Therefore, according to ICH M7 criteria with negative predictions in two complementary in silico systems, it is considered non-mutagenic (Class 5).

 

The Ames Test - Computational Mutagenicity study was not powered for statistical significance.

 

BioMap Studies

 

 

 

The BioMAP® Platform provides an unbiased, target-agnostic and data-driven approach to understanding the effect of mono- or combination therapies on human disease models and translational biomarkers. Validated with clinically approved drugs and known test agents, the BioMAP Platform is powered by human primary cultures or co-cultures stimulated to recapitulate specific disease states and qualified with clinically approved and investigative test agents, a Reference Database of over 4,500+ molecules, data analytics, and expert interpretation to provide clients with actionable insights.

 

System Name   Icon   Human Cell Types   Stimulation   Disease/Tissue Relevance   Biomarker Readouts   System Description
3C   Red blood cells in a blue circle

Description automatically generated   Venular endothelial cells   TNFα, IL-1β, IFNγ   Cardiovascular Disease, Chronic Inflammation   CCL2/MCP-1, CD106/VCAM-1, CD141/Thrombomodulin, CD142/Tissue Factor, CD54/ICAM-1, CD62E/E-Selectin, CD87/uPAR, CXCL8/IL-8, CXCL9/MIG, HLA-DR, Proliferation, SRB   The 3C system models Th1 type vascular inflammation and is an anti-angiogenic environment that promotes monocyte and T cell adhesion and recruitment. The 3C system is relevant for chronic inflammatory diseases, vascular inflammation and restenosis.
4H   A red cell in a circle

Description automatically generated   Venular endothelial cells   IL-4, Histamine   Autoimmunity, Allergy, Asthma   CCL26/Eotaxin-3, CCL2/MCP-1, CD106/VCAM-1, CD62P/P-Selectin, CD87/uPAR, SRB, VEGFR2   The 4H system models Th2 type vascular inflammation and is a pro-angiogenic environment that promotes mast cell, basophil, eosinophil, T and B cell recruitment. The 4H system is relevant for diseases where Th2-type inflammatory conditions play a role such as allergy, asthma, and ulcerative colitis.
LPS   A red and blue cell

Description automatically generated   Venular endothelial cells, Peripheral blood mononuclear cells   TLR4 ligand   Chronic Inflammation, Cardiovascular Disease   CCL2/MCP-1, CD106/VCAM-1, CD141/Thrombomodulin, CD142/Tissue Factor, CD40, CD62E/E-Selectin, CD69, CXCL8/IL-8, IL-1 alpha, M-CSF, sPGE2, SRB, sTNF-alpha   The LPS system models Th1 type chronic inflammation and monocyte activation responses. The LPS system is relevant to chronic inflammatory conditions where monocytes play a key role including atherosclerosis, restenosis, rheumatoid arthritis, and metabolic diseases.
SAg   A yellow circle with red and blue circles

Description automatically generated   Venular endothelial cells, Peripheral blood mononuclear cells   TCR ligands (1X)   Chronic Inflammation, Autoimmune Disease   CCL2/MCP-1, CD38, CD40, CD62E/E-Selectin, CD69, CXCL8/IL-8, CXCL9/MIG, PBMC Cytotoxicity, Proliferation, SRB   The SAg system models Th1 type chronic inflammation and T cell effector responses related to T cell proliferation and activation in the context of the vascular endothelium. Modeling chronic inflammation of the Th1 type and T cell effector responses, the SAg systemis relevant for T-cell driven inflammatory conditions including organ transplantation, rheumatoid arthritis, psoriasis, Crohn’s disease and hematological oncology.
BT   A red circle with blue and purple circles

Description automatically generated   Peripheral blood mononuclear cells, B cells   α-lgM, TCR ligands (0.001X, sub-mitogenic levels)   Asthma, Oncology, Autoimmunity, Allergy   B cell Proliferation, PBMC Cytotoxicity, Secreted lgG, sIL-17A, sIL-17F, sIL-2, sIL-6, sTNF-alpha   The BT system models the T cell dependent B cell proliferation, activation and class switching that occurs in the germinal centers of secondary lympnoid organs. The BT system is relevant for indications in which B cell activation and antibody production have been implicated including systemic lupus erythematosus (SLE), hematological oncology, autoimmune indications, asthma and allergy.
BF4T   A logo of a cartoon character

Description automatically generated with medium confidence   Bronchial epithelial cells, Dermal fibroblasts   IL-4, TNFα   Fibrosis, Lung Inflammation, Asthma, Allergy   CCL26/Eotaxin-3, CCL2/MCP-1, CD106/VCAM-1, CD54/ICAM-1, CD90, CXCL8/IL-8, IL-1 alpha, Keratin 8/18, MMP-1, MMP-3, MMP-9, PAI-I, SRB, tPA, uPA   The BF4T system models Th1 type lung inflammation and is an environment that promotes the recruitment of eosinophils, mast cells and basophils as well as effector memory T cells. The BF4T system is relevant for allergy and asthma, pulmonary fibrosis, as well as COPD exacerbations.
BE3C   A group of green and red objects in a blue circle

Description automatically generated   Bronchial epithelial cells   IL-1β, IFNγ, TNFα   COPD, Lung Inflammation   CD54/ICAM-1, CD87/uPAR, CXCL10/IP-10, CXCL11/I-TAC, CXCL8/IL-8, CXCL9/MIG, EGFR, HLA-DR, IL-1 alpha, Keratin 8/18, MMP-1, MMP-9, PAI-I, SRB, tPA, uPA   The BE3C system models Th1 type lung inflammation and is an environment that promotes monocyte and T cell adhesion and recruitment. The BE3C system is relevant for sarcoidosis and pulmonary responses to respiratory infections.
CASM3C   A blue circle with red and white objects in it

Description automatically generated   Coronary artery smooth muscle cells   IL-1β, IFNγ, TNFα   Cardiovascular Inflammation, Restenosis   CCL2/MCP-1, CD106/VCAM-1, CD141/Thrombomodulin, CD142/Tissue Factor, CD87/uPAR, CXCL8/IL-8, CXCL9/MIG, HLA-DR, IL-6, LDLR, M-CSF, PAI-I, Proliferation, Serum Amyloid A, SRB   The CASM3C system models Th1 type vascular inflammation and is an environment that promotes monocyte and T cell recruitment. The CASm3C system is relevant for chronic inflammatory diseases, vascular inflammation and restenosis.
HDF3CGF   A blue circle with orange and red shapes

Description automatically generated   Dermal fibroblasts   IFNγ, TNFα, IL-1β, EGF, bFGF, PDGF-BB   Fibrosis, Chronic Inflammation   CCL2/MCP-1, CD106/VCAM-1, CD54/ICAM-1, Collagen I, Collagen III, CXCL 10/IP-10, CXCL11/1-TAC, CXCL8/IL-8, CXCL9/MIG, EGFR, M-CSF, MMP-1, PAI-I, Proliferation_72hr, SRB, TIMP-1, TIMP-2   The HDF3CGF system models wound healing and matrix/tissue remodeling in the context of Th1-type inflammation. The HDF3CGF system is relevant for various diseases including fibrosis, rheumatoid arthritis, psoriasis as well as stromal biology in tumors.
KF3CT   A green circle with yellow and orange shapes

Description automatically generated   Keratinocytes, Dermal fibroblasts   IL-1β, IFNγ, TGFβ, TNFα   Dermatitis, Psoriasis   CCL2/MCP-1, CD54/ICAM-1, CXCL 10/IP-10, CXCL8/IL-8, CXCL9/MIG, IL-1 alpha, MMP-9, PAI-I, SRB, TIMP-2, uPA   The KF3CT system models model Th1 type cutaneous inflammation and is an environment that promotes monocyte and T cell adhesion and recruitment. The KF3CT system is relevant for cutaneous responses to tissue damage caused by mechanical, chemical, or infectious agents as well as certain states of psoriasis and dermatitis.
MyoF   A group of orange and red stripes

Description automatically generated with medium confidence   Lung fibroblasts   TGFβ, TNFα   Wound Healing, Matrix Remodeling, Fibrosis, Chronic Inflammation   Alpha-SM Actin, bFGF, CD106/VCAM-1, Collagen I, Collagen III, Collagen IV, CXCL8/IL-8, Decorin, MMP-1, PAI-I, SRB, TIMP-1   The MyoF system models general myofibroblast differentiation and tissue remodeling relevant for multiple fibrotic diseases. Biomarker readouts capture impacts on translationally relevant matrix remodeling, tissue repair and inflammation related responses in fibrotic tissue.
Mphg   A logo of a heart

Description automatically generated   Macrophages, Venular endothelial cells   TLR2 ligand   Chronic Inflammation, Restenosis, Cardiovascular Disease   CCL2/MCP-1, CCL3/MIP-1 alpha, CD106/VCAM-1, CD40, CD62E/E-Selectin, CD69, CXCL8/IL-8, IL-1 alpha, M-CSF, sIL-10, SRB, SRB, SRB-Mphg   The /Mphg system models Th1 type chronic inflammation and macrophage activation responses. The /Mphg system is relevant to inflammatory conditions where monocytes play a key role including atherosclerosis, restenosis, rheumatoid arthritis, and other chronic inflammatory conditions.

 

https://www.eurofinsdiscovery.com/solution/biomap-platform

 

This study was conducted with the assistance of Eurofins, a third-party vendor.

 

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Eurofins BioMap® Diversity PLUS

 

BioMap® Diversity PLUS panel of human primary cell-based systems designed to model complex human tissue and diseases. The studies below with BioMap were conducted to investigate the anti-cancer activities versus Rapamycin. The BT system models the T cell proliferation activation and is relevant for indications in which B cell activation and antibody production have been implicated including autoimmune disorders. Rapamycin is selected to compare against TELOMIR-1 because rapamycin and TELOMIR-1 both inhibit similar cytokines involved in the immune responses. The common proteins affected between TELOMIR-1 and rapamycin are several pro-inflammatory cytokines.

 

The studies were not powered for statistical significance. They had no relationship to hemochromatosis. This study was conducted with the assistance of Eurofins, a third-party vendor.

 

BT   A red circle with blue and purple circles

Description automatically generated   Peripheral blood mononuclear cells, B cells   α-lgM, TCR ligands (0.001X, sub-mitogenic levels)   Asthma, Oncology, Autoimmunity, Allergy   B cell Proliferation, PBMC Cytotoxicity, Secreted lgG, sIL-17A, sIL-17F, sIL-2, sIL-6, sTNF-alpha   The BT system models the T cell dependent B cell proliferation, activation and class switching that occurs in the germinal centers of secondary lympnoid organs. The BT system is relevant for indications in which B cell activation and antibody production have been implicated including systemic lupus erythematosus (SLE), hematological oncology, autoimmune indications, asthma and allergy.

 

 

BioMAP® Diversity PLUS® Data Analysis Report: TELOMIR-1 versus Rapamycin

 

 

 

Biological and Disease Relevance Activity   Decreased Activity   Increase Activity   Modulated Activity
Inflammation-related activities   VCAM-1, ITAC, Psel, SAA, MCP-1, IL6, MIG, Eot3   sPGE2   sTNFa, IL8
Immunomodulatory activities   HLA-DR, sIgG, sIL-2, CD38, sIL-6, CD40, sIL-17A, sIL17F, sIL-10   CD69    
Tissue Remodeling Activities   TIMP1, PAI-I, CoI-III, CoI-IV        
Hemostasis-related activities   TM   TF    
Other activities   LDLR        

 

The study showed that TELOMIR-1 is an antiproliferative (suppress malignant cells) to human primary endothelial cells and fibroblasts vs Rapamycin.

 

This study was conducted with the assistance of Eurofins, a third-party vendor.

 

BioMAP® panels consist of human primary cell-based systems designed to model different aspects of the human body in an in vitro format. Rapamycin was approved in 1999 to prevent rejection of transplanted organs. There are approximately 1,073 clinical trials including rapamycin located on clinicaltrials.gov.

 

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BioMAP® Colorectal Cancer (CRC) Data Report: TELOMIR-1 vs. Rapamycin

 

 

 

 

Biological and Disease Relevance Activity   Decreased Activity   Increase Activity   Modulated Activity
Inflammation-related activities   sTNFa, VCAM-1, IP-10, MCP-1, MIG        
Matrix remodeling activities   TIMP2, CoI-I, COL-IV, MMP9       CoI-III
Angiogenesis-related activities   PAI-I, tPA, uPA   sVegF    
Tumor-related activities   CEACAM5, Keratin 20        
Immune-related activities   sIFNg, sGranB, sIL-10, sIL-2, sIL-6, CD40, CD69, sIL17-A        

 

In the Colorectal Cancer study, TELOMIR-1 was not cytotoxic at the concentrations tested in this study and was broadly active in the BioMAP Colorectal Cancer Panel with 32 activities versus Rapamycin.

 

TELOMIR-1 impacted inflammation-related, matrix remodeling, angiogenesis-related, tumor-related, and immune-related activities.

 

This study was conducted with the assistance of Eurofins, a third-party vendor.

 


BioMAP® Non-small Cell Lung Carcinoma (NSCLC) Data Report: TELOMIR-1 vs. Rapamycin 

 

 

 

Biological and Disease Relevance Activity   Decreased Activity   Increase Activity   Modulated Activity
Inflammation-related activities   sTNFa, VCAM-1, sMDC   IP-10    
Matrix remodeling activities       EGFR, CoI-III, HGF    
Angiogenesis-related activities   uPA   tPA   sVegF
Immune-related activities   sIL-4, sIFNg, sGranB, sIL-10, sIL-2, sIL17-A       sIL-13

 

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In the NSCLC study, TELOMIR-1 was active with 24 annotated readouts, 17 of which were in the StroNSCLC system, impacting inflammation-related, matrix remodelling, angiogenesis-related, and immune-related activities.

 

The Eurofins in vitro studies showed that TELOMIR-1 was more active against malignant cells, colorectal cancer cells, and lung cancer cells than rapamycin. Other studies showed that versus Doxycycline TELOMIR-1 was more active at metal complexing.

 

This study was conducted with the assistance of Eurofins, a third-party vendor.

 

TELOMIR-1 complexing ability compared to doxycycline study

 

Metal Complexing of TELOMIR-1 vs Doxycycline

 

Drug  Metal  Metal  LogK1   K1 
TELOMIR-1  Pb (II)  Lead   4.2    15,849 
TELOMIR-1  Cd (II)  Calcium   4.62    41,687 
TELOMIR-1  Zn (II)  Zinc   5.0    91,201 
TELOMIR-1  Co (II)  Cobalt   5.0    398,107 
TELOMIR-1  Ni (II)  Nickel   6.6    3,981,072 
Doxycycline  Zn (II)  Zinc   4.7    50,119 

 

K1 = stability constant (also called formation constant or binding constant) that represents the strength of association of compound with metal ion. The K1 measures the strength of the interaction between the TELOMIR and each metal when they come together to form the complex. TELOMIR-1 complexing ability compared to doxycycline and Zinc complexing ability. TELOMIR-1 is a good overall metal chelator. Results indicate TELOMIR-1 is potentially more effective than Doxycycline in Zinc complexing or binding.

 

This study was not powered for statistical significance. This study started in Q1 2023 and was completed in Q2 2023.

 

This study was conducted with the assistance of the University of North Carolina - Wilmington, a third party vendor.

 

Extensive analysis is being conducted on the potential of TELOMIR-1 to protect and treat stem cells and lower blood ferritin. We have also completed a variety of toxicology studies, and several others are ongoing or planned. Upon completion of the toxicology studies, we plan to submit to the FDA an IND for TELOMIR-1, focused on investigating TELOMIR-1 for the treatment of hemochromatosis. We may consider a second IND for post-chemotherapy recovery.

 

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(Source: Rini, D. John Hopkins School of Medical Arts)

 

In silico Affinity Studies

 

Subject to approval by the FDA and comparable foreign regulators, our pre-clinical studies demonstrate that TELOMIR-1 may uniquely and selectively bind critical metals to interrupt enzyme function, and may return cellular metals like zinc, copper and, iron to homeostasis. TELOMIR-1 may selectively affect the key cytokine-driven inflammatory processes that drive the concentration and accumulation of zinc, copper and iron in the serum that affects diseases like hemochromatosis and cancer. However, pre-clinical data may prove inaccurate and is not necessarily indicative of future results and drug safety and efficacy is within the sole authority of the FDA and comparable foreign regulators. If approved by the FDA or applicable foreign regulatory bodies, we believe that TELOMIR-1 may have the potential to be a therapeutic intervention against hemochromatosis and cancer by interrupting and preventing the enzymatic actions that create the systemic imbalance of cellular metals that allows elevated reactive oxygen species to oxidize and erode the protective DNA telomere caps.

 

The graphic on the left below represents the structure of TELOMIR-1. Fe2+ (iron) is embedded in the structure (orange) and is attracted by the nitrogen (blue) in each of the 5-member rings and 1 six-member ring.

 

The graphic on the right below represents is Fe2+ (iron) and the other molecules binding to the nitrogen (blue).

 

 

Source: InSilicoTrials; Insilico Affinity Computations Between TELOMIR-1 and DIEN to Fe2+ ions in Water Solution.

 

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This in silico study, conducted in the third quarter of 2023, aimed to investigate the affinity of TELOMIR-1 for Fe2+.The results of the affinity tests show that TELOMIR-1 has a higher affinity to Fe2+ in comparison to Diethylenetriamine (“DIEN”), but for 1 micromolar Fe2+ more than 10 micro molar of TELOMIR-1 is needed to form an effective complex. Additionally, Fe2+ is the bound form of iron in the human body; the majority of biological iron in the free state occurs in the Fe3+ (Ferric) state, and in the context of Hemochromatosis the excess iron is in the ferric form, hence the chelating agents currently used primarily target Fe3+. This was a computerized computation; therefore, serum was not used. Water is a complex substance that can mix with various substances, especially certain charged molecules including iron complexes, in many different ways. Currently, scientists have ways to study and understand these interactions, ranging from basic calculations to very detailed ones that look at tiny molecular details. To make sure these methods are accurate, scientists need a standard test to compare it with. Accordingly, we decided to use a database called NIST SRD 46 (A Critically Selected Stability Constants of Metal Complexes database) to get information about how well iron sticks to various molecules and find one that has similar binding properties to our drug, TELOMIR-1. We narrowed our search down to diethylenetriamine (DIEN). We picked DIEN with a pH of 6.33, or energy value of 36.11 kJ/mol, as our benchmark. This study was conducted with the assistance of InSilicoTrials, a third-party vendor.

 

Completed Pre-Clinical Toxicology Studies

 

The table below lists the type, species, purpose and results of our completed pre-clinical toxicology studies. Each of these studies began during the first quarter of 2023 and were completed during the third quarter of 2023. This study was conducted with the assistance of Frontage Laboratories, Inc., a third-party vendor.

 

Type of Study  

Species

(in vitro studies)

  Purpose   Results
Metabolite Identification   CD-1 mouse, SD-rat, Beagle dog, Cynomolgus monkey and human   To determine the metabolic pathways of TELOMIR-1 across species   TELOMIR-1 showed little/no metabolism by CYP enzymes.
TELOMIR-1 Reaction Phenotyping Using Liver S9   Mouse, rat, monkey and human liver   Identification of the Enzymes Involved in the Metabolism of TELOMIR-1 Using Hepatic S9 Fraction   The compound does not appear to be a substrate of cytochrome P450 enzymes. There was little/no metabolism of TELOMIR-1 in dog S9, consistent with the fact that the dog lacks aldehyde oxidase activity.
Cytochrome P450 (CYP) reaction phenotyping   Human   To evaluate the potential of TELOMIR-1 to be a victim of drug-drug interactions   TELOMIR-1 was extensively metabolized in mouse, rat, monkey, dog, and human hepatocytes.
Cytochrome P450 (CYP) inhibition   Human   To evaluate the potential of TELOMIR-1 to cause drug-drug interactions as a perpetrator   TELOMIR-1 (up to 100 µM) did not inhibit any of the tested CYP enzymes.
Plasma protein binding   CD-1 mouse, SD rat, Beagle dog, Cynomolgus monkey and human   To know the unbound fraction of TELOMIR-1 in plasma   Not highly Protein Bound
Maximum Tolerated Dose and 7 Day Repeat-Dose Toxicity/Toxicokinetic Study in Rats   Sprague Dawley Rats   To evaluate and characterize the toxicokinetic and toxicity of TELOMIR-1 and to estimate the MTD of TELOMIR-1 following   The MTD following 7 days of repeated administration of TELOMIR-1 at dose levels of 50, 200, and 750 mg/kg/day in rats was determined to be ≥ 750 mg/kg/day
Maximum Tolerated Dose and 7 Day Repeat-Dose Toxicity/Toxicokinetic Study in Dogs   Beagle Dogs   To evaluate and characterize the toxicokinetic and toxicity of TELOMIR-1 and to estimate the MTD of TELOMIR-   there were no treatment-related clinical observations, body weights, clinical pathology and anatomic gross pathology findings up to 7.5 mg/kg/day.
SafetyScreen44 TM   N/A   To evaluate, in Enzyme, and Radioligand Binding assays, the activity of TELOMIR-1   No significant results noted

 

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Ongoing and Planned Pre-Clinical Proof-of Concept Studies

 

The following is a description of the ongoing and planned pre-clinical proof-of-concept studies that we are conducting or anticipate conducting with respect to TELOMIR-1:

 

Efficacy of TELOMIR-1 in reducing ferritin in aged mice: A third-party vendor will complete a tolerability assessment, ferritin assay optimization, whole blood Methylation assay optimization, efficacy assessment with ferritin assay, and whole blood DNA methylation assay in 24-month-old mice, which have been shown to bioaccumulate hepatic ferritin compared with young mice. This was not powered for statistical significance.

 

The study began on August 22, 2023 and will progress over nine weeks as shown below. This study was conducted with the assistance of Frontage Laboratories, Inc., a third-party vendor.

 

 

Assay development to detect plasma concentration of rat and dog ferritin: The sample analysis for rat to measure concentrations of rat ferritin from rat plasma samples will be done by using ELISA assay (LS Bio commercial kit cat# LS-F36491). The sample analysis for dog to measure concentrations of dog ferritin from dog plasma samples will be done by using ELISA assay (LS Bio commercial kit cat# LS-F11494). This was not powered for statistical significance. This study started in June 2023; results are anticipated December 2023. This study is being conducted with the assistance of Evotec, a third-party vendor.

 

In Vitro stem cell therapy study: This study is expected to start in December 2023; Results are anticipated Q2 of 2024, which will consist of cell line selection, telomere length determination, cell proliferation/renewal determination, cell death determination, R&D cell bank.

 

a. Cell line selection: Based on the literature highlighting their appropriateness for telomere and aging research, a third-party collaborator will acquire three vials for each cell line: Primary human umbilical vein endothelial cell, non-immortalized human fibroblast, and the human BM-derived mesenchymal stem cell line.

 

b. Telomere length determination: A quantitative polymerase chain reaction (qPCR) kit will be employed to treat and assess the absolute telomere length of the cells. As part of the R&D analytical method development, assay performance will be determined to ensure the assay meets the linearity, selectivity, and precision attributes.

 

qPCR is the accumulation of amplification product is measured as the reaction progresses, in real time, with product quantification after each cycle.

 

c. Cell Proliferation/renewal determination: The cells will be exposed to different concentrations of TELOMIR-1 at varying time intervals. Multiple analytical attributes, including repeatability and linearity, will be evaluated to assess the experimental outcomes.

 

d. Cell death determination: Cellular apoptosis and necrosis are recognized as distinctive indicators of telomere shortening in stem cells and other cell types. The assay’s performance will be evaluated to ascertain repeatability and linearity criteria compliance.

 

e. R&D cell bank: It is of utmost importance to preserve the original cell vial to ensure consistent cell line behavior throughout the development of analytical assays and subsequent Good Manufacturing Practice (GMP) analytical testing. This approach aims to maintain the integrity and stability of the cell strain throughout the experimental procedures.

 

This study will not be powered for statistical significance. This study will be conducted with the assistance of Frontage Laboratories, Inc., a third-party vendor.

 

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Planned Pre-Clinical Toxicology Studies

 

The table below lists the type, species, and purpose, of additional planned pre-clinical toxicology designed to demonstrate that TELOMIR-1 will be not toxic before starting human clinical trials. Each of these studies are expected to begin during the fourth quarter of 2023 and be completed during the second quarter of 2024. These studies will be conducted with the assistance of third-party vendors.

 

Type of Study   Species   Purpose
In Vivo Micronucleus Study in Rats Using Bone Marrow   Sprague Dawley Rat   To assess the potential of TELOMIR-1 and/or its metabolites to induce micronuclei (MN) in bone marrow reticulocytes (RETs) of male and female Sprague Dawley
hERG (Manual Patch-Clamp)   N/A   To detect the risk of the protein product of the human-Ether-a-go-go -Related Gene (hERG) is an ion channel which plays an integral role in the repolarization and relaxation of the myocardium.
Cardiovascular Study Dog (Telemetry)   Beagle Dog   Cardiovascular analysis: ECG and Blood Pressure will be monitored continuously in conscious animals in their home cage for a minimum 1 hour of baseline (prior to dosing) and a minimum 24 hours after dosing.
Respiratory Study Rat   Sprague Dawley Rat   Plethysmography: Animals will be place in a whole-body plethysmograph and allowed to stabilize for a minimum of 1 hour prior to administration of the control or test article.
Neurobehavioral Evaluation Rats   Sprague Dawley Rat  

Functional observational battery will occur pre-dose and 1 time point (txmax) post-dose.

Motor activity conducted following functional observational battery using opto-varimax boxes.

28-Day Dog Study   Beagle Dog   to evaluate the toxicity and toxicokinetic profile following oral gavage administration of TELOMIR-1 to male and female Beagle dogs
28-Day Rat Study   Sprague Dawley Rat   The objectives of this study are to evaluate the toxicity and toxicokinetic profile following oral gavage administration of TELOMIR-1 to male and female Sprague Dawley rats.

 

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These are IND enabling studies with the purpose of demonstrating there are no animal toxicity issues before dosing in humans.

 

CMC Activities

 

The table below identifies our ongoing chemistry, manufacturing, and controls activities relating to TELOMIR-1. We started the analytical development and manufacturing of TELOMIR-1 in the first quarter of 2023. By the fourth quarter of 2023, we anticipate our suppliers will be developing TELOMIR-1 at scale and manufactured under cGMP conditions, expanding on earlier non-GMP volumes of TELOMIR-1 for use in our advanced testing programs. We plan to work closely with our suppliers to generate sufficient volumes of cGMP-grade TELOMIR-1 materials for the planned pre-clinical toxicity programs, expanded animal testing in human clinical trials expected to be performed in 2024, if the FDA approves our clinical trial design. This study is being conducted with the assistance of Frontage Laboratories, Inc., a third-party vendor.

 

    Chemistry, Manufacturing, and Controls
Status   Planned/In process Activity
Drug Substance Preparation  

● Analytical Development

 

○ Method Development

■Assay/impurity

■ Dissolution

○ Method Validation (cleaning method)

 

● Drug Excipient Compatibility

● Formulation Development and Prototypes

● Lead Formulation Stability

● Scale-up Demo Batch Manufacturing

● R&D Demo Batch Stability

● Method Validation

 

■Assay/impurity

■Dissolution

 

● GMP Manufacturing

● Release Testing

● GMP Stability Study

 

Our Clinical Development Plan

 

Regulatory Submissions

 

Following the completion of pre-clinical development program as described above, we plan to submit to the FDA an IND, focused on investigating TELOMIR-1 for the treatment of hemochromatosis. We may consider a second IND for post-chemotherapy recovery.

 

Our first IND application submission investigating TELOMIR-1 for the treatment of hemochromatosis is currently planned for the third quarter of 2024. If allowed to proceed by the FDA, a Phase I double-blind, randomized, placebo-controlled trial to evaluate the safety, tolerability, and pharmacokinetics of TELOMIR-1 in 40-60 healthy male and female adult subjects will be initiated approximately 30 days post-IND submission. We have contracted with Frontage Laboratories Phase I clinic in New Jersey consisting of 160 beds across three units for normal volunteers. After the Phase I trial is complete, a Phase II trial in the hemochromatosis population is planned to commence after results and guidance from the FDA.

 

Our second IND application will likely focus on investigating TELOMIR-1 for the treatment of post-chemotherapy recovery and is planned for submission with guidance from the FDA.

 

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Our clinical development plans will depend on FDA acceptance of our IND applications. As appropriate and pursuant to discussions with the FDA, we may periodically adjust the timeline for certain filings and associated clinical trials. It is important to note that the process for conducting clinical trials is uncertain and there is no assurance that our clinical development activities will meet the planned timelines set forth above.

 

Manufacture of Product for Clinical Development Activities

 

Anthem, a leading CDMO, has been developing a large-scale synthesis protocol for us and will be supplying quantities of TELOMIR-1 needed for our pre-clinical and clinical development activities. We are currently in discussions with Frontage to have TELOMIR-1 formulated into solid oral dosage forms for clinical trials.

 

Market Opportunity

 

TELOMIR-1, if approved, is expected to initially compete in the hemochromatosis market. TELOMIR-1, if approved, may have the potential to secondarily compete in the post-chemotherapy recovery market.

 

Disease Epidemiology – Patient Targets

 

The target patient pool for TELOMIR-1 will vary depending on which indication is under review. The initial target patient pool relates to hemochromatosis. Hemochromatosis is largely an inherited condition. The secondary category, post-treatment recovery, is a derivative of broader oncology epidemiology and treatment seeking behaviors.

 

Potential Market in Hemochromatosis

 

Patients with two mutated copies of the HFE gene (C282Y and H63D) are at the greatest risk for developing hemochromatosis, alongside patients with a family history of hemochromatosis. As a result, approximately 13 million U.S. patients carry mutations for hemochromatosis but only a fraction of them has symptoms, and not every symptomatic patient develops hemochromatosis. On average, about 750,000 US patients express one or more iron overload symptoms. There are 2 types of hemochromatosis, with the following patient mix: 150,000 primary hemochromatosis, and 65,000 secondary hemochromatosis confirmed diagnoses in the United States. Today, we estimate that over 150,000 patients have sought treatment since 2018, with most receiving phlebotomy, which is the withdrawal of blood to bring iron to normal levels. At present, 99% of hemochromatosis patients receive phlebotomy, with a portion of patients also receiving iron chelators as a co-treatment. Note that in the last 12 months, there have been 76,000 confirmed diagnoses of hereditary hemochromatosis and approximately 1% of hemochromatosis patients received deferasirox or deferoxamine off-label for 12 weeks (1 year for primary). The chart below outlines the epidemiological flow for hemochromatosis.

 

 

 

SOURCES

 

1. Bacon BR, Adams PC, Kowdley KV, Powell LW, Tavill AS; American Association for the Study of Liver Diseases. Diagnosis and management of hemochromatosis: 2011 practice guideline by the American Association for the Study of Liver Diseases. Hepatology. 2011;54(1):328-343. doi:10.1002/hep.24330

2. Gurrin, Lyle C., et al. “The natural history of serum iron indices for HFE C282Y homozygosity associated with hereditary hemochromatosis.” Gastroenterology 135.6 (2008): 1945-1952.

3. P360 Scripts and Claims Database, retrieved on October 3, 2022.

 

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Potential Market in Post-Chemotherapy

 

Patients undergoing chemotherapy irrespective of type of cancer are at risk. That said, older patients aged 65+ and above, have a higher risk of developing post-chemotherapy side effects such as digestive issues, sleep and memory issues, fatigue, and body aches. Other risk factors include the presence of metabolic conditions, dose levels of chemotherapy medication, blood count, and BMI.US CDC health data estimates that 650,000 patients receive chemotherapy annually in the US, with 45% experiencing severe side effects and 40% having more moderate side effects. Thus, ~158,000 patients were diagnosed with the adverse effects of chemotherapy (antineoplastic and immunosuppressive drugs. Today, Neulasta is given to patients undergoing chemotherapy as a “catch-all” therapeutic to help manage side effects. Neulasta is typically given to patients after chemotherapy for recovery. 95,000 patients with chemotherapy side effects got Neulasta over the last 12 months while 5,500 patients with immunosuppressive therapy side effects got Jakafi, also used for myelofibrosis and polycythemia vera. Given existing compliance levels around the use of Neulasta (~70%), we believe that adding TELOMIR-1 may boost overall compliance levels higher especially if levels of side effects are reduced.

 

 

SOURCES

 

1. Bacon BR, Adams PC, Kowdley KV, Powell LW, Tavill AS; American Association for the Study of Liver Diseases. Diagnosis and management of hemochromatosis: 2011 practice guideline by the American Association for the Study of Liver Diseases. Hepatology. 2011;54(1):328-343. doi:10.1002/hep.24330

2. Gurrin, Lyle C., et al. “The natural history of serum iron indices for HFE C282Y homozygosity associated with hereditary hemochromatosis.” Gastroenterology 135.6 (2008): 1945-1952.

3. P360 Scripts and Claims Database, retrieved on October 3, 2022.

 

Competition

 

We are subject to competition from pharmaceutical and biotechnology companies and academic and research institutions.

 

TELOMIR-1 will face competition in its initial target indication, hemochromatosis, as well as in its potential second target indication, post-chemotherapy recovery. Below we discuss the known competitors by target indication.

 

  Hemochromatosis: The current standard of care is phlebotomy, which typically costs $80 - $300/visit in community health facilities or offered for free at blood banks. Based on historical treatment data, phlebotomy serves more than 95% of hemochromatosis patients, as there are no FDA-approved indications. Each year, about 70,000 patients receive phlebotomy in healthcare facilities, excluding the blood banks (that provide the service for free). Patients who use it continue to embrace it given the lack of clinical options, its broad accessibility and low treatment cost. However, this option is not available to patients with hemophilia and other blood disorders. About 5% of patients are treated with iron chelators such as Exjade and Jadenu (and associated generics). This option is more expensive with pricing for Exjade at $532/day and Jadenu at $517/day. The last treatment option, deferasirox / deferoxamine was used with only 7,000 patients (2018-2022) based on IQVIA claims and scripts data. Future clinical options for treating hemochromatosis exist and remain limited. For example, 2 molecules are pre-clinical, 2 in Phase I, 1 in Phase II, and 1 is in Phase III.

 

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SOURCE

 

1. Citeline, Pharma Projects, and Trial Trove Database Results for “hemochromatosis”, retrieved on September 23, 2022.

 

  Post-Chemo Recovery: The current standard of care involves prescribing a small set of compounds designed to reduce patient discomfort from side effects. Filgrastim, pegfilgrastim, sargramostim are some examples of prescribed medication. These medications stimulate the growth of healthy white blood cells and granulocytes by subcutaneous injection. However, prices are high and can cost around $4,000-6,400 per dose of injection. Jakafi, a tyrosine kinase inhibitor is used in polycythemia vera and graft-versus-host disease. Pricing for Jakafi is $16,200 per bottle, with a $270 wholesale acquisition cost (WAC) unit price. Thus, given cost, only a portion of the high-risk patients are given the script; these are usually patients who are older than 65, and with a weakened immune system and low white blood cell count. Patients who use the class of medication do so to reduce post-treatment infection risk, even if they suffer from fevers and aches because of usage. The development pipeline of competing assets is robust but targeted at specific side effects such as iron deficiency or thrombotic constraints.

 

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SOURCE

 

1. Citeline, Pharma Projects, and Trial Trove Database Results for “hemochromatosis”, retrieved on September 23, 2022.

 

Intellectual Property

 

We license the U.S. patent rights for the use of TELOMIR-1 in human applications from MIRALOGX, an intellectual property development and holding company established by Jonnie R. Williams, Sr., the founder of our company and the sole inventor of TELOMIR-1. MIRALOGX is owned by the Bay Shore Trust, an irrevocable trust established by Mr. Williams. MIRALOGX filed a Patent Cooperation Treaty (PCT) application, PCT/US2023/073106 on August 29, 2023 for TELOMIR-1. The PCT application, which is titled, “ANTI-INFLAMMATORY COMPOUNDS, PHARMACEUTICAL COMPOSITIONS, AND METHODS OF TREATING HEMOCHROMATOSIS, AND OTHER DISORDERS,” covers, among other things, the TELOMIR-1 compound and methods of treating hemochromatosis, hyperammonemia, or a disorder associated with chronic inflammation or a cancer, by administering the compound to humans. The PCT application designated the U.S. and will enter U.S. national phase. The application, if granted and subject to payment of patent maintenance fees, would offer protection extending through at least August 29, 2043, in the U.S. The patent rights for TELOMIR-1 outside of the United States are not included in our current patent rights. Mr. Williams, our founder, does not hold any formal degrees in chemistry or certifications related to the development of pharmaceuticals; however, he developed extensive knowledge of tobacco alkaloid chemistry in the 1990’s through collaboration with industry experts during his development of techniques for preventing the formation of carcinogenic tobacco-specific nitrosamines (TSNAs) during the tobacco curing process. To better understand the mechanism by which TSNAs are formed, Mr. Williams collaborated closely with a leading expert in tobacco chemistry at the University of Kentucky, where he developed a working knowledge of tobacco alkaloid chemistry, which led to his development of patented curing technologies which would become the industry standard. This work led to him focusing on the therapeutic properties of tobacco alkaloids, and later to his design of novel small molecule therapeutics. He is a named inventor on 31 issued US patents and approximately 30 pending or published US patent applications, all of which are in the life sciences, a field that embraces the study of living organisms, including biology, botany, zoology, and biochemistry. Approximately 20 of his issued patents and 27 of his applications involve therapeutic treatment methods, and 4 issued patents and approximately 18 applications are directed to novel small molecules, principally involving alkaloid and cannabinoid chemistries. His work on TELOMIR-1 began in late 2020.

 

Our license from MIRALOGX is set forth in the Initial MIRALOGX License Agreement, as amended on November 10, 2023, by the Amendment No. 1 to the Amended and Restated License Agreement, pursuant to which the field of use relating to the license was amended to include therapeutic treatments and other medical or health uses in animals, in addition to humans, and related preclinical studies and activities conducted in furtherance of obtaining regulatory approval for and commercialization of veterinary, in addition to human, therapeutic treatments and uses (together with the “Initial MIRALOGX License Agreement, the “MIRALOGX License Agreement”). “Licensed Product” is defined as a drug product containing as an active agent 2,4,6-tris(3,4-dihydro-2H-pyrrol-2-yl)pyridine or a pharmaceutically acceptable salt, ester, or solvate thereof. We also have the right to grant corresponding sublicenses under the licensed patent rights. The MIRALOGX License Agreement provides for the payment to MIRALOGX of an 8% royalty (payable quarterly) on our net sales of Licensed Products by us or our sublicensees and on non-royalty bearing milestone revenue, with the royalty obligation ceasing upon the later of the expiration of the last-to-expire licensed patent or the expiration of the last strategic partnership/sublicensing agreement entered into by us. There are no up-front, execution, or milestone payments required under the license agreement. Further, no payments have been made to date under the agreement. The agreement provides for a perpetual license and does not contain a provision that permits MIRALOGX to terminate the agreement. The MIRALOGX License Agreement provides that MIRALOGX will have sole control over the filing, prosecution, maintenance, and management of the licensed patent rights, provided that we will be responsible for the cost of prosecuting, maintaining, and managing the licensed patents. The agreement grants to us the primary right, but not the obligation, to enforce the licensed patent rights.

 

Besides relying on patents, we will also rely on trade secrets, proprietary know-how and continuing innovation to develop and maintain our competitive position, especially when we do not believe that patent protection is appropriate or can be obtained. We will seek protection of these trade secrets, proprietary know-how and any continuing innovation, in part, through confidentiality and proprietary information agreements. However, these agreements may not provide meaningful protection for, or adequate remedies to protect, our technology in the event of unauthorized use or disclosure of information. Furthermore, our trade secrets may otherwise become known to, or be independently developed by, our competitors. We may in the future seek appropriate patent protection for technology in our research and development programs to the extent not licensed from MIRALOGX or another third party, where applicable, and their uses by filing patent applications in the appropriate jurisdiction. We intend for these patent applications to cover, where possible, claims for compositions of matter, medical uses, processes for preparation and formulations.

 

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We currently use TELOMIR and TELOMIR-1 as unregistered trademarks, and we intend to rely on such trademarks and trade names and service marks, as well as our domain names and logos, as appropriate, to distinguish, build, and market our brand.

 

Regulation

 

The U.S. Food and Drug Administration (FDA) and comparable regulatory authorities in state and local jurisdictions impose substantial and burdensome requirements upon companies involved in the clinical development, manufacture, marketing, and distribution of drugs. These agencies and other federal, state, and local entities regulate, among other things, the research and development, testing, manufacture, quality control, safety, effectiveness, labeling, storage, record keeping, approval, advertising and promotion, distribution, post-approval monitoring and reporting, sampling and export and import of our drug candidates.

 

U.S. Government Regulation

 

In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, or FDCA, and its implementing regulations. The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local and foreign statutes and regulations requires the expenditure of substantial time and financial resources. Failure to comply with the applicable U.S. requirements at any time during the product development process, approval process or after approval, may subject an applicant to a variety of administrative or judicial sanctions, such as the FDA’s refusal to approve pending New Drug Applications (NDAs), withdrawal of an approval, imposition of a clinical hold, issuance of warning letters, product recalls, product seizures, total or partial suspension of production or distribution, injunctions, fines, refusals of government contracts, restitution, disgorgement or civil or criminal penalties.

 

The process required by the FDA before a drug may be marketed in the United States generally involves the following:

 

  completion of pre-clinical laboratory tests, animal studies and formulation studies in compliance with the FDA’s good laboratory practice (“GLP”) regulations;
     
  submission to the FDA of an Investigational New Drug (“IND”) application, which must become effective before human clinical trials may begin;
     
  approval by an independent Institutional Review Board (“IRB”), at each clinical site before each trial may be initiated;
     
  performance of adequate and well-controlled human clinical trials in accordance with good clinical practices (“GCP”) requirements to establish the safety and efficacy of the proposed drug product for each indication;
     
  demonstration that the API and finished product are manufactured under cGMP conditions and meet all applicable standards of identity, strength, quality, and purity;
     
  submission to the FDA of an NDA;
     
  satisfactory completion of an FDA advisory committee review, if applicable;

 

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  satisfactory completion of an FDA inspection of the manufacturing facility or facilities at which the product is produced to assess compliance with cGMP requirements and to assure that the facilities, methods, and controls are adequate to preserve the drug’s identity, strength, quality, and purity;
     
  FDA review and approval of the NDA, including consideration of the views of any FDA advisory committee, prior to commercial marketing or sale of the drug in the United States; and
     
  compliance with any post-approval requirements, including the potential requirement to implement a Risk Evaluation and Mitigation Strategy (“REMS”) or to conduct a post-approval study.

 

Pre-clinical studies

 

Before testing any drug or biological product candidate in humans, the product candidate must undergo rigorous pre-clinical testing. The pre-clinical developmental stage generally involves laboratory evaluations of drug chemistry, formulation, and stability, as well as studies to evaluate toxicity in animals, to assess the potential for adverse events (“AEs”) and, in some cases, to establish a rationale for therapeutic use. The conduct of pre-clinical studies is subject to federal regulations and requirements, including GLP regulations for safety/toxicology studies. An IND sponsor must submit the results of the pre-clinical studies, together with manufacturing information, analytical data, any available clinical data or literature and a proposed clinical protocol, to the FDA as part of the IND.

 

An IND is a request for authorization from the FDA to ship an investigation product and then administer it to humans and must be allowed to proceed by the FDA before human clinical trials may begin. Some long-term pre-clinical testing, such as animal tests of reproductive AEs and carcinogenicity, may continue after the IND is submitted. An IND automatically becomes effective 30 days after receipt by the FDA, unless the FDA raises concerns or questions before that time related to one or more proposed clinical trials and places the trial on clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns before the clinical trial can begin. As a result, submission of an IND may not result in the FDA allowing clinical trials to commence.

 

Clinical trials

 

The clinical stage of development involves the administration of the investigational product to healthy volunteers or patients under the supervision of qualified investigators, generally physicians not employed by, or under control of, the trial sponsor, in accordance with GCPs, which include the requirement that all research patients provide their informed consent for their participation in any clinical trial. Clinical trials are conducted under protocols detailing, among other things, the objectives of the clinical trial, dosing procedures, subject selection and exclusion criteria and the parameters to be used to monitor subject safety and assess efficacy. Each protocol, and any subsequent amendments to the protocol, must be submitted to the FDA as part of the IND. Furthermore, each clinical trial must be reviewed and approved by an IRB for each institution at which the clinical trial will be conducted to ensure that the risks to individuals participating in the clinical trials are minimized and are reasonable in relation to anticipated benefits. The IRB also approves the informed consent form that must be provided to each clinical trial subject or his or her legal representative and must monitor the clinical trial until completed. There also are requirements governi