Cocrystal Pharma, Inc. · NASDAQ:COCP
Cocrystal Pharma — Investor Presentation (April 2018)
48 slides from Cocrystal Pharma's investor presentation, Apr 9, 2018
Slide 1: Corporate Presentation April 2018
C#CRYSTAL Antiviral Therapies PHARMA “INC Corporate Presentation April 2018 NASDAQ: COCP. ‘www.cocrystalpharma.com
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation contains forward-looking statements, the anticipated timing of our drug development programs, including near-term milestones, and anticipated completion or initiation of studies, IND filings, and opportunities in the hepatitis C and influenza antiviral ma
Slide 3: Corporate Overview
Corporate Overview Clinical Stage Antiviral Company Hepatitis Wholly Owned Product Portfolio Influenza Norovirus Pi ietary Drug Di: Platf roprietary Drug Discovery Platform Gastibantentts
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 FDA i
Slide 5: Robust Development Pipeline
Robust Development Pipeline Hepatitis C (Hcv) Discovery Preclinical cc-31244 (Pan-genotypic NSSB NNI) cc-2850 (Pan-genotypic NSSB Nuc) cc-2069 (Pan-genotypic NSSA inhibitor) Influenza cc-42344 (Influenza A PB2 inhibitor) Influenza A/B inhibitor Norovirus Noro Polymerase Inhibitor www.cocrystalpharma
Slide 6: Near-Term Milestones Expected to Drive Value
Near-Term Milestones Expected to Drive Value HCV CC-31244: ¥ Filed IND with FDA ¥ Received IND clearance from FDA for Phase 2a study HCV CC-31244: + Commence patient enrollment in Phase 2a study + Commence patient dosing in Phase 2a study HCV CC-31244: + Complete Phase 2a study dosing Influenza CC-4
Slide 7: Seasoned Management Team
Seasoned Management Team . - e © ope Gary Wilcox, Ph.D. os (CGialis Vice Chairman and Chief Executive Officer i< tadalafil Over 35 years of executive biotech leadership experience and played a key role in the development of Cialis ‘OM. IA UCLA Sam Lee, Ph.D. President = . e o~ 20 years of anti-infec
Slide 8: Board of Directors
Board of Directors Raymond F. Schinazi, Ph.D. Chairman Frances Winship Walters Professor of Pediatrics, Director, Laboratory of Biochemical Pharmacology and Director, HIV Cure Scientific Working Group at Emory University Gary Wilcox, Ph.D. Vice Chairman and Chief Executive Officer Vice Chairman and
Slide 9: Cocrystal Technology
Cocrystal Technology Nobel Prize Winning Technology i | Near atomic resolution, “ X-ray quality crystal production TER © Drug pocket selection a « Hit-to-lead process ee « Lead optimization Sos Drug candidates www.cocrystal
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 CC-31244 HCV NS5B polymerase Proven track record for broad spectrum antiviral leads
Slide 11: Hepatitis C Treatment Market Share
Hepatitis C Treatment Market Share 2017 Annual Sales, $12.69 Billion Gilead $9.14 B = Gilead (Harvoni, Epclusa, Sovaldi, Vosevi) mJ&J (Olysio) mAbbVie (Viekira, Mavyret) m Merck (Zepatier) Source: 2017 Form 10-K
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 NS5A Inhibitor =) CC-31244 SSE NN oe NS5B Nuc ==> 2 or 3 broad spectrum =) drugs for shorter therapy NS3 Protease Inhibitor
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 NS5B polymerase inhibitor - Developed by Cocrystal’s proprietary structure-based discovery platform - High barrier to drug resistance - Effecti
Slide 15: Phase 1a Study Completed
Phase 1a Study Completed 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 laboratory abnormalities 20m 50mg | 100mg 200mg* | 400m Single ascending doses 3 é 5 B é (eomipiete) = N = 40; 30 CC-31244, 10 plac
Slide 16: Phase 1b Study Completed
Phase 1b Study Completed 200 mg BID x 7 days Status N=4;3CC-31244,1 placebo Complete 400 mg QD x 7 days Status N=5;4CC-31244,1 placebo Complete Single doses QD or BID x7 days 4 HCV GT1 HCV RNA2 5 log, .lU/ml Naive 600 mg QD x 7 days Status N=5;4CC-31244,1 placebo Complete Endpoints * Efficacy: chang
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 (CC-31244 at 400 mg QD or 200 mg BID x7 days) i CC-31244 No CC-31244 400 mg QD 200 mg BID HCV RNA IUimL (log 10
Slide 18: Best-in-Class Potential of Any NNI
Best-in-Class Potential of Any NNI Dose Treatment Mislead Genoty, (mg) Duration (days) reduction (Log,,lU/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) 15 (* approved DAA)
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
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 @rseN] C@CRYSTAL Bae InterX * Combines synergistic drug discovery platforms developed by Cocrystal- HitGen-InterxX SF ¢ Break through hit-to-lead process * High quality novel leads will be developed www.cocrystalpharma.
Slide 26: Selected Value Indicators Suggest Potential Significant Upside
Selected Value Indicators Suggest Potential Significant Upside Company Name Ticker Market Cap (M)* Overview Status . Develops medicines to treat intractable deste by silencing the 2 Phase 1 Beem es Herne ro ee Se6 genes that cause them 6 Preclinical 1 hase2 Biopharmacevtcal company developing cure f
Slide 27: Financial Snapshot
Financial Snapshot — Nasdaq: COCP $145MM 24.4MM 7.7K Market cap* Common shares 3 month average outstanding daily volume* rice of $5.95 per share www.cocrystalpharma.com
Slide 28: COCP Capitalization Table
COCP Capitalization Table capitalization Table (As of 12/31/2017) # of Shares ore, Diluted {Common Shares Outstanding (Directors & Officers) 14,420,436 56.68% ‘ommon Shares Outstanding (Other) 9,854,058 38.73% Warrants 209,166 $15.00 $3,137,490 qos ‘Stock Options 711,308 $8.39 $5,967,874 Aen \Conver
Slide 29: Near-Term Milestones Expected to Drive Value
Near-Term Milestones Expected to Drive Value HCV CC-31244: ¥ Filed IND with FDA ¥ Received IND clearance from FDA for Phase 2a study HCV CC-31244: + Commence patient enrollment in Phase 2a study + Commence patient dosing in Phase 2a study HCV CC-31244: + Complete Phase 2a study dosing Influenza CC-4
Slide 30: Thank You!
C#CRYSTAL Thank You! NASDAQ: COCP. ‘www.cocrystalpharma.com
Slide 31: C#CRYSTAL
C#CRYSTAL PHARMA,INC NASDAQ: COCP. ‘www.cocrystalpharma.com
Slide 32: Appendix
C#CRYSTAL 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, Chairman of 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
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 among HCV genotypes| \ I a PA GDD Motif a, Cc @ Cc R Y Ss 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 Geen CDI-31244 ECs, Sofosbuvir ECs, EC,,, mM Fold change EC,,, mM fold change 1b 0.005 1.0 0.042 1.0 ta 0.009 1.8 0.034 0.8 2b 0.026 5.2 0.028 0.66 3a 0.011 22 0.14 3.2 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 IC,, fold change NNI IC. fold change cC-31244 <5 HCV-796 >1,500 HCV-796 sso 1600 1400 . . ‘200 Gres Drug resistance variants 1000 Backup Leads [Bl S305T (NNI-4) mi yy uy I n316y (NNI-4) ual Hh L419 (NNI-2) ‘eo |
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 GT‘1b replicons containing NS5B variants identified by CC-31244 resistant colony selection GT1b CA45F/S549G ECs HM HCV replicon
Slide 39: CC-31244 Binding Mode
CC-31244 Binding Mode: GT1b Drug Resistant NS5B C445F RAHA (4) ct2b wild type | 4 | (B) GT1b C445F Zen]
Slide 40: CC-31244 Exhibits Excellent Liver Targeting
CC-31244 Exhibits Excellent Liver Targeting Rat liver study Avg: 27 pM. Avg: 5.44 uM. Avg: 0.124 uM. (x5,400 EC50) (x1,088EC50) —( x24.8 EC50) om 1hr 4hr 24 hr 25000 CC-31244, ng/mL
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 Endonucle Cap-binding Domain (P, Domain (PB2)
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) ret eons Orne MA www.cocrystalpharma.
Slide 43: CC-42344 Met Preclinical Lead Selection Criteria
CC-42344 Met Preclinical Lead Selection Criteria Properties Selection criteria Pharmacological properties. Q_ Good antiviral activity (EC5O, single digit nanomlar) High-affinity binding to a highly conserved drug binding site Broad spectrum against seasonal and pandemic influenza trains Excellent an
Slide 44: Approved Influenza Antivirals
Approved Influenza Antivirals Anti I veloper OA/Dosing 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 Shionogi/Roche (PA) * Other companies: Merck, Gilead, and Novartis Antiviral Developer Route Stage MOA/Indication Oral Pimodivir PB2 inhibitor/ (VX-787 or JNJ-636233872) Hee (600
Slide 46: In Vitro Potency Comparison: Cocrystal's Leads vs VX-787
In Vitro Potency Comparison: Cocrystal’s Leads vs VX-787 Cocrystal J&d Preclinical = Cocrystal’s backup PB2 inhibitors =) Lead VX-787 42344 42343 42487 42500 42530-42534 Influenza strai EC50nM EC50nM EC50 Ec50nM EC50nM EC50nM EC50nM Pandemic H1N1 Influenza A/CA/07/2009 24 0.12 5 0.9 27 ND ND H1N1 In
Slide 47: CC-42344 Shows Broad Spectrum Antiviral Activity Against Pandemic Influenza Strains
CC-42344 Shows Broad Spectrum Antiviral Activity Against Pandemic Influenza Strains Strain CC-42344 EC50, nM HSN1 Duck/MN/1524/81 86 H5N1- Amantadine resistant Duck/MN/1524/81 <3.2 HSN1 Gull/PA/4175/83 45 HSN1 Hong Kong/213/2003 <3.2 HSN1 Thailand/16/2004 <3.2 HSN1 AIVN/1194/2004 1.3 66 H7N7 Netherl
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 TEST UganaT a ne2 [Gees] site ns Compound ID Ky 20 VX-787 37.4nM 2° CC-42344 9.90 nM - En ° cr ) Time(s) ‘Sample H1N1 Sensor
Cocrystal Pharma — Investor Presentation (April 2018) on 6ix