CorporateHighlights 30th AnnualPiperJaffray Healthcare Conference LotteNew YorkPalace,New York November 27th, 2018
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; 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 Inc.
Corporate Highlights • • Emerging clinical & early-stage pipeline Focus on orphan, neurological & pediatric indications • • Building a commercial capability Pediatric franchise generating positive cash flow • • Fully-integrated commercial and R&D organization New management team with proven track record 3 Transforming Cerecor Commercial Footprint Innovative Pipeline
Overview Management Team 1 Historical Milestones 2 3 Neurology & Pediatric Rare Disease Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 4
Management Team 20+ years industry experience 25+ years industry experience • Chairman and CEO, Sucampo Pharmaceuticals CEO, Histogenics Corporation President, MedImmune Ventures Manager, Centocor Biotech (Johnson & Johnson) • • • • President and COO of Zylera Pharmaceuticals Executive Director, Victory Pharma Director, Eisai Co, Ltd. Account Manager, Dura Pharmaceuticals, Inc. • • • 20+ years 25+ years industry experience • Vice President of Finance, Sucampo Pharmaceuticals Senior Director of Accounting, Qiagen Chief Financial Officer, Eppendorf 5Prime Certified Public Accountant • • Sr. Vice President | Principal The NSCI Group General Manager Specialty Pharmaceuticals, Covidien Vice President Marketing Pediatric Infectious Disease, MedImmune Sr. Director Marketing IMIDs, Centocor J&J Company Hospital Specialist, ATOD Rhone Poulenc Rorer • • • • • • 20+ years industry experience 8+years industry experience • V.P. Global CMC & Development, Sucampo Pharmaceuticals CSO, Pharming Group Sr. Director Rare CNS Diseases and Device Lead, Shire plc Sr. Director Drug Delivery and Chemistry, Eyetech Pharmaceuticals Ph.D., Tufts University, Bioorganic Chemistry • • Chairman, President at Ichorion Therapeutics SVP, Business Development at Vyera Pharmaceuticals BD Analyst at Retrophin MSc, CPhil in Statistics, UCLA • • • • • • 5 Patrick Crutcher VP Business Development Dr. Pericles Calias Chief Scientific Officer Joseph Miller Chief Financial Officer James A. Harrell EVP Marketing, Investor Relations Matthew V. Phillips Chief Commercial Officer Peter S. Greenleaf President & CEO
Historical Milestones 2017 2018 Acquisition TRx Pharma (Zylera) Acquisition Avadel (AVDL) Pediatric assets CERC-501 Acquired worldwide rights from Eli Lilly CERC-611 Acquired worldwide rights from Eli Lilly CERC-501 Sold to Janssen Acquisition Ichorion Rare Disease Pipeline CERC-301 Enters Phase 1 study for nOH 6 2013201420152016 CERC-301Cerecor IPO CERC-406October 15, 2015 Acquired worldwide rights of two NMDA receptors from Merck Management Change
Cerecor Evolution Innovative Approaches to CNS Diseases FDA-Approved Products Poly-Vi-Flor® | Tri-Vi-Flor® Karbinal™ ER AcipHex® Sprinkle™ Cefaclor Flexichamber™ Millipred® | Veripred® Ulesfia® 505(b)(2) Assets & Platform Chemistry CERC-801 CERC-802 CERC-803 CERC-913 • • • • • • • • • • • CERC-301 CERC-406 CERC-501 CERC-611 • • • • 7 Robust R&D Pipeline Capability & Cash Flow In-Licensed CNS Assets Pediatric Rare Diseases Pediatric Franchise Neurological Disorders
Emerging Clinical & Early-Stage Pipeline Each program supported by Clinical and/or Genetic Validation Enabling Receptor antagonist Hypotension (nOH) COMT inhibitor (2nd Gen) symptoms of Parkinson’s Receptor antagonist epilepsy Syndrome (MDS) CERC-700(s) Four Additional 505(b)(2) Programs utilizing MicroPump and LiquiTime Dosing Technology are also within the pipeline being developed in conjunction with Avadel Pharmaceuticals in the area of Pediatrics 8 ProgramMechanism of ActionTarget IndicationLead OptIND-Phase 1 Neurology Division NCE CERC-301GluN2B selective, NMDANeurogenic Orthostatic CERC-406CNS-targeted, selectiveMotoric and non-motoric CERC-611TARP-8 dependent AMPAPartial onset seizures in Pediatric Division 505(b)(2) NCEs CERC-801D-Galactose replacementPGM1-CDG CERC-802D-Mannose replacementMPI-CDG CERC-803L-Fucose replacementSLC35C1-CDG NCE CERC-913Nucleoside replacementmtDNA Depletion
Market Potential 40M 500 500 250 50 CERC-301 CERC-406 CERC-611 CERC-801 CERC-802 CERC-803 CERC-913 MDS nOH Parkinson’s Disease Epilepsy PGM1-CDG MPI-CDG SLC35C1-CDG 9 1.9M Ultra Rare Pediatric Orphan Diseases 700k World-Wide Estimated Number of Patients
Upcoming Clinical Program Milestones Multiple value generating inflection points over next 6 to 18 months Program Mechanism-of-Action Milestone IND Filing 2019 PGM1-CDG IND Filing 2019 MPI-CDG IND Filing 1H 2020 SLC35C1-CDG 10 *505(b)(2) Pathway Neurological Disorders CERC-301 Neurogenic Orthostatic Hypotension CERC-406 Adjunct for Parkinson’s Disease CERC-611 Partial Onset Seizures Epilepsy NR2B selective NMDA receptor antagonistPhase I Readout 1H19 CNS penetrant & selective COMT inhibitorIND Filing 1H 2020 TARP-8 dependent AMPA receptor antagonistIn Process Pediatric / Metabolic Disorders CERC-801* Inborn Error of Metabolism (IEM) CERC-802* Inborn Error of Metabolism (IEM) CERC-803* Inborn Error of Metabolism (IEM) CERC-913 Mitochondrial Disorder Substrate replacement therapy Substrate replacement therapy Substrate replacement therapy ProTide nucleotideIND Filing 2020
CERC-301 NR2B selective NMDA receptor antagonist for nOH Initiated Phase 1 study of CERC-301 in Parkinson's patients diagnosed with neurogenic Orthostatic Hypotension (nOH) • Characterized by a sudden drop in blood pressure upon standing Caused by autonomic vasoconstrictor failure, increasing risk of falls or injury Estimated less than 200,000 patients in the U.S. (ODD eligible) Only FDA-Approved therapy: Northera 2018 revs ~$260mm • Orally-available NR2B antagonist with established safety profile in >400 patients & HVs Multiple studies demonstrate rapid, robust & dose-dependent increase in Systolic Blood Pressure (SBP) • • • • nOH Dopamine beta hydroxylase (DBH) deficiency Multiple System Atrophy (MSA) Parkinson’s disease Non-Diabetic neuropathy Pure autonomic Failure (PAF) 11 nOH CERC-301 Clinical Data
CERC-406 COMT inhibitor for Parkinson’s Disease 2nd generation, CNS selective COMT inhibitor to minimize toxicity & 1st enhance efficacy seen with generation therapies • COMT drives the catabolism of dopamine and levodopa Inhibition of dopamine catabolism used to treat CNS manifestations Parkinson’s Disease A CNS selective COMT inhibitor Polycystic Kidney Disease • is of Impulsivity Addiction COMT inhibition could serve as adjunct therapy for conditions driven by Dopamine deficiencies • presents the opportunity to minimize systemic toxicity associated with generation COMT inhibitors 1st Restless Leg Syndrome Fibromyalgia • Targeted therapy biomarker with clinical Transthyretin Associated Amyloidosis 12
CERC-611 TARP-8 dependent AMPA receptor antagonist for partial onset seizures Phase 1-ready candidate with therapeutic potential for partial onset seizures in patients with epilepsy • Epilepsy affects over 65 million patients worldwide 30%-40% of patients refractory; high degree of poly-pharmacy common All anti-seizure drugs have side effects (e.g. motoric) limiting use and the timely achievement of therapeutic dose levels • AMPA receptors mediate fast synaptic neurotransmission within the CNS and are a proven target for anti-seizure efficacy CERC-611 is the first known AMPA receptor antagonist that selectively targets the hippocampus CERC-611 shows lack of motoric impairment at efficacious exposures in animal models of epilepsy • • • • 13 Unique Mechanism of Action Significant Unmet Need
CERC-800’s Substrate replacement therapies for CDGs Oral, small molecule, genetically-targeted therapies Multi-system disease manifestation in PGM1-CDG D-Galactose leads to significant improvement in key clinical symptoms CERC-801 Life-threatening gastrointestinal disorder in MPI-CDG D-Mannose rapidly resolves hematological & intestinal abnormalities CERC-802 Immunodeficiency with CNS impairment in SLC35C1-CDG L-Fucose normalizes cell counts & reduces infection risk CERC-803 • • • <500 patients WW per indication High pediatric morbidity & mortality No approved treatments • • • Documented efficacy & safety Clinical symptoms improve rapidly Reduced nonclinical requirements 14 Ultra-orphan IEMs with serious and life-threatening medical needs
CERC-800’s Substrate replacement therapies for CDGs Congenital Disorders of Glycosylation (CDGs): Monogenic disorders resulting in glycosylation defects with broad clinical spectrum D-Galactose CERC-801 D-Mannose CERC-802 L-Fucose CERC-803 505(b)(2) NDA Pathway NCE 5-yrs Exclusivity ODD 7-yrs Exclusivity Priority Review Voucher EMA ODD 10-yrs Exclusivity 15 Eligibility801802803
CERC-913 ProTide Nucleotide for Mitochondrial DNA Depletion Syndrome (MDS) Overcome key limitations of direct substrate replacement: stability, permeability & kinase bypass Phenotypic Rescue • • • 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-913 Attributes
Overview Management Team 1 Historical Milestones 2 3 Neurology & Pediatric Rare Disease Pipeline 4 Commercial Pediatric Portfolio 5 Strategic Growth Plans and Outlook 6 Financial Highlights 17
Building Commercial Capabilities in Pediatrics ~50% of the Overall Market Potential Covered with 32 Territories Sales Force Expansion Underway to Increase SOV • • • • • • • • • 1 Chief Commercial Officer 1 EVP Marketing & Investor Relations 1 Director of Sales 3 Regional Sales Managers 1 Director of Sales Training and Operations 1 Director of Trade Sales and Operations 1 Institutional Sales Manager 1 Sales Analyst 32 Territory Managers Expanding to 44 • • • • 3 Focused / 8 Promoted Products Territory Expansion Underway Cloud-based CRM System Novel SICP 18
Pediatric Franchise with Eight Product Lines 19 Avadel Pediatric Assets TRX Pharmaceuticals
Why Pediatrics? Pediatrics Represents a Focused, Defined and Specific Patient Population Treated by One Specialty Segment 1. 2. 3. 4. 5. 6. 7. 8. Routine Child Health Examination Acute Upper Respiratory Infection Otitis Media Acute Pharyngitis Asthma Follow-up Exam Allergic Rhinitis Sinusitis 9. 10. 11. 12. 13. 14. 15. 16. Dermatitis Attention-Deficit / Hyperactivity Disorder Cough Viral Infection Streptococcal Sore Throat Bronchitis Conjunctivitis Esophageal Reflux 17. 18. 19. 20. 21. 22. 23. 24. Influenza with Respiratory Manif. Gastroenteritis / Colitis Fever Constipation Vaccination Abdominal Pain Viral Diseases Pneumonia 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). 20 1. AAP pediatric coding newsletter coding.aap.org August 2013 Top 25 Pediatric Codes 2013 AAP Pediatric Coding Newsletter1 Our Existing Pediatric Product Portfolio Treats Nearly 75% of the Top 25 Pediatric Diagnosis Codes
Net Product Sales FY18 Net Sales Guidance = $18 to $20 million 21 Net Product Sales Last 4 QTR Trend (in $000’s)
Balance Sheet 22 Total Assets Trend Analysis (in $000’s) Cash Trend Analysis (in $000’s)
2019 Growth Plans 1 3 2 CERC-301 CERC-406 CERC-611 CERC-801 CERC-802 CERC-803 CERC-913 Acquire/in-license commercial-ready or marketed asset(s) Acquire/in-license complimentary pipeline assets Grow Market Share Expand Commercial Footprint 23 Advance Pipeline Accelerate Business Development Activity Build Commercial Excellence
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