Forward Looking Statements

Transcription

1 Antiviral Therapies

2 Forward Looking Statements This presentation contains forward-looking statements, including the timing of our drug development programs. Risks include delays in manufacturing created by third parties and the ability of clinical research organizations to recruit patients. Forwardlooking statements also are prefaced by words such as "expect," "plan," "intend," "anticipate," and similar words. Forward-looking statements are based on our current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements for a variety of reasons, including those contained in our Form 10-K, as amended, for the year ended December 31, 2015, form 10Q for the quarter ending June 30, We caution you, therefore, against relying on any of these forward-looking statements. They are neither statements of historical fact nor guarantees or assurances of future performance. We do not undertake any duty to update these forward-looking statements. 2

3 Company Highlights Clinical stage antiviral company Multiple opportunities in different viral diseases Influenza Hepatitis C Norovirus PB2 inhibitors, PA inhibitors, PB-1 inhibitors NNI, nucleoside inhibitors, helicase inhibitors, NS5A inhibitors Nucleoside inhibitors, NNI Hepatitis B CRISPR/Cas 9 Human papilloma virus CRISPR/Cas9 3

4 Crystallography Technology Platform Fragment hits Fragment A Fragment B Fragment E Fragment D Fragment C Ability to quickly grow ultra-high resolution crystals of replication enzymes Rapid turnaround of structural information through highly automated X-ray data processing and refinement Discovery of novel binding sites overlay structure of fragments bound to novel sites Provides 3D structure of inhibitor complexes at near-atomic resolution provides immediate insight to guide drug discovery 4

5 Cocrystal Drug Discovery Process Near atomic resolution, X-ray quality crystal production Drug pocket selection Hit-to-lead process Lead optimization Drug candidates 5

6 Opportunities There exists significant unmet medical needs across a large variety of viral infections including Influenza A & B Seasonal and pandemic 3-5 million infections/year Estimated economic impact of seasonal flu in US: $50B to $150B Hepatitis B & C Leading causes of liver failure and liver cancer Chronic infections >100 million HCV >400 million HBV Opportunity for shorter duration in HCV and a cure in HBV Norovirus Chronic (potentially orphan indication) Acute gastroenteritis >250 million acute cases/year No treatment available Economic cost in the US alone >$5 Billion 6

7 Company Pipeline Therapeutic area Hepatitis C CC (NNI) Hepatitis C CC-2069 (NS5A) Hepatitis C CC-2850 (Nuc) Influenza A PB2 inhibitor Norovirus Nuc & NNI Hepatitis B and HPV (CRISPR/Cas9) Lead Discovery Preclinical Phase I Phase II 7

8 HCV Market Dynamics Today and in the future Huge market: >10 million patients across the US, Japan, Western Europe Currently treating 400,000+ patients per year Many undiagnosed patients Pricing will be competitive, but the market will still be significant in the foreseeable future Great need for a shorter duration therapy * Bloomberg Intelligence (BI) projections, as of

9 Cocrystal s HCV DAA Combinations Multiple opportunities in developing combination ultra-short, all oral pan-genotypic cure (in-house or with partners) Pan-genotypic NS5B NNI Pan-genotypic NS5A Inhibitor Pan-genotypic NS5B Nuc Oral, Pan-genotypic, Ultra-short therapy 9

10 CC-31244: Pan-genotypic NNI Highly potent NS5B polymerase inhibitor (EC50 = 7nM) Pan-genotypic activity (genotypes 1-6) High barrier to drug resistance Phase Ia dose escalating safety study completed Viral load data in HCV subjects available in Q4,

11 Unique Drug Resistance Property of Cocrystal NNIs CC shows pan-genotypic and broad activity against common drug resistant variants HCV-796 IC 50 fold change Cocrystal s NNI-4 Leads Cocrystal s Backup Leads Drug resistance variants S365T (NNI-4) N316Y (NNI-4) L419M (NNI-2) S282T (Nuc) CC HCV

12 CC Phase Ia Clinical Trial Update A single- and multiple-dose assessment of the safety and pharmacokinetics of pan-genotypic NNI, CC Single-dose completed: five cohorts of healthy volunteers at 10, 50, 100, 200, and 400 mg Multiple-dose completed: two cohorts of healthy volunteers at 200 mg x 7 days and 400 mg x 7 days) No adverse effects observed 12

13 CC Phase Ib Clinical Trial Update Proof-of-concept Phase 1b study ongoing HCV infected subjects with minimal fibrosis and no significant co-morbidities Repeat-dose, randomized, monotherapy trial (400 mg x 7 days) Viral load, drug resistance, and pharmacokinetic properties will be determined 13

14 CC-2069: Pan-genotypic NS5A Novel, highly potent, pan-genotypic, NS5A inhibitor (GT1b EC 50 < 10 pm) Active against common NS5A drug resistant variants Preclinical characterization ongoing 14

15 HCV Nucleoside Program: CC-2850 Search for next generation backbone for combination therapy Novel, potent, pan-genotypic nucleoside inhibitor Active against common nucleoside inhibitor drug resistant (S282T) Preclinical characterization ongoing 15

16 HCV Helicase Lead Discovery Program Provides unique opportunities for drug combinations Inhibits essential viral RNA unwinding process First-in-class pan-genotypic inhibitors (new mechanism of action) Highly conserved drug binding mode demonstrated in all genotype crystals developed (Genotypes 1-6) Potentially an ideal combination candidate with HCV Nuc, NNI, NS5A, and/or protease inhibitors 16

17 Influenza Program Influenza leads: PB2, PB1 and PA Inhibitors Focus on three different types of influenza polymerase inhibitors: PB2 (cap-binding), PB1 (polymerase), and PA (endonuclease) Novel, potent structure-based influenza A PB2 inhibitors are at preclinical stage 17

18 Influenza Program Potent PB2 leads bind to highly conserved binding pocket Selected influenza A PB2 crystals Influenza PB2 inhibitor H1N Virginia H1N Spanish H7N Zhejiang H5N Guangdong H1N H5N H7N H1N

19 Norovirus Program Unmet & underappreciated medical need Prophylaxis Treatment Ø Acute (foodborne) Ø Chronic (Immunocompromised) Ø Chronic (transplant patients) million illness each year (1 in 14 Americans become ill each year) 19

20 Norovirus Program Broad spectrum Noro Polymerase Inhibitors Noro nucleosides Active nucleoside candidates identified Noro NNI Drugable pocket identified Animal model data supports activity in vivo Lead discovery is ongoing Human Noro Human Norwalk Murine Noro Human Norwalk Human Noro Murine Noro 20

21 Hepatitis B Market There is no approved cure at this time Huge market: 400 million chronically infected globally; as many as 2 million infected with chronic HBV in US alone Current therapies only suppress, but do not eliminate 21

22 CRISPR/Cas9 For Potential HBV Therapy In-licensed from Duke University and Emory University for Hepatitis B and Human Papilloma Virus CRISPR/Cas9 allows for editing of viral DNA Potential cure for chronic HBV 22

23 Cocrystal Board of Directors Raymond Schinazi (Chairman) Gary Wilcox (Vice Chairman) David Block Phil Frost Jane Hsaio Steven Rubin 23

24 Cocrystal Management Gary Wilcox, Ph.D., Interim CEO Chairman and CEO of Cocrystal Pharma ; Board of Directors and Executive Vice President of Operations of Icos Corporation (ICOS-NASDAQ) from 1993 to Vice Chairman, Executive Vice President, and Director of Xoma Corporation (XOMA-NASDAQ); Chairman, CEO, and President of Ingene (IGEI-NASDAQ) from ; Professor of Microbiology at UCLA for 10 years. Sam Lee, Ph.D. President President of Cocrystal Pharma 2008-present; Anti-infective drug discovery project management ICOS Corporation Dr. Lee received his Ph.D. in Biological Sciences from the University of Notre Dame and postdoctoral training in viral biochemistry with Dr. I. R. Lehman at Stanford University. Curtis Dale, Interim Chief Financial Officer and Controller Corporate Controller Solvay Pharmaceuticals; Executive Finance Director, Stiefel Laboratories; Multiple financial positions, Bristol-Meyers Squibb. Walt Linscott, General Counsel and Corporate Secretary General Counsel and Corporate Secretary, Carestream Health; Vice President, General Counsel and Corporate Secretary, Solvay Pharmaceuticals; Chair of Life Sciences Practice of an international law firm. 24

25 Scientific Leadership Dr. Roger Kornberg Cocrystal Co-founder 2006 Nobel prize winner in chemistry Dr. Raymond Schinazi Co-Founder of Pharmasset, Idenix, Triangle; Founder, RFS Pharma 25

26 Transformational Year: 2016 HCV Initiated clinical trial for CC (NNI) Completed Phase Ia dose escalation safety study Antiviral activity data in HCV subjects for CC in Q Preclinical characterization of CC-2069 (NS5A) - ongoing Preclinical characterization of CC-2850 (Nuc) - ongoing Influenza Lead selection of influenza A PB-2 inhibitors Norovirus Nucleoside/NNI in discovery stage HBV (CRISPR Cas 9) In vitro proof of concept and animal model studies of CRISPR/Cas9 for hepatitis B ongoing 26