Cocrystal Pharma, Inc. · NASDAQ:COCP
Cocrystal Pharma — 2025 International Calicivirus Conference
25 slides from Cocrystal Pharma's conference presentation, Sep 11, 2025
Slide 1: Oral Direct-Acting Antiviral CDI-988 for Norovirus Infection Prevention and Treatment
Oral Direct-Acting Antiviral CDI-988 for Norovirus Infection Prevention and Treatment: Mechanism of Action and Phase 1 Study Results 2025 International Calicivirus Conference September 11, 2025 Sam Lee, PhD President and co-CEO slee@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 our development pipeline; our technology platform’s ability to produce viable drug candidates at reduced development timelines and co
Slide 3: About Cocrystal Pharma
About Cocrystal Pharma ▪ Advancing programs in direct-acting small molecule antivirals • Norovirus • Influenza Applying powerful, proprietary • Coronavirus drug discovery platform • Rhinovirus technology to develop first- and best-in-class broad-spectrum ▪ Drug candidates with clinically validated m
Slide 4: Norovirus Viral Gastroenteritis Represents Significant Unmet Need
Norovirus Viral Gastroenteritis Represents Significant Unmet Need No treatment or vaccines available 700M Cocrystal’s pan-viral protease Infections annually worldwide inhibitor, CDI-988: 7 Norovirus GII.4 pandemic • First-in-class antiviral for norovirus outbreaks Norovirus $60B • Potential for both
Slide 5: Proprietary Drug Discovery Platform Technology for Direct-Acting Antivirals
Proprietary Drug Discovery Platform Technology for Direct-Acting Antivirals Grow drug target Select drug Hit-to-lead Lead No treatment or vaccines available X-ray crystals binding pocket process optimization Direct-Acting Antiviral Drug Candidate (A) In-house screening: (B) Focus on highly conserved
Slide 6: Big Surge of Norovirus Outbreaks in 2024-2025 After COVID-19 Pandemic
Big Surge of Norovirus Outbreaks in 2024-2025 After COVID-19 Pandemic Norovirus GII.17 rapidly overtaken GII.4 as the leading cause of norovirus outbreaks, >70% GII.4 GII.17 COVID COVID https://www.eurosurveillance.org/content/10.2807/1560-7917.ES.2024.29.39.2400625 6 6 Confidential
Slide 7: Must-Do in Norovirus Antiviral Development: Broad-Spectrum Coverage
Must-Do in Norovirus Antiviral Development: Broad-Spectrum Coverage Against Newly Emerging and Drug-Resistant Strains ▪ Crystal structures of GII.4 and GII.17 proteases revealed approximately 90% amino acid identity (Green), including the protease active site (Yellow) ▪ The location of variable amin
Slide 8: High Resolution X-ray Data Further Confirmed Broad-Spectrum Coverage of CDI-988
High Resolution X-ray Data Further Confirmed Broad-Spectrum Coverage of CDI-988 X-ray crystal structures of norovirus proteases complexed with CDI-988 (1.2 – 1.9 Å) ) 8
Slide 9: In Vitro Potency Assessment of Norovirus Protease Inhibitor CDI-988
In Vitro Potency Assessment of Norovirus Protease Inhibitor CDI-988 Assay In Vitro Potency, IC50 M GI.1 (Norwalk) 0.06 Overlay norovirus protease crystal structures complexed with CDI-988 GI.2 (Southampton) 0.21 GII.1 (Hawaii) 0.03 GII.2 (Snow Mountain) 0.12 GII.3 (Texas 2004) 0.15 GII.3 (Saitama U
Slide 10: Comprehensive Safety Assessment Using Human 3-D Small Intestinal Tissue System
Comprehensive Safety Assessment Using Human 3-D Small Intestinal Tissue System (EpiIntestinalFT Model System) (A) TEER (Transepithelial Electrical 120 Resistance) Assay (B) MTT cell viability assay 120 Reference compound, 120.0 100 100 SN-38, causes GI 100.0 toxicity, causing % Viability (Relative t
Slide 11: CDI-988 Shows Favorable Safety and Functional Profiles in Human Small Intestinal Tissue
CDI-988 Shows Favorable Safety and Functional Profiles in Human Small Intestinal Tissue (A) TEER (Transepithelial Electrical Resistance) Assay (B) MTT cell viability assay SN-38 CDI-988 CDI-988 120 120.0 20 M 100 M 0.41 M 100 M 0.41 M 100 100.0 % Viability (Relative to NC) % TEER (Relative to N
Slide 12: CDI-988 Shows No Gastrointestinal Toxicity in Human Small Intestine Organoid System
CDI-988 Shows No Gastrointestinal Toxicity in Human Small Intestine Organoid System 12
Slide 13: Promising Pharmacological Property of CDI-988: Higher Exposure and Longer Half-Life
Promising Pharmacological Property of CDI-988: Significantly Higher Exposure and Longer Half-Life of Oral CDI-988 in Intestine and Stomach ▪ GI-targeted activity was one of the 100,000 norovirus lead selection criteria Drug concentration (ng/mL) 10,000 Stomach Stomach Small intestine ▪ Demonstrated
Slide 14: Phase 1 Study of First-in-Class Direct-Acting Oral Antiviral Protease Inhibitor CDI-988
Phase 1 Study of First-in-Class Direct-Acting Oral Antiviral Protease Inhibitor CDI-988 A Phase 1, Randomized, Double-Blinded, Placebo-Controlled, First-in-Human Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single-Ascending and Multiple-Ascending Doses of Oral CDI-988 in Healt
Slide 15: CDI-988 Phase 1 Study Conducted In Australia
CDI-988 Phase 1 Study Conducted In Australia 15
Slide 16: Oral Pan-viral Protease Inhibitor CDI-988 Showed Favorable Safety and Tolerability
Oral Pan-viral Protease Inhibitor CDI-988 Showed Favorable Safety and Tolerability ▪ Single-center, randomized, double-blind, placebo-controlled ▪ Single-ascending dose (SAD) and Multiple-ascending dose (MAD) cohorts ▪ Healthy adult volunteers (18 – 55 years old) ▪ Each cohort comprised 8 participan
Slide 17: Key Entry Criteria
Key Entry Criteria ▪ Healthy males and females ≥ 18 and ≤ 55 years ▪ Body weight ≥ 50 kg ▪ Body mass index ≥ 18 and ≤ 32 kg/m2 ▪ Non-pregnant, non-lactating ▪ Must abstain from alcohol or caffeine from 48 hours before study confinement through duration of study ▪ Must not have taken prescribed medic
Slide 18: Demographics of SAD and MAD cohorts
Demographics of SAD and MAD cohorts SAD Placebo MAD Placebo (N=36) (N=10) (N=36) (N=12) Age (Years) 29.5 32.5 30.0 30.6 Mean 27.6 27.4 28.3 29.6 Median 21-49 20-56 21-45 25-39 Range Male, n (%) 14 (39%) 2 (20%) 23 (64%) 7 (58%) Female 22 (61%) 8 (80%) 13 (36%) 5 (42%) Ethnicity, n (%) Hispanic or La
Slide 19: SAD Clinical Safety Summary (N=46)
SAD Clinical Safety Summary (N=46) ▪ All dose cohorts well tolerated (100mg to 1200mg) ▪ Safety profile • 100% of AEs were mild severity (CDI-988 (N=11, 100%) vs Placebo (N= 4, 100%)) • Only 7 treatment-related AEs across all dose cohorts (CDI-988 (N=5, 14%) vs Placebo (N=2, 20%)) • Most commonly oc
Slide 20: MAD Clinical Safety Summary (N=48)
MAD Clinical Safety Summary (N=48) ▪ All dose cohorts well tolerated (200mg to 1200mg) ▪ Safety profile • Total of 30 reported AEs (CDI-988 (N=19, 53%) vs Placebo (N=11, 92%)) • 26 of these were mild severity (CDI-988 (N=16, 84%) vs Placebo (N=10, 91%)) • 8 AEs of moderate severity (CDI-988 (N=4, 21
Slide 21: CDI-988 Demonstrates Strong Food Effect
CDI-988 Demonstrates Strong Food Effect ▪ High fat meal prior to dosing results in a 5-fold higher plasma exposure 400 mg Fed (single ascending dose) 400 mg Fasted (single ascending dose) Human pharmacokinetic evaluation of CDI-988 21 21
Slide 22: Topline Safety Data Summary and Next Steps
Topline Safety Data Summary and Next Steps SAD cohorts MAD cohorts Overall treatment-emergent AE (TEAE) rate Overall treatment-emergent (TEAE) rate ▪ 28% (10/36) in CDI-988 cohorts ▪ 53% (19/36) in CDI-988 cohorts ▪ 40% (4/10) in placebo subjects ▪ 92% (11/12) in placebo subjects Headache was the mo
Slide 23: 57 Years of Norovirus: Our Commitment to Prevention and Treatment Continues
57 Years of Norovirus: Our Commitment to Prevention and Treatment Continues 23 23 Confidential
Slide 24: Next Step: Pivotal Role for Human Challenge Studies in Norovirus Antiviral Development
Next Step: Pivotal Role for Human Challenge Studies in the Development of Norovirus Antiviral CDI-988 CDI-988 GII.2 SMV protease Cocrystal structure of GII.2 SMV protease:CDI-988 complex ▪ Cocrystal CDI-988 human challenge study using Snow Mountain Virus (GII.2) planned (Reference, Rouphael et al. J
Slide 25: Acknowledgements
I thank our collaborators, norovirus experts, scientists, clinical investigators, adult volunteers, the staff at the study centers and the research organizations, and my colleagues at Cocrystal Pharma
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