EX-99.1 2 cerecorcorporatehighligh.htm EXHIBIT 99.1 cerecorcorporatehighligh
Exhibit 99.1 Cerecor Corporate Highlights March | 2019


 
Forward-Looking Statements This presentation may include forward-looking statements made pursuant to the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts. Such forward-looking statements are subject to significant risks and uncertainties that are subject to change based on various factors (many of which are beyond Cerecor’s control), which could cause actual results to differ from the forward-looking statements. Such statements may include, without limitation, statements with respect to Cerecor’s plans, objectives, projections, expectations and intentions and other statements identified by words such as “projects,” “may,” “will,” “could,” “would,” “should,” “continue,” “seeks,” “aims,” “predicts,” “believes,” “expects,” “anticipates,” “estimates,” “intends,” “plans,” “potential,” or similar expressions (including their use in the negative), or by discussions of future matters such as: our 2018 outlook; the development of product candidates or products; potential attributes and benefits of product candidates; the expansion of Cerecor’s drug portfolio, Cerecor’s ability to identify new indications for its current portfolio; and new product candidates that could be in-licensed, and other statements that are not historical. These statements are based upon the current beliefs and expectations of Cerecor’s management but are subject to significant risks and uncertainties, including: risks associated with acquisitions, including the need to quickly and successfully integrate acquired assets and personnel; Cerecor’s cash position and the potential need for it to raise additional capital; reliance on key personnel, including Mr. Greenleaf; drug development costs and timing (including government shutdowns); and those other risks detailed in Cerecor’s filings with the Securities and Exchange Commission. Actual results may differ from those set forth in the forward-looking statements. Except as required by applicable law, Cerecor expressly disclaims any obligations or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Cerecor’s expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based. 2|


 
Cerecor is an integrated biopharmaceutical company developing innovative therapies at the cutting edge of science. Our pipeline filled with forward-thinking ideas propels us forward. We are driven to change the lives of patients with rare orphan diseases in pediatrics and neurology. Driven by Science | Inspired by Hope


 
Cerecor Today Focused on Advancing R&D, while Optimizing Commercial Capabilities • Emerging clinical & early-stage pipeline Innovative • Three 505(b)(2) programs with expedited path to NDA Pipeline • Focus on orphan, neurological & pediatric diseases Commercial • Building a commercial capability Footprint • Pediatric franchise generating positive cash flow Transforming • Fully-integrated commercial and R&D organization Cerecor • New management team with proven track record 4|


 
Overview 1 Management Team 2 Historical Milestones 3 Neurology & Pediatric Rare Disease Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 5|


 
Management Team Extensive experience in development and commercialization 20+ years industry experience 25+ years industry experience • Chairman and CEO, Sucampo • President and COO of Zylera Pharmaceuticals Matthew V. Peter S. Pharmaceuticals • Executive Director, Victory Pharma Phillips Greenleaf • CEO, Histogenics Corporation • Director, Eisai Co, Ltd. • President, MedImmune Ventures Chief President & • Account Manager, Dura Pharmaceuticals, Inc. • Commercial CEO Manager, Centocor Biotech (Johnson & Johnson) Officer 20+ years 25+ years industry experience • Vice President of Finance, Sucampo • Sr. Vice President | Principal The NSCI Group Joseph James A. Pharmaceuticals Harrell • General Manager Specialty Pharmaceuticals, Miller • Senior Director of Accounting, Qiagen Covidien EVP Chief • Chief Financial Officer, Eppendorf 5Prime • Vice President Marketing Pediatric Infectious Marketing, Financial • Disease, MedImmune Certified Public Accountant Investor Officer • Sr. Director Marketing IMIDs, Centocor a J&J Relations Company • Hospital Specialist, ATOD Rhone Poulenc Rorer 20+ years industry experience 8+ years industry experience • V.P. Global CMC & Development, Sucampo • Chairman, President at Ichorion Therapeutics Dr. Pericles Patrick Calias Pharmaceuticals • SVP, Business Development at Vyera • CSO, Pharming Group Crutcher Pharmaceuticals Chief • Sr. Director Rare CNS Diseases and Device VP • BD Analyst at Retrophin Scientific Lead, Shire plc Business • Officer & Head MSc, CPhil in Statistics, UCLA • Sr. Director Drug Delivery and Chemistry, Development of R&D Eyetech Pharmaceuticals • Ph.D., Tufts University, Bioorganic Chemistry 6|


 
Historical Milestones Management Change 2013 2014 2015 2016 2017 2018 CERC-301 Cerecor IPO Acquisition Acquisition CERC-406 October 15, 2015 TRx Pharma Avadel (AVDL) (Zylera) Pediatric assets Acquired worldwide rights of two NMDA receptors from Merck CERC-501 CERC-611 CERC-501 Acquisition Acquired worldwide Acquired worldwide Sold to Janssen Ichorion rights from Eli Lilly rights from Eli Lilly Rare Disease Pipeline CERC-301 Enters Phase 1 study for nOH 7|


 
Cerecor Evolution Neurological Disorders Pediatric Franchise Pediatric Rare Diseases Innovative Approaches FDA-Approved 505(b)(2) Assets & to CNS Diseases Products Platform Chemistry • CERC-301 • Poly-Vi-Flor̺ | Tri-Vi-Flor̺ • CERC-801 • CERC-406 • Karbinalဆ ER • CERC-802 • CERC-501 • AcipHex̺ Sprinkleဆ • CERC-803 • CERC-611 • Cefaclor • CERC-913 • Flexichamberဆ • Millipred̺ | Veripred̺ • Ulesfia̺ In-Licensed Pipeline Robust Orphan Rare Capability & Cash Flow CNS Assets Disease Pipeline 8|


 
Emerging Clinical & Early-Stage Pipeline Mechanism of Program Target Indication Development Stage Action D-Galactose CERC-801 PGM1 Deficiency replacement Phase 1 505(b)(2) D-Mannose CERC-802 MPI Deficiency replacement IND-Enabling 505(b)(2) L-Fucose CERC-803 CDG-IIc replacement IND-Enabling 505(b)(2) Pediatric Division Nucleoside IND- CERC-913 DGUOK Deficiency replacement Enabling GluN2B selective, Neurogenic CERC-301 NMDA Receptor Orthostatic Phase 1 POC antagonist Hypotension CNS-targeted, COMT CERC-406 Parkinson’s Disease inhibitor (2nd Gen) IND-Enabling TARP-ɀ8 dependent Partial Onset CERC-611 AMPA Receptor Phase 1 Ready Neurology Division Seizures antagonist Denotes Pediatric Program and Expedited 505(b)(2) Approval Pathway Denotes Pediatric Program Denotes Neurology Program 9|


 
Market Potential World-Wide Estimated Patient Populations Develop Internally & Early Clinical (Ph1/2) Commercialize Independently Proof-of-Concept with Optionality 40M Larger CNS Indications 1.9M Ultra Rare Pediatric Orphan Diseases 700k 500 500 250 50 CERC-801 CERC-802 CERC-803 CERC-913 CERC-301 CERC-406 CERC-611 PGM1 MPI CDG-IIc DGUOK nOH* Parkinson’s Epilepsy Deficiency Deficiency Deficiency Disease 10| *Orphan Disease Designation Eligibility with < 200,000 Patients Estimated in the U.S.


 
R&D Milestones Multiple value generating inflection points over next 12 to 18 months Program Target Indication Upcoming Milestone CERC-801* PGM1 Deficiency Phase I Data 1H19 CERC-802* MPI Deficiency IND Filing 1H19 CERC-803* CDG-IIc IND Filing 2020 Disorders Metabolic CERC-913 DGUOK Deficiency IND Filing 2020 CERC-301 Neurogenic Orthostatic Hypotension Phase I Data 1H19 CERC-406 Parkinson’s Disease IND Filing 1H20 Disorders Neurology Neurology CERC-611 Partial Onset Seizures Under Strategic Review 11| *505(b)(2) Pathway


 
Congenital Disorders of Glycosylation (CDGs) Orphan diseases with an estimated prevalence of 1 in 100,000 • Genetic diseases that result in impaired glycoprotein production and function • Glycoproteins are critical for cell structure and function, particularly for circulating proteins and enzymes such as hormones and coagulation factors • Improper sugar architecture diminishes and/or disrupts protein function • Patients typically have multi-organ disfunction, some with nervous system involvement, including: structural abnormalities, myopathies, coagulopathies, hypoglycemic episodes, epileptic seizures and developmental delay • High morbidity and mortality diseases with approximately 25% childhood mortality 12|


 
CERC-800s Substrate replacement therapies for CDGs Monogenic disorders resulting in glycosylation defects with broad clinical spectrum, including life-threatening complications Multi-system disease manifestation in PGM1 Deficiency CERC-801 D-Galactose leads to significant improvement in key clinical symptoms Life-threatening gastrointestinal disorder in MPI Deficiency CERC-802 D-Mannose rapidly resolves hematological & intestinal abnormalities Immunodeficiency with CNS impairment in CDG-IIc CERC-803 L-Fucose normalizes cell counts & reduces infection risk Ultra-orphan IEMs with serious and life-threatening medical needs • <500 patients WW per indication • High pediatric morbidity & mortality No approved treatments 13|


 
CERC-800s Substrate replacement therapies for CDGs Oral, small molecule, naturally occurring monosaccharides used as standards-of-care for CDGs OH • Safe HO O HO O • Efficacious HO OH HO OH • Rapid Onset of Action OH OH D-GalactoseD-Mannose L-Fucose Eligibility CERC-801 CERC-802 CERC-803 ض ض ض b)(2) NDA Pathway)505 ض ض ض NCE 5-yrs Exclusivity ض ض ض ODD 7-yrs Exclusivity ض ض ض Priority Review Voucher ض ض ض EMA ODD 10-yrs Exclusivity 14|


 
CERC-800s Substrate replacement therapies for CDGs Retrospective chart reviews & registry data have been successfully used to minimize or obviate prospective clinical studies Developer Therapeutic (Indication) Pivotal Study Strategy Retrospective case series summary (13/23 Carbaglu patients with complete data) & 3 patients (NAGS Deficiency) treated prospectively (2010) Case report form from retrospective chart Cholbam review of patients in open-label, single-arm (Bile Acid Disorders) Expanded Access Protocol (2015) Retrospective case reports from a multicenter ProVay Blue chart review in addition to cases found in (Acquired Methemoglobinemia) published literature (2016) Expanded label (from 10 mutations to 33) based Kalydeco on registry data & mechanistic information (Cystic Fibrosis) from lab studies (2017) Reference to survival data from an international Lumizyme registry of infantile-onset disease (Pompe Disease) demonstrating mortality benefit (2010) CDG Connect Patient Insights Network (PIN) https://connect.invitae.com/org/cdg 15|


 
CERC-913 ProTide Nucleotide for Deoxyguanosine Kinase (DGUOK) Deficiency Overcome key limitations of direct substrate replacement: Stability, Permeability & Kinase Bypass Phenotypic Rescue CERC-913 Attributes • Proof-of-concept in patient-derived & animal-based disease models • ProTide similar to advanced clinical candidates & approved drugs • Metabolite ID & PK profile in dog support translational PKPD 16|


 
CERC-301 NR2B selective NMDA receptor antagonist for nOH Autonomic nervous system fails to regulate vasoconstriction due to underlying neurological disease • Rapid and significant (20mm Hg) drop in blood pressure caused by postural change • Increased risk of falls or injury, leading 1 in 5 to decreased quality of life • Estimated less than 200,000 patients Parkinson’s Patients in the U.S. (ODD eligible) Experience nOH • Single FDA-Approved therapy, Northera̺ (droxidopa) – Effectiveness beyond 2 weeks of treatment has not been established – Significant proportion of non-responders (>1/3 in pivotal study) – Estimated 2018 revs ~$270mm 17|


 
CERC-301 NR2B selective NMDA receptor antagonist for nOH Initiated Phase 1 study of CERC-301 in Parkinson’s patients diagnosed with nOH CERC-301 Clinical Database • Established safety profile in ~400 patients and healthy volunteers • Multiple studies demonstrate rapid, durable & dose-dependent increase in Systolic Blood Pressure (SBP) Time (h) 18|


 
CERC-301 NR2B selective NMDA receptor antagonist for nOH Opportunity to rapidly demonstrate proof-of-concept in patients Phase 1 SAD in PD patients with nOH Visit 1 Visit 2 Visit 3 Visit 4 Visit 5a Arm 1 Enrollment • 12 active centers in US pbo 8 mg 12 mg 16 mg 20 mg (n = 5) • N = 20 (8, 12, 16 & 20 mg) Arm 2 • Double-blind, randomized, 8 mg pbo 12 mg 16 mg 20 mg Design (n = 5) pbo-controlled • Interim Analysis at 10 patients Arm 3 8 mg 12 mg pbo 16 mg 20 mg (n = 5) • Safety, Tolerability & PK Endpoints • BP measurement Arm 4 8 mg 12 mg 16 mg pbo 20 mg • Symptomatic assessment (n = 5) • 5 visits (7 to 10 days apart), 4 single escalating doses of CERC-301 or placebo • Assess safety, tolerability & PK • Measure BP effects during orthostatic challenge(s) • Symptomatic assessment (OHSA Item #1) at each visit 19| a Randomized 4:1


 
CERC-406 COMT inhibitor for Parkinson’s Disease Next generation, CNS-penetrant COMT inhibitor to enhance efficacy and minimize toxicity seen with 1st generation therapies • As PD progresses the therapeutic window of L-Dopa becomes smaller, increasing the on/off periods Extended ‘on’ Time A Typical Day (‘on’ time) (‘on’ Controlled Symptoms Adequately Medication Starts to Work Time PD PD PD Medication Medication Medication (‘off’ time) Controlled Adequately levodopa/dopa-decarboxylase inhibition plus COMT inhibition Symptoms Not Symptoms levodopa/dopa-decarboxylase inhibition COMT inhibitors are taken with levodopa to reduce off-time and increase on-time without dyskinesia 20| Figure Source: European Parkinson’s Disease Association (EPDA)


 
CERC-406 COMT inhibitor for Parkinson’s Disease COMT inhibition provides an in vivo biomarker for target engagement by measuring DOPAC and HVA • COMT regulates dopamine (levodopa) breakdown via the conversion of dihydroxyphenyl-acetic acid (DOPAC) to homovanillic acid (HVA) 1st Gen COMT Drawbacks / CSF Biomarkers: Inhibitors Marketing Status Single, 100 mg/kg CERC-406 dose in rats black box warning Tolcapone generic peripherally-restricted Entacapone generic peripherally-restricted Opicapone NDA 1H19* 21| *Projected


 
CERC-611 TARP-ɣ8 dependent AMPA receptor antagonist for partial onset seizures Phase 1-ready candidate with therapeutic potential for partial onset seizures in patients with epilepsy Significant Unmet Need Unique Mechanism of Action • Epilepsy affects over 65 million • AMPA receptors mediate fast patients worldwide synaptic neurotransmission within the CNS and are a proven target for • 30%-40% of patients refractory; anti-seizure efficacy high degree of poly-pharmacy common • CERC-611 is the first known AMPA receptor antagonist that selectively • All anti-seizure drugs have side targets the hippocampus effects (e.g. motoric) limiting use and the timely achievement of • CERC-611 shows lack of motoric therapeutic dose levels impairment at efficacious exposures in animal models of epilepsy Ongoing discussions with the FDA regarding appropriate dosing regimen 22|


 
Overview 1 Management Team 2 Historical Milestones 3 Neurology & Pediatric Rare Disease Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 23|


 
Building Commercial Capabilities in Pediatrics Eight commercial products promoted across 42 U.S. Territories Fully-integrated commercial team with revenues reinvested into operations and R&D Capability-Building: Sales, Operations, Regulatory, Marketing and Training 24|


 
Why Pediatrics? Pediatrics Represents a Focused, Defined and Specific Patient Population Treated by One Specialty Segment Our Existing Pediatric Product Portfolio May be Used in Up To 75% of the Top 25 Pediatric Diagnosis Codes Top 25 Pediatric Codes 2013 AAP Pediatric Coding Newsletter1 1. Routine Child Health Examination 9. Dermatitis 17. Influenza with Respiratory Manif. 2. Acute Upper Respiratory Infection 10. Attention-Deficit/ 18. Gastroenteritis / Colitis Hyperactivity Disorder 3. Otitis Media 19. Fever 11. Cough 4. Acute Pharyngitis 20. Constipation 12. Viral Infection 5. Asthma 21. Vaccination 13. Streptococcal Sore Throat 6. Follow-up Exam 22. Abdominal Pain 14. Bronchitis 7. Allergic Rhinitis 23. Viral Diseases 15. Conjunctivitis 8. Sinusitis 24. Pneumonia 16. Esophageal Reflux ICD-10-CM codes are displayed as 24 code categories that include the 25 diagnoses from the International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM) list (2 otitis media codes were included in ICD-9-CM). 25| 1. AAP pediatric coding newsletter coding.aap.org August 2013


 
Net Product Sales Net Product Sales Last 4 QTR Trend ($ Millions) $6 $5 $4 $3 $2 $1 $0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 FY19 Net Sales Guidance = $20 to $22 Million 26|


 
Balance Sheet Cash Trend Analysis ($ Millions) 20,000 17,500 15,000 12,500 10,000 7,500 5,000 2,500 0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Mar 10 2019* Total Assets Trend Analysis ($ Millions) 80,000 70,000 60,000 50,000 40,000 30,000 20,000 10,000 0 Q1 2018 Q2 2018 Q3 2018 Q4 2018 Mar 10 2019* 27| *Estimated balances following recently completed offering


 
2019 Growth Plans 1 2 3 Accelerate Business Advance Build Commercial Development Pipeline Excellence Activity CERC-301 Grow Market Share Acquire/in-license commercial-ready or CERC-406 Expand Commercial marketed asset(s) Footprint CERC-611 Acquire/in-license CERC-801 complimentary pipeline assets CERC-802 CERC-803 CERC-913 28|


 
Driven by Science NASDAQ:CERC www.cerecor.com Inspired by Hope