Cocrystal Pharma, Inc. · NASDAQ:COCP
Cocrystal Pharma — Investor Presentation (June 2018)
48 slides from Cocrystal Pharma's investor presentation, Jun 4, 2018
Slide 1: Corporate Presentation June 2018
C#CRYSTAL Antiviral Therapies Paige MA el NC. Corporate Presentation June 2018 NASDAQ: COCP www.cocrystalpharma.com
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the anticipated timing of our drug development programs, including 2018 milestones, and anticipated completion o
Slide 3: Corporate Overview
Corporate Overview Clinical Stage Antiviral Company Hepatitis Wholly Owned Product Portfolio Influenza Norovirus Gastroenteritis Proprietary Drug Discovery Platform crystalpharma.com
Slide 4: Investment Highlights
Investment Highlights « Nobel Prize winning expertise leveraged to design first- and best-in-class antiviral drug candidates addressing well established and growing markets « Lead program CC-31244 for the treatment of hepatitis C infection entering Phase 2a in Q2 2018 - Received IND clearance from F
Slide 5: Robust Development Pipeline
Robust Development Pipeline Discovery Preclinical Program cc-31244 (Pan-genotypic NS5B NNI) Hepatitis C | CCc-2850 (Hcv) (Pan-genotypic NSSB Nuc) CC-2069 (Pan-genotypic NSSA inhibitor) (CC-42344 (Influenza A PB2 inhibitor) Influenza Influenza A/B inhibitor Norovirus Noro Polymerase Inhibitor www.coc
Slide 6: Near-Term Milestones Expected to Drive Value
Near-Term Milestones Expected to Drive Value HCV CC-31244: v Filed IND with FDA Vv Received IND clearance from FDA for Phase 2a HCV CC-31244: * Commence patient enrollment in Phase 2a study HCV CC-31244: * Complete Phase 2a study dosing Influenza CC-42344: HCV CC-31244: * Announce Phase 2a topline r
Slide 7: Seasoned Management Team
Seasoned Management Team Gary Wilcox, Ph.D. Vice Chairman and Chief Executive Officer Over 35 years of executive biotech leadership experience and played a key role in the development of Cialis Cialis: tadalafil Sam Lee, Ph.D. President 20 years of anti-infective drug discovery research experience a
Slide 8: Board of Directors
Board of Directors Raymond F. Schinazi, Ph.D. Frances Winship Walters Professor of Pediatrics, Director, Laboratory of Biochemical Chairman Pharmacology and Director, HIV Cure Scientific Working Group at Emory University Gary Wilcox, Ph.D. Vice Chairman and Vice Chairman and Chief Executive Officer,
Slide 9: Cocrystal Technology
Cocrystal Technology Nobel Prize Winning Technology Le | Near atomic resolution, “ X-ray quality crystal production bow © Drug pocket selection a « Hit-to-lead process ee ~ Lead optimization é& Drug candidates www.cocrystalpharma.com
Slide 10: CC-31244: Broad Spectrum HCV NNI
CC-31244: Broad Spectrum HCV NNI Demonstration of Cocrystal’s Enabling Technology HCV GT1 — GT6 NSSB polymerase crystals HCV NS5B polymerase Proven track record for broad spectrum antiviral leads Cc NS (@ RYS TA L www.cocrystalpharma.com
Slide 11: Hepatitis C Treatment Market Share
Hepatitis C Treatment Market Share 2017 Annual Sales: $12.69 Billion Q1 2018 Sales: $2.39 Billion J&J $142M Gilead $9.14 B = Gilead (Harvoni, Epclusa, Sovaldi, Vosevi) mJ&J(Olysio) mAbbVie (Viekira, Mavyret) m Merck (Zepatier) Source: 2017 Form 10-K Source: 2018 Form 10-Q. www.cocrystalpharma.com
Slide 12: AbbVie's Mavyret Demonstrated a Shorter Treatment
AbbVie’s Mavyret Demonstrated a Shorter Treatment: From 12 Weeks To 8 Weeks Approved broad spectrum HCV combination therapy Nuc + NSSA inhibitor PI + NSSA inhibitor Gilead’s EPCLUSA AbbVie’s Mavyret (sofosbuvir 400mg/ (glecaprevir 100 mg/ velpatasvir 100 mg) pibrentasvir 40 mg) 12-week treatment 8-w
Slide 13: Cocrystal's HCV Strategy: Shorter Combination Therapy
Cocrystal’s HCV Strategy: Shorter Combination Therapy Multiple opportunities in developing shorter combination therapy with approved HCV drugs NSSA Inhibitor =) CC-31244 NS5B NNI fs NS5B Nuc => 2 or 3 broad spectrum 3) drugs for shorter therapy NS3 Protease Inhibitor www.cocrystalpharma.com
Slide 14: Cocrystal's Next Wave Combination Therapy
Cocrystal’s Next Wave Combination Therapy: CC-31244 with Approved HCV Drugs * Potential best-in-class HCV NNI with a strong profile - Broad spectrum, potent NSS5B polymerase inhibitor Developed by Cocrystal’s proprietary structure-based discovery platform High barrier to drug resistance Effective ag
Slide 15: Phase 1a Study Completed
Phase 1a Study Completed s 2 20 mg 50 mg, 100mg 200mg* | 400mg Single ascending doses (complete) = N = 40; 30 CC-31244, 10 placebo; * food effect assessed Multiple ascending doses => 200 mg BID 400 mg QD (complete) N = 16; 12 CC-31244, 4 placebo x 7 days Endpoints ° Safety: adverse events (AEs) and
Slide 16: Phase 1b Study Completed
Phase 1b Study Completed 200 mg BID x7 days Status N = 4; 3 CC-31244,1 placebo Complete HCV GT1 | HCV RNA2 5 log, .|U/ml Naive 400 mg QD x 7 days Status | N = 5; 4 CC-31244, 1 placebo Complete Single doses 1 QD or BID x7 days N = 5; 4 CC-31244, 1 placebo Complete Endpoints ° Efficacy: changes in HCV
Slide 17: Superior Viral Load Reduction
Superior Viral Load Reduction * HCV RNA viral load decline of 3 logs by 48 hours « After the NNI treatment, the viral load levels were slowly increased Mean Viral Load Change From Baseline os (CC-31244 at 400 mg QD or 200 mg BID x7 days) ———————————————————— 01 =e CC-31244 No CC-31244 “a 400 mg QD 4
Slide 18: Best-in-Class Potential of Any NNI
Best-in-Class Potential of Any NNI Dose Treatment se lleete) Drug Genotype mn Duration (dave reduction (mg) (days) (Log,olU/ml) CC-31244 @ Genotype1-6 @ 400 @ 7 (QD) -3.0 ABT-333* (Dasabuvir) Genotype 1 400 3 (BID) -1.08 800 3 (BID) -0.95 GS-9190 (Tegobuvir) Genotype 1 40 3 (BID) -1.0 120 3 (BID) 45
Slide 19: CC-31244 Phase 2a Study Design
CC-31244 Phase 2a Study Design * An open-label, Phase 2a study evaluating the safety, tolerability and preliminary efficacy of CC-31244 with approved HCV DAAs CC-31244 (400 mg) QD + a 2-week treatment Approved DAAs * Endpoints « Efficacy: changes in HCV RNA viral load * Safety: adverse events (AEs)
Slide 20: HCV Summary and Conclusion
HCV Summary and Conclusion * Showed an acceptable safety profile in both healthy volunteers and GT1 patients up to 400 mg x 7 days * No serious adverse events or discontinuations due to adverse events * Demonstrated HCV RNA viral load reduction of ~ 3 logs by 48 hours * Demonstrated a sustained post
Slide 21: New Antivirals For Influenza
New Antivirals For Influenza * High mortality rate: - 1918 Spanish Flu, 20-100 million deaths - 1957 Asian Flu, 1-1.5 million deaths - 1968 Hong Kong Flu, 0.75-1 million deaths - 2009 Swine Flu, 0.15-0.5 million deaths * Emerging influenza viruses - Highly virulent avian influenza viruses - Tamiflu-
Slide 22: Great Opportunity in Influenza Antiviral Market
Great Opportunity in Influenza Antiviral Market * Seasonal and pandemic infection - 3-5 million cases of severe illness per year - 250,000 — 500,000 deaths worldwide* « Approved influenza therapies have major limitation * Multiple product routes of delivery, inhalation, oral, and intravenous (IV) *
Slide 23: Influenza A Preclinical Lead CC-42344
Influenza A Preclinical Lead CC-42344 Influenza Crystals Potent and favorable PK profiles CC-42344 Excellent anti-influenza activity against pandemic, . seasonal, and Tamiflu resistant influenza strains Binds a highly conserved site Novel mechanism of action IND filing scheduled in 2018 www.cocrysta
Slide 24: Early Stage Programs
Early Stage Programs ¢ Influenza A/B Inhibitor Program - Influenza cocrystals developed - Structure-based lead discovery ongoing - IND filing in 2019 ¢ Noro Polymerase Inhibitor - Structure-based NNI discovery ongoing - Multiple polymerase crystals developed - Noro nucleoside lead discovery ongoing
Slide 25: Cocrystal-HitGen-InterX Collaboration
Cocrystal-HitGen-InterX Collaboration: Aimed at Rapid Lead Discovery Process GR ecrYSTAL (BP inter * Combines synergistic drug discovery platforms developed by Cocrystal- HitGen-InterX * Break through hit-to-lead process * High quality novel leads will be developed CBC RYST L www.cocrystalpharma.com
Slide 26: Selected Value Indicators Suggest Potential Significant Upside
Selected Value Indicators Suggest Potential Significant Upside Company Name Ticker © Market Cap (M)* Overview RO renee we geen Develops medicines to treat intractable diseases by silencing the 2 Phase 1 genes that cause them 6 Preclinical i ; . , 1 Phase 2 Arbutus BioPharma ‘ABUS $323 Blophiarmaredt
Slide 27: Financial Snapshot
Financial Snapshot — Nasdaq: COCP $67.3MM 29.9MM 60K Market cap* Common shares Average daily outstanding volume** * Based on May 31, 2018 closingprice of $2.25 pershare. ** Based on May 31, 2018 Yahoo Finance. www.cocrystalpharma.com
Slide 28: COCP Capitalization Table
COCP Capitalization Table y Capitalization Table (As of May 31, 2018) # of Shares WAEP $ Value moray (Common Shares Outstanding (Directors & Officers) 15,219,800 49.75% (Common Shares Outstanding (Other) 14,703,276 48.06% Warrants. 243,375 $10.28 $2,501,895 0.80% Stock Options 425,637 $12.44 $5,294,
Slide 29: Near-Term Milestones Expected to Drive Value
Near-Term Milestones Expected to Drive Value HCV CC-31244: v Filed IND with FDA Vv Received IND clearance from FDA for Phase 2a HCV CC-31244: * Commence patient enrollment in Phase 2a study HCV CC-31244: * Complete Phase 2a study dosing Influenza CC-42344: HCV CC-31244: * Announce Phase 2a topline r
Slide 30: Thank You!
C¥CRYSTAL PHARMA,IN Thank You! NASDAQ: COCP www.cocrystalpharma.com
Slide 31: C¥’CRYSTAL
C¥’CRYSTAL POH A RIM A, EEC. NASDAQ: COCP www.cocrystalpharma.com
Slide 32: Appendix
C¥CRYSTAL PHARMA,IN Appendix NASDAQ: COCP www.cocrystalpharma.com
Slide 33: Scientific Advisors
Scientific Advisors Roger Kornberg, Ph.D. Professor of Structural Biology at Stanford University School of Medicine. Nobel Laureate in Chief Scientist, Chainrmanof Chemistry, Member of the National Academy of Sciences, and the American Academy of Arts and Scientific Advisory Board Sciences Stanford
Slide 34: Cocrystal Patent Portfolio
Cocrystal Patent Portfolio * HCV: 15 patents including three PCT applications * Influenza: 2 patent applications filed * Noro: patent application(s) will be filed in 2018 Cc NS (S RY www.cocrystalpharma.com AR
Slide 35: CC-31244 Binds To a Highly Conserved Drug Binding Site
CC-31244 Binds To a Highly Conserved Drug Binding Site (NNI-4) of NS5B Polymerase (A) HCV NSSB polymerase (B) Highly conserved NNI-4 site g C- < Sf GDD Motif ) ce (S RYSTA L www.cocrystalpharma.com
Slide 36: CC-31244: Pan-genotypic NS5B NNI
CC-31244: Pan-genotypic NS5B NNI * CC-31244 HCV replicon EC,, fold change, <6 fold HCV replicon/chimeric replicon EC¢, results Genotype CDI-31244 EC. Sofosbuvir EC.) EC,),mM Fold change EC,),mM fold change 1b 0.005 1.0 0.042 1.0 1a 0.009 1.8 0.034 0.8 2b 0.026 52 0.028 0.66 3a 0.011 2.2 0.14 32 4a 0
Slide 37: CC-31244 Exhibits Excellent Activity Against Common NNI and Nuc Drug Resistant Variants
CC-31244 Exhibits Excellent Activity Against Common NNI and Nuc Drug Resistant Variants NNI IC; fold change CC-31244 <5 HCV-796 >1,500 HCV-796 o™ a 1600 & ino Drug resistance variants $s 1200 Cocrystal’s ZS 1000 Backup Leads [i S365T (NNI-4) {2} _ 800 y y y J Ml N316Y (NNI-4) os” @ L419m (NNI-2) 00
Slide 38: GT1b NS5B C445F: The Major Drug Resistance Variant of CC-31244
GT1b NS5B C445F: The Major Drug Resistance Variant of CC-31244 * HCV GT1b replicons containing NS5B variants identified by CC-31244 resistant colony selection GT1b C445F/S549G HCV replicon) ECog HM Purified NS5B: polymerase ICco fold change www.cocrystalpharma.com
Slide 39: CC-31244 Binding Mode: GT1b Drug Resistant NS5B C445F
CC-31244 Binding Mode: GT1b Drug Resistant NS5B C445F a | j (B) GT1b C445F ah (A) GT1b wild type 4 (B)
Slide 40: CC-31244 Exhibits Excellent Liver Targeting
CC-31244 Exhibits Excellent Liver Targeting Rat liver study CC-31244, ng/mL 30000 25000 20000 15000 10000 5000 Avg: 27 uM Avg: 5.44 uM. Avg: 0.124 uM (x5,400 EC50) (x1,088 EC50) —_ ( x24.8 EC50) 1hr 4hr 24 hr www.cocrystalpharma.com
Slide 41: Cocrystal Influenza Program
Cocrystal Influenza Program * Influenza A PB2 lead, CC-42344: IND filing scheduled in 2018 + Influenza A/B PA: Discovery stage Cap-binding Domain (PB2) PA a PB1 PB2 a www.cocrystalpharma.com
Slide 42: Polymerase Complex (PB2:PA:PB1) Is Essential For Influenza Viral Replication
Polymerase Complex (PB2:PA:pb1) Is Essential For Influenza Viral Replication « Polymerase: cap binding (PB2) + endonuclease (PA) + polymerase (PB1) retook f ' Orrnrw MAA chimeric viral mRNA snatching’ a 4 www.cocrystalpharma.com
Slide 43: CC-42344 Met Preclinical Lead Selection Criteria
CC-42344 Met Preclinical Lead Selection Criteria Properties Selection criteria Pharmacological properties Good antiviral activity (EC50, single digit nanomlar) High-affinity binding to a highly conserved drug binding site Broad spectrum against seasonal and pandemic influenza trains Excellent antivi
Slide 44: Approved Influenza Antivirals
Approved Influenza Antivirals ral Developer MOA/Dosi Oseltamivir Gilead/ Oral neuraminidase (Tamiflu) Genentech Inhibitor/75 mg bid for five days Zanamivir Biota/ Inhaled neuraminidase inhibitor/ (Relenza) GSK 5 mg inhalation bid for five days Peramivir Biocryst/ A single-dose intravenous (Rapivab)
Slide 45: Influenza Clinical Landscape
Influenza Clinical Landscape: CC-42344 Can Be Developed For Inhalation, IV, and Oral * Key players: J&J (PB2 and PA) AND Shionogji/Roche (PA) * Other companies: Merck, Gilead, and Novartis Antiviral Developer Route Stage MOAI/indication Oral Pimodivir PB2 inhibitor/ (VX-787 or JNJ-636233872) se) (eo
Slide 46: In Vitro Potency Comparison: Cocrystal's Leads vs VX-787
In Vitro Potency Comparison: Cocrystal’s Leads vs VX-787 Cocrystal J&J Preclinical = — Cocrystal’s backup PB2 in -> Lead Infl 42344 42343 42487 42500 42530 42534 etd EC50nM EC50nM EC50nM EC50nM EC50nM EC50 nM Pandemic H1N1 Influenza A/CA/07/2009 24 0.12 5 0.9 26 ND ND H1N1 Influenza PR/8/34. 1 1 1.2
Slide 47: CC-42344 Shows Broad Spectrum Antiviral Activity Against Pandemic Influenza Strains
CC-42344 Shows Broad Spectrum Antiviral Activity Against Pandemic Influenza Strains Influenza Seroty, Strain cena peeves HSN1 Duck/MN/1524/81 8.6 0.07 H5N1- Amantadine resistant Duck/MN/1524/81 <3.2 <3.2 HSN1 Gull/PA/4175/83 45 0.17 HSN1 Hong Kong/213/2003 <3.2 <3.2 HSN1 Thailand/16/2004 <3.2 —32 HS
Slide 48: CC-42344 Exhibits Greater Affinity Than VX-787
CC-42344 Exhibits Greater Affinity Than VX-787 * Highly sensitive biophysical method (SPR technology) was applied to determine the affinity (Kd) of CC-42344 to the PB2 protein © [CDiprost- Ligand 7 =a = Compound ID Kp elt Cows] —031s00m VX-787 37.4nM il CC-42344 9.90 nM i * Bn 7 r O 100 200 300 “400