8-K

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported) December 23, 2019

CERECOR INC.

(Exact name of registrant as specified in its charter)

		Delaware
	(State or other jurisdiction of incorporation)
001-37590			45-0705648
(Commission File Number)			(IRS Employer Identification No.)

540 Gaither Road, Suite 400, Rockville, Maryland 20850

(Address of principal executive offices) (Zip Code)

Registrant’s Telephone Number, Including Area Code: (410) 522-8707

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

ý Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

¨ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

¨ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

¨ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, $0.001 Par Value	CERC	Nasdaq Capital Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging Growth Company ý

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 13(a) of the Exchange Act. ý

Item 8.01.Other Events.

On December 23, 2019, Cerecor Inc. (“Cerecor”) posted an updated Investor Presentation regarding the business of the combined company after completion of the previously announced proposed merger with Aevi Genomic Medicine, Inc. (“Aevi”) to its website at www.cerecor.com. A copy of the investor presentation is attached hereto as Exhibit 99.1.

Additional Information about the Merger and Where to Find It

This document does not constitute an offer to sell or the solicitation of an offer to buy any securities of Aevi or Cerecor or the solicitation of any vote or approval. In connection with the proposed Merger, on December 20, 2019, Cerecor filed with the SEC a Registration Statement on Form S-4 containing a proxy statement/prospectus. The proxy statement/prospectus contains important information about Aevi, Cerecor, the Merger and related matters. Aevi will mail or otherwise deliver the proxy statement/prospectus to its stockholders when it becomes available. Investors and security holders of Aevi and Cerecor are urged to read carefully the proxy statement/prospectus relating to the Merger (including any amendments or supplements thereto) in its entirety, because it contains important information about the proposed Merger.

Investors and security holders of Aevi and Cerecor will be able to obtain free copies of the proxy statement/prospectus for the proposed Merger (when it is available) and other documents filed with the SEC by Aevi and Cerecor through the website maintained by the SEC at www.sec.gov. In addition, investors and security holders of Aevi will be able to obtain free copies of the proxy statement/prospectus for the proposed Merger (when it is available) by contacting Aevi, Attn: Mike McInaw, michael.mcinaw@aevigenomics.com. Investors and security holders of Cerecor will be able to obtain free copies of the proxy statement/prospectus for the merger by contacting Cerecor, Attn: James Harrell, jharrell@cerecor.com.

Participants in the Merger

Aevi, Cerecor and certain of their respective directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of Aevi in respect of the transactions contemplated by the Merger Agreement between Aevi and Cerecor. Information regarding Aevi’s directors and executive officers is contained in Aevi’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018, which was filed with the SEC on March 29, 2019, and is available in the proxy statement/prospectus that was filed by Cerecor with the SEC in connection with the proposed Merger. Information regarding Cerecor’s directors and executive officers is contained in Cerecor’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018, which was filed with the SEC on March 18, 2019, and in the proxy statement/prospectus that was filed by Cerecor with the SEC in connection with the proposed Merger.

Cautionary Statement Regarding Forward-Looking Statements

This document contains forward-looking statements within the meaning of Section 27A of the Securities Act, Section 21E of the Exchange Act and as that term is defined in the Private Securities Litigation Reform Act of 1995, including, but not limited to, Aevi’s and Cerecor’s expectations or predictions of future financial or business performance or conditions. Forward-looking statements are sometimes identified by their use of the terms and phrases such as “estimate,” “project,” “intend,” “forecast,” “anticipate,” “plan,” “planning, “expect,” “believe,” “will,” “will likely,” “should,” “could,” “would,” “may” or the negative of such terms and other comparable terminology. These forward-looking statements are subject to numerous assumptions, risks and uncertainties, which change over time, are difficult to predict and are generally beyond the control of either company. Actual results may differ materially from current projections.

Important factors that may cause actual results to differ materially from the results discussed in the forward-looking statements or historical experience include risks and uncertainties, including the timing and completion of the Merger, the parties’ ability to satisfy the closing conditions of the Merger Agreement, the failure by Aevi or Cerecor to secure and maintain relationships with collaborators and/or investors; risks relating to clinical trials; risks relating to the commercialization, if any, of Aevi’s or Cerecor’s proposed product candidates (such as

1

marketing, regulatory, product liability, supply, competition, and other risks); dependence on the efforts of third parties; dependence on intellectual property; and risks that Aevi or Cerecor may lack the financial resources and access to capital to fund proposed operations. Further information on the factors and risks that could affect Aevi’s and Cerecor’s respective businesses, financial conditions and results of operations are contained in Aevi’s and Cerecor’s filings with the SEC, which are available at www.sec.gov. The forward-looking statements represent Aevi’s and Cerecor’s estimate as of the date hereof only, and Aevi and Cerecor specifically disclaim any duty or obligation to update forward-looking statements.

Item 9.01.     Financial Statements and Exhibits.

(d)    Exhibits.

Exhibit No.	Description
99.1	Investor Presentation

2

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	CERECOR INC.
Date: December 23, 2019	/s/ Joseph M. Miller
	Joseph M. Miller
	Chief Financial Officer

3

EX-99.1

Exhibit 99.1 Driven by Science Inspired by Hope Establishing a leading, rare disease-focused biopharmaceutical company to deliver impactful new medicines to patients December | 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, Inc. (‘Cerecor”) or Aevi Genomic Medicine, Inc.’s (“Aevi”) 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: Aevi and Cerecor’s respective 2019 outlook; the development of product candidates; timing and success of trial results and regulatory reviews; potential attributes and benefits of product candidates; the expansion of Cerecor’s drug portfolio; the benefits of the proposed merger with Aevi described herein; and other statements that are not historical. These statements are based upon the current beliefs and expectations but are subject to significant risks and uncertainties, including: drug development costs, timing and other risks; the companies’ cash position and the need to raise additional capital; risks associated with dispositions and acquisitions, such as the pediatric asset sale and the proposed merger with Aevi, including their consumption of company resources to complete, risks they might not close, and the need to quickly and successfully reorganize operations and integrate acquired assets and personnel if they do; reliance on and the need to attract, integrate and retain key personnel; and those other risks detailed in Cerecor’s and Aevi’s respective 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 and Aevi expressly disclaim 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 or Aevi’s expectations with respect thereto or any change in events, conditions or circumstances on which any statement is based. 2|

Additional Information Concerning the Merger Additional Information about the Merger and Where to Find It In connection with the proposed merger with Aevi, Cerecor will file with the SEC a Registration Statement on Form S-4 containing a proxy statement/prospectus. The proxy statement/prospectus will contain important information about Aevi, Cerecor, the merger and related matters. Aevi will mail or otherwise deliver the proxy statement/prospectus to its stockholders when it becomes available. Investors and security holders of Aevi and Cerecor are urged to read carefully the proxy statement/prospectus relating to the merger (including any amendments or supplements thereto) in its entirety when it is available, because it will contain important information about the proposed merger. Investors and security holders of Aevi and Cerecor will be able to obtain free copies of the proxy statement/prospectus for the proposed merger (when it is available) and other documents filed with the SEC by Aevi and Cerecor through the website maintained by the SEC at www.sec.gov. In addition, investors and security holders of Aevi will be able to obtain free copies of the proxy statement/prospectus for the proposed merger (when it is available) by contacting Aevi, Attn: Mike McInaw, michael.mcinaw@aevigenomics.com. Investors and security holders of Cerecor will be able to obtain free copies of the proxy statement/prospectus for the merger by contacting Cerecor, Attn: James Harrell, jharrell@cerecor.com. Participants in the Merger Aevi, Cerecor and certain of their respective directors and executive officers may be deemed to be participants in the solicitation of proxies from the stockholders of Aevi in respect of the transactions contemplated by the Merger Agreement between Aevi and Cerecor. Information regarding Aevi’s directors and executive officers is contained in Aevi’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018, which was filed with the SEC on March 29, 2019, and will also be available in the proxy statement/prospectus that will be filed by Cerecor with the SEC in connection with the proposed merger. Information regarding Cerecor’s directors and executive officers is contained in Cerecor’s Annual Report on Form 10-K for the fiscal year ended December 31, 2018, which was filed with the SEC on March 18, 2019, and will also be available in the proxy statement/prospectus that will be filed by Cerecor with the SEC in connection with the proposed merger. Non-Solicitation This presentation shall not constitute an offer to sell or the solicitation of an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such jurisdiction. No offer of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the Securities Act, as amended. 3|

Cerecor Continues Transformation Building a rare disease biotech company committed to developing innovative medicines in areas of high unmet need Advancing CERC-800s to NDA filings and FDA approvals over the next 18 to 24 months; potential PRVs could provide non-dilutive capital for R&D investment Emphasizing R&D with recent sale of pediatric portfolio, narrowing focus and improving the balance sheet; additional optionality with Millipred® Strategically positioning the company as a leader in orphan disease drug development to deliver high impact medicines to underserved patient populations Identified AEVI Genomic Medicine, Inc. (NASDAQ: GNMX) as a potential target for M&A due to complimentary fit of rare disease pipeline assets and management personnel Advancing CNS pipeline to value inflection for strategic optionality as partnered or out- licensed assets 4|

Cerecor (CERC) & Aevi (GNMX) Have Entered Into a Merger Agreement to Merge Propose to form a leading biopharmaceutical company focused on pediatric orphan diseases Rare disease programs establish robust pipeline with 6 addition of novel, first-in-class medicines + PRV-eligible, fast-to-market programs with anticipated 4 POC data in 2020, first NDA approval targeted for 2021 + Expansion of leadership team to include 2 CEO Michael Cola and CMO Garry Neil = Leading biopharmaceutical company with 1 a world-class orphan disease pipeline 5|

Combined Company Well-Positioned to Execute on Delivering Innovative New Medicines to Patients CERC-800s AEVI-007 AEVI-006 • Substrate replacement • Anti-IL-18 mAb for • mTORC1/2 inhibitor for therapies for CDGs auto-inflammatory diseases Lymphatic Malformations • 2 pivotal-ready programs • Phase 2-ready • Phase 2-ready with FTD • Strong biological rationale • Existing clinical proof-of- • All 3 programs received evidenced by serum concept with off-label use RPDD and ODD biomarkers of sirolimus • First NDA filing anticipated • First NDA filing anticipated • First NDA filing anticipated in 2021 in 2022 in 2023 • Estimated collective TAM of • Estimated $400 – 500M PYS • Estimated $450 – 900M PYS >$100M in Top 7 WW markets in US market Aevi’s R&D efforts include working with the Center for Applied Genomics (CAG) at Children's Hospital of Philadelphia (CHOP) CDG: Congenital Disorders of Glycosylation; RPDD: Rare Pediatric Disease Designation; ODD: Orphan Drug Designation 6| FTD: Fast Track Designation; TAM: Target Addressable Market; PYS: Peak Year Sales

Emerging Clinical-Stage, Rare Disease Pipeline Programs in • Building a rare disease biotech company Orphan Diseases focused on developing innovative medicines with Significant • Opportunity for multiple novel product 6 Unmet Needs launches through 2023 Development Stage Mechanism Lead Upcoming Program of Action Indication Preclinical Phase 1 Milestone D-Galactose PGM1-CDG CERC-801* replacement Pivotal Study Ready Initial data D-Mannose MPI-CDG from CERC-802* replacement Pivotal Study Ready CDG-FIRST 1H20 L-Fucose SLC35C1-CDG CERC-803* replacement IND-Enabling Anti-IL-18 Auto-inflammatory Initial POC AEVI-007 mAb diseases (AOSD, MM) Phase 1/2 Ready 4Q20/1Q21 mTORC1/2 Complex Lymphatic Initial POC AEVI-006** inhibitor Malformations Phase 1/2 Ready 4Q20/1Q21 Anti-LIGHT Pediatric Onset Initial POC AEVI-002 mAb Crohn’s Disease Phase 1/2 Study Ongoing 1H20 *Rare Pediatric Disease Designation Granted 7| **Rare Pediatric Disease Designation Eligible

Recent BD&L Improves Financials, Emphasizes Pipeline Sale of commercial assets provides cash and removes debt overhang, while novel asset acquisitions further strengthen pipeline and focus on R&D CERC BD&L Highlights GNMX BD&L Highlights Sale of pediatric commercial portfolio In-licensed two Phase 2-ready to Aytu Bioscience (NASDAQ:AYTU) programs from large pharma • Overall deal valued in excess of $43M • $500K upfront for WW license to AEVI-006, • $17M upfront composed of $4.5M in cash mTORC1/2 inhibitor for Lymphatic & $12.5M of AYTU preferred stock Malformations • Assumption of Cerecor’s outstanding • Recently exercised option with AstraZeneca payment obligations payable to Deerfield and for the WW license to AEVI-007; anti-IL-18 other liabilities in excess of $15M mAb for auto-inflammatory diseases • Estimated annual expense reduction of $7 to • Strong biological rationale with well- $9M associated with commercial sales characterized safety & pharmacology organization transfer to AYTU • Existing clinical proof-of-concept leveraging genetics-based and precision medicine Right-sized balance sheet and added high probability-of-success, mid-stage clinical programs targeting unmet needs in orphan diseases 8|

Management Team Extensive experience in orphan drug development & commercialization Michael F. Cola Dr. Pericles Calias Chief Executive Officer Chief Scientific Officer 25+ Years Industry Experience 20+ Years Industry Experience • President of Specialty Pharmaceuticals, Shire plc • VP Global CMC & Development, • EVP of Global Therapeutic Business Units and Sucampo Pharmaceuticals Portfolio Management, Shire plc • CSO, Pharming Group • President of the Life Sciences Group, Safequard • Senior Director Rare CNS Diseases and Device Scientifics, Inc. Lead, Shire plc • Senior positions in Product Development • Senior Director Drug Delivery and Chemistry, and Commercialization, AstraZeneca Eyetech Pharmaceuticals Joseph Miller Dr. Garry A. Neil Chief Financial Officer Chief Medical Officer 20+ Years Industry Experience 25+ Years Industry Experience • VP of Finance, Sucampo Pharmaceuticals • Corporate VP of Science & Technology, • Senior Director of Accounting, Qiagen Johnson & Johnson • Chief Financial Officer, Eppendorf 5Prime • Group President, Johnson & Johnson Pharmaceutical Research and Development • Certified Public Accountant • VP of R&D, Merck KGaA/EMD Pharmaceuticals • VP of Clinical Research, AstraZeneca Denotes former Aevi personnel 9|

CERC-800s Fast-to-market substrate replacement therapies for Congenital Disorders of Glycosylation

Congenital Disorders of Glycosylation (CDG): Life-Threatening, Ultra-Orphan, Inborn Errors of Metabolism (IEMs) Autosomal recessive diseases that result in impaired glycoprotein production and function, but are treatable with substrate replacement therapy Glycosylation is essential for protein Factor XI structure & function, particularly for Thrombin circulating proteins and enzymes such Liver Enzymes as hormones and coagulation factors Hemoglobin Due to a mutation in one of more than Normal Glycosylation 100 genes (including PGM1, MPI, and 3 Pattern SLC35C1), CDG patients lack the ability to synthesize functioning glycoproteins Life-threatening multi-system diseases: developmental delay, Substrate replacement 2 hypotonia, neurologic abnormalities, w/ oral monosaccharides hepatic disease, and coagulopathy Substrate replacement via oral monosaccharides results in clinical benefit in many patients 1 Glycosylation Deficiency due to CDG 11|

CERC-800s Substrate replacement therapies for CDGs Oral, small molecule, naturally-occurring monosaccharides used as standard-of-care for CDGs • Rapid onset of action OH OH • Safe and well-tolerated HO O HO O HO O • Documented clinical experience of efficacy HO OH HO OH HO OH • Symptoms return upon OH OH OH treatment withdrawal D-Galactose D-Mannose L-Fucose Eligibility CERC-801 CERC-802 CERC-803 Accelerated Pathway ✓ ✓ ✓ NCE 5-yrs Exclusivity ✓ ✓ ✓ FDA ODD 7-yrs Exclusivity ✓ ✓ ✓ EMA ODD 10-yrs Exclusivity ✓ ✓ ✓ Priority Review Voucher ✓ ✓ ✓ 12|

CDG Addressable Markets & Commercial Opportunity Opportunity for indication expansion to build a >$100mm franchise CDG Incidence Total Addressable Market Potential 1 in 50,000 to 100,000 with Label Expansion births annually Product Patients PPPY Revs CERC-801 1,000 $100,000 $100M CERC-802 500 $100,000 $50M 100 – 200 new cases per year CERC-803 50 $350,000 $17.5M 801 802 803 PGM1-CDG (3%) MPI-CDG (2%) SLC35C1-CDG (<1%) 3–6 new cases per year 2–4 new cases per year 1–2 new cases per year TMEM165-CDG Other CDGs SLC35A2-CDG 13| Peanne et al. Eur J Med Genet. 2017, Kjaergaard. Dan Med Bull. 2004, 51, pp. 350-63

CDG FIRST Trial: Retrospective Study to Accelerate Development & Approval of CERC-800s; Targeted NDA Filing(s) Starting in 2021 Multi-center, international, non-interventional, retrospective study of CDG patients who have been treated with unapproved sugar supplements North America Europe Retrospective Chart Reviews & Registry Data Have Been Successfully Used to Minimize or Obviate Prospective Clinical Studies Data to be collected: Natural History, Safety & Treatment Outcomes CDG Connect Patient Insights Network (PIN) https://connect.invitae.com/org/cdg 14|

Key Upcoming CERC-800s Milestones Multiple value-generating inflection points over the next 12 to 18 months • FDA meetings to define pathway to NDA submission and approval – Briefing package expected to include data collected under CDG FIRST – Determine pivotal study requirement and/or design Program Target Indication Upcoming Milestone • Initial data from CDG FIRST 1H 2020 CERC-801 PGM1-CDG • BTD Request 2Q/3Q 2020 (D-Galactose) • NDA Submission 1H 2021 • Initial data from CDG FIRST 1H 2020 CERC-802 MPI-CDG • BTD Request 2Q/3Q 2020 (D-Mannose) • NDA Submission 2H 2021 • Initial data from CDG FIRST 1H 2020 CERC-803 • IND Filing 1H 2020 Accelerated NDA Pathway NDA Accelerated SLC35C1-CDG (L-Fucose) • BTD Request 2Q/3Q 2020 • NDA Submission 1H 2022 15| BTD = Breakthrough Therapy Designation

AEVI-007 Phase 2-ready, anti-IL-18 monoclonal antibody for orphan auto-inflammatory diseases

IL-18-Mediated Autoimmune Disorders IL-18 is a pro-inflammatory cytokine produced by macrophages, stimulating production/release of IFNγ and regulating many immune processes Elevated Serum IL-18 Levels in AOSD Patients IL-18bp Response Rates 1000000 80 100000 10000 60 * 18 (pg/ml) 1000 40 100 Serum IL - 10 Active AOSD in AOSD in Comparison Comparison 20 AOSD Partial Remission Group Group Remission CRP>5 CRP<=5 of Responders Percentage • IL-18 is a key driver of several orphan 0 auto-inflammatory diseases 80 mg 160 mg – Adult Onset Still’s Disease (AOSD) (Week 12) (Week 12) – Multiple Myeloma (MM) Patients received subcutaneous • Serum IL-18 correlates with disease severity administration of 80 or 160 mg – AB2 Bio clinical proof-of-concept in AOSD (n = 23) three times per week using IL-18bp (T1/2 = 40 h); 4/4 patients with undetectable serum IL-18 had a clinical response 17|

Elevated IL-18 is Associated with Poor Prognosis in Myeloma IL-18 blockade has the potential to significantly prolong survival in multiple myeloma patients with elevated IL-18 levels • Patients with low IL-18 have IL-18 levels are elevated in many MM patients significantly longer median and correlate with poor survival survival (>84 mos vs. 42 mos) than patients with high IL-18 (p = 0.0026, HR = 1.84) • No association between bone marrow IL-18 levels and classical myeloma risk factors; IL-18 is an independent determinant of poor survival • Reducing IL-18 levels prolongs survival in rodent models • IL-18 could be a useful biomarker to select patients, (A) Bone marrow IL-18 levels, (B) Kaplan-Meier survival determine optimal dose curve of IL-18 high and IL-18 low patients, (C) Hazard ratio of survival based on bone marrow IL-18 levels 18| 1. Nakamura et al. (2018) Cancer Cell. 33(4):634-648.e5.

AEVI-007: Anti-IL-18 mAb Developed by Medimmune A high affinity, neutralizing monoclonal antibody against IL-18 • Potent and durable IL-18 inhibition – Evaluated in Phase 1 SAD for COPD – IV doses of 10, 30, 100, 300 or 1000 mg – Safe and well-tolerated (n = 31) • Phase 2-ready asset – 13-week monkey tox completed – Frozen, unformulated bulk material available to support clinical proof-of- concept in patients and nonclinical 6-month chronic tox studies AOSD MM US prevalence 3,500 – 7,000 ~124,000 Estimated % of patients treated 20 – 30% of market 10 – 12% of market PPPY $250,000 – 300,000 $100,000 – 125,000 Estimated PYS $400 – 500M $1 – 1.2B* 19| *US market only

AEVI-007 Development Plan Prioritize lower-risk, less capital intensive development strategy • Both programs are de-risked by preclinical and clinical proof-of-concept • Precision medicine approach using disease biomarkers is highly efficient and further de-risks clinical programs • Opportunity to efficiently demonstrate POC in target patient population(s) Open-Label NDA submission Signal Finding Studies in 2022 Drug (2H 2020 / 1H 2021) Formulation, (N=12) Requalification & Phase 3-enabling Toxicology (1H 2020) 20|

AEVI-006 Phase 2-ready, mTORC1/2 small molecule inhibitor for complex Lymphatic Malformations

mTOR Pathway in Lymphatic Malformations (LM) Lymphatic Malformations are a family of potentially life-threatening congenital diseases of the lymphatic system • Orphan disease(s) with combined US mTORC1/2 pathway prevalence of 30,000 to 60,000 patients – Kaposiform Lymphangiomatosis (KLA) – Generalized Lymphatic Anomaly (GLA) – Gorham Stout Disease (GSD) – Conducting Channel Anomalies (CCA) • High morbidity and mortality, pediatric diseases with unmet needs – GLA 7-year mortality rate = ~20% – KLA median OS = 2.75 years, 5-year OS = ~50% – No approved therapy © 2014 American Association for Cancer Research Molecular Cancer Therapeutics Reviews • Caused by mutations in PI3K/AKT/mTOR Cell Proliferation – Clinically meaningful responses with off-label use of mTORC1 inhibitor sirolimus Cell Survival Angiogenesis 1. Estimated from Perkins et al. (2010) Otolaryngol Head Neck Surg. 142(6):789-94. 2. Brouillard et al. (2014) J Clin Invest. 124(3):898-904. 3. Ozeki et al. (2016) Pediatr Blood Cancer. 63(5):832-8. 4. Croteau et al. (2014) J Pediatr. 164(2):383-8. 22| 5. Adams et al. (2016) Pediatrics. 137(2):e20153257.

Off-label Use of mTOR Inhibitor Sirolimus in LM Open-label clinical studies support efficacy, however use is limited by tolerability issues and lack of FDA approval • Phase 2 trial enrolled patients with complicated vascular anomalies – Study enrolled patients with different subtypes of LM not controlled by previous medication, sclerotherapy and/or surgery – Sirolimus was administered orally for 12 courses of 28 days each – 57 patients were evaluable for efficacy at the end of course 6, and 53 were evaluable at the end of course 12 • Safety and tolerability profile leads to low compliance, requires frequent monitoring – Physicians reported that sirolimus causes high rates of stomatitis (~60%) – Sirolimus bears black box warning for immunosuppression and malignancies Overall Response 6-month (n=57) 12-month (n=53) Grade 2 or > AEs Complete Response 0 0 • Blood/bone marrow (50%) Partial Response 47 (83%) 45 (85%) • Gastrointestinal (55%) Progressive Disease 7 (12%) 8 (15%) • Metabolic/laboratory (20%) • Infection (15%) Stable Disease 3 (5%) 0 23| 1. Adams et al. (2016) Pediatrics. 137(2):e20153257.

AEVI-006: mTORC1/2 Inhibitor Developed by Astellas High potency, 2nd generation, dual inhibitor of mTORC1/2 with potential for improved efficacy and tolerability • Phase 2-ready asset – 4-week nonclinical tox studies completed – Previously studied in Phase 1 MAD (n = 128) – Development discontinued upon determination that target efficacious doses were above MTD (30mg QD)1 – Significantly lower doses than MTD likely required to • Dual mTOR inhibitor maximizes impact of mTOR blockade, as mTORC2 is treat Lymphatic Malformations insensitive to rapalogs • LM presents an attractive market opportunity • Orally available, ATP-competitive kinase inhibitor; IC50 = 22 nM and 65 nM for – TAM estimated to be 10% of LM patients (~3,000) mTORC1 and mTORC2, respectively2 – Estimated PYS for US market = $450 – 900M • Active against a broad panel of tumor – PRV eligible, pricing similar to Afinitor ($190K/yr) cell lines, with IC50 values <10 µM, including models resistant to rapalogs2 LM/KLA: Open-Label Two-arm Open-Label PK/PD POC Trial (n = 10 - 12) Phase 2/3 Basket Trial (n = 60 - 80) Long Term Safety Extension (All Patients) 1. Mateo et al. (2016) Br J Cancer. 114(8):889-96. 24| 2. Bhagwat et al. (2011) Mol Cancer Ther. 10(8):1394-406.

AEVI-002 Anti-LIGHT monoclonal antibody in clinical studies for Severe Pediatric-Onset IBD

Elevated LIGHT is Associated with Active Inflammatory Disease A novel target for autoimmune disease identified via CHOP discovery • LIGHT (Lymphotoxin-like, exhibits Free LIGHT Levels in Pediatric Crohn’s Disease Inducible expression, and competes with HSV Glycoprotein D for HVEM, a receptor expressed by T lymphocytes) – Novel TNF superfamily member implicated as a key mediator of # p <0.1 inflammation * p <0.05 – Co-stimulator of T cells and can stimulate *** p <0.001 a Th1 profile of cytokines, including IFNγ1 – Expressed on activated T cells, NK cells, monocytes, granulocytes, and immature dendritic cells2 • Elevated free LIGHT levels detected in Pediatric Crohn’s Disease patients using n=9 n=16 n=24 n=34 Aevi’s proprietary assay Sample type grouped by time from diagnosis – Free LIGHT correlates with disease In a non-interventional study of 81 patients activity in patient plasma samples from undergoing colonoscopy and biopsy, CHOP BioBank elevated mRNA levels of LIGHT were – Plasma and tissue LIGHT levels are detected in the inflamed tissues of patients elevated in patients with IBD with IBD, compared to healthy individuals 1. Ware CF. (2005) Annu Rev Immunol. 23:787-819. 26| 2. Wang & Fu. (2004) Immunol Res. 30(2):201-214.

AEVI-002: Anti-LIGHT mAb Partnered with KHK Clinical-stage, first-in-class monoclonal antibody against LIGHT, originally discovered by La Jolla Allergy Institute • Fully-human, IgG4 antibody with successfully completed Phase 1 SAD study in healthy volunteers – Demonstrated good safety up to 1200 mg single doses LIGHT – Favorable PK profile supportive of q14d, sc dosing – 8-week monkey tox showed minimal immunogenicity and benign safety profile • Anti-LIGHT blockade presents on opportunity for an immuno-regulatory agent with a novel mode of action • LIGHT homo-trimer binds three – Initial orphan indication: Severe Pediatric-Onset IBD, a DcR3 molecules capable of life-altering, serious disease with significant unmet need neutralizing activity • Free LIGHT assay developed in collaboration with • Anti-LIGHT mAb AEVI-002 binds to the same sites as DcR3, MyriadRBM enables a precision medicine resulting in blockade of signal development approach transduction Phase 1/2 open-label signal finding study currently ongoing; initial data expected 1H 2020 1. Ware CF. (2005) Annu Rev Immunol. 23:787-819. 27| 2. Wang & Fu. (2004) Immunol Res. 30(2):201-214.

Key Financial Information

Financial & Investor Information Key financial highlights NASDAQ:CERC NASDAQ:GNMX • Share Price (as of 12/16/19) = $4.47 • Share Price (as of 12/16/19) = $0.14 – 52-week high = $7.66 – 52-week high = $1.31 – 52-week low = $2.71 – 52-week low = $0.11 • O/S = 45.8M • O/S = 64.8M • Market Cap (as of 12/16/19): $205M • Market Cap (as of 12/16/19): $9M • Average Daily Volume: 69K • Average Daily Volume: 359K • Pro-Forma Cash as of September 30, 2019 (following the closing of the Aytu transaction) = $9.1M Continued optionality and cash flows from Millipred®, with a means to assist in funding portfolio assets 29|

Executive Summary Unique potential for multiple drug approvals through 2023 • Execution on orphan pipeline to deliver value for patients and shareholders – Attractive commercial opportunities and 4 potential PRV awards from first-in-class medicines for diseases with no approved treatment options CERC-801 Pivotal CERC-801 Potential Study Initiation NDA APPROVAL CERC-802 Pivotal CERC-802 Potential Study Initiation NDA APPROVAL Initial Data from CERC-803 Pivotal CERC-803 Potential CDG FIRST Study Initiation NDA APPROVAL = PRV eligible 2020 2021 2022 2023 AEVI-007 Initial AEVI-007 Pivotal AEVI-007 Potential Proof-of-Concept Study Initiation NDA APPROVAL AEVI-006 Initial AEVI-006 Pivotal AEVI-006 Potential Proof-of-Concept Study Initiation NDA APPROVAL AEVI-002 Initial Proof-of-Concept 30|

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

Appendix

AOSD Treatment Paradigm Currently there are no approved targeted therapies for AOSD in the US, and no interventional clinical trials underway • Fever, rash, pharyngitis, arthritis, Opportunity to target sub-population presenting liver disease and increased ferritin with significantly elevated serum IL-18 for first-line AEVI-007 therapy – No definitive genetic or infectious cause – At least half have severe, chronic disease – Elevated serum IL-181 Line Corticosteroids st • Initially treated corticosteroids & 1 NSAIDs; 30 – 40% response rate 60–70% Non-responder – Non-responders treated with biologics (anti-IL-1/IL-6) +/- MTX; 30 – 40% still fail to respond Interleukin Line +/- Methotrexate – Initially position AEVI-007 for use in nd Inhibitors patients refractory to existing biologics 2 (~ 900 patients in US) 30–40% Non-responder – Precision medicine approach with predictive biomarkers will facilitate Likely TNF positioning as first-line therapy Line Role for rd IL-18 Inhibitors (2000–2500 patients in the US) 3 33| 1. Kudela et al. (2019) BMC Rheumatol. 3:4.

CERC-301: Phase I Results in nOH Highlighting 20mg Dose 20mg dose results in +29.8mm Hg increase in SBP over placebo at 6-hour post- dose OST, while only increasing +7.6mm Hg while seated 20mg (n = 8) vs Placebo (n = 12) pre-dose vs 6-hours post-dose 155 Seated Standing +7.6 (–20.7) 135 (–42.9) SBP (mm Hg) +29.8 115 CERC-301 reduces the magnitude of SBP drop following an OST 95 SBP SBP SBP SBP SBP SBP SBP SBP Seated -10 mins -5 mins Imed Prior +1 min +3 min +5 mins Seated Placebo - PD 20 mg - PD Placebo - 6hr 20 mg - 6 Hr 34|

CERC-301: A Pipeline Within a Product CERC-301’s differentiated profile expands addressable patient population to additional diseases characterized by low blood pressure Neurogenic Orthostatic Diabetic Orthostatic Intradialytic Hypotension Hypotension Hypotension (IDH) Estimated Patient Population Estimated Patient Population Estimated Patient Population 46.2M 3.0M 1.5M 220K 250K 405K US ROW US ROW US ROW Phase I Demonstrated Rapid, Initiating Phase I in Patients Initiating Phase II in Patients Robust & Sustained BP Effect by YE 2019 in 2020 in Patients • Large target addressable markets with limited therapeutic options – Northera® and midodrine act as direct adrenergic agonists with narrow clinical utility, resulting in unmet medical needs – Clinical proof-of-concept throughout 2019 and 2020 will create an opportunity for strategic partnership(s) – Seeking strategic partnership to support continued clinical development 35| Population estimates at mean values from the literature

Value of a Priority Review Voucher Recent PRV sales demonstrate stabilization in asset valuation Median purchase price = ~90mm 120 $110 $105 100 $95 $81 $80 80 60 40 20 0 2017 2018 2018 2019 2019 36| Reference: https://priorityreviewvoucher.org/

Preclinical Pipeline Additional early-stage, preclinical programs leveraging novel approaches to diseases with high unmet needs • Potential for 3 new IND filings throughout 2020 Mechanism Lead Development Stage Program of Action Indication Discovery Lead Opt IND-Enabling CNS-Targeted Parkinson’s CERC-406 COMT inhibitor Disease CERC Nucleoside DGUOK CERC-913 replacement Deficiency First-in-class mAb Rare auto- AEVI-005 (undisclosed target) inflammatory GNMX 37|