Hepion Pharmaceuticals, Inc. · OTC:HEPA

Hepion Pharmaceuticals — Investor Presentation (March 2020)

21 slides from Hepion Pharmaceuticals's investor presentation, Mar 9, 2020

  1. Slide 1: Corporate Presentation 2020

    1 C O R P O R AT E P R E S E N TAT I O N • 2 0 2 0 Nasdaq: HEPA Nasdaq: HEPA

  2. Slide 2: Forward-Looking Statements

    Forward-Looking Statements 2 This presentation may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such forward-looking statements are characterized by future or conditional verbs such as “may,

  3. Slide 3: Corporate Overview

    Corporate Over view 3 Lead Asset CRV431: Novel, high-potency, cyclophilin inhibitor that targets multiple stages of liver disease, including NASH  ANTI-FIBROTIC, ANTI-VIRAL, AND ANTI-CANCER PROPERTIES (PLEIOTROPIC)  STRONG PRECLINICAL PROOF OF CONCEPT  STRONG SAFETY PROFILE IN PRECLINICAL AND PHA

  4. Slide 4: Development Phase

    Development Phase 4 Discovery Phase 1 Phase 3 P ROJ EC T E D CRV431 L AU N C H Preclinical Phase 2 2026 Nasdaq: HEPA

  5. Slide 5: CRV431 in NASH

    CRV431 in NASH 5 Cyclophilin Inhibitors Target Multiple Liver Disease Stages INJURY/STEATOSIS INFLAMMATION FIBROSIS CIRRHOSIS/CANCER BLOCK CANCER CELL ANTIVIRAL ACTIVITY SUPPRESS REDUCE COLLAGEN ADAPTATION TO HYPOXIA (HBV, HCV, HDV, HIV-1) PRO-INFLAMMATORY PRODUCTION FROM HEPATIC PATHWAYS MEDIATED B

  6. Slide 6: Anti-Fibrotic Activity in NASH Models

    Anti-Fibrotic Activity in NASH Models 6 STAM Mouse Model “Friedman” Mouse Model (HIGH FAT DIET + STREPTOZOTOCIN) (WESTERN DIET + CCl4) 3 - 11 WEEKS ORAL CRV431 TREATMENT 6 WEEKS ORAL CRV431 TREATMENT P≤0.0001 p < 0 .0 1 10 p = 0 .0 3 p = 0 .0 1 Sirius Red (% of area) 4 6 S ir iu s r e d - p o s itiv

  7. Slide 7: Anti-Fibrotic Activity in Liver Toxin Models

    Anti-Fibrotic Activity in Liver Toxin Models 7 RAT THIOACETAMIDE MODEL CARBON TETRACHLORIDE MOUSE MODEL 9 WEEKS THIOACETAMIDE 6 WEEKS CCL4 9 WEEKS VEHICLE OR CRV431 6 WEEKS VEHICLE, CRV431, OR OBETICHOLIC ACID (OCA) Fibrosis (% Sirius Red) Fibrosis (% Sirius Red) Fibrosis score P = 0.008 15 P=0.008

  8. Slide 8: Anti-Cancer Activity in Late-Stage NASH Model

    Anti-Cancer Activity in Late-Stage NASH Model 8 STAM Mouse Model Number of Tumor Nodules (STREPTOZOTOCIN + HIGH FAT DIET) p = 0.05 15 Number of nodules per liver 10 Score 0 5 10 WEEKS ORAL CRV431 TREATMENT 0 Vehicle CRV431 p = 0.014 44% REDUCTION IN TUMOR NUMBER 5 Sirius red-positive fibrosis Score

  9. Slide 9: CRV431 on Human Precision Cut Liver Slices

    CRV431 on Human Precision Cut Liver Slices ( F i b ro F i n d ) 9 CRV431 Decreases Tissue Fibrosis in Human Liver Samples PICROSIRIUS RED STAINING OF FIBROTIC COLLAGEN SUMMARY  Precision cut liver slices from 4 human donors had baseline % Normalized to Nonstimulated and Stimulated 1.3 - 9.5% Sirius

  10. Slide 10: Anti-Fibrotic Mechanisms of Action

    Anti-Fibrotic Mechanisms of Action 10 CRV431 is proposed to decrease fibrosis by affecting two processes in hepatic stellate cells, the primary, collagen-producing cell type in hepatic fibrosis: • decrease expression of fibrosis-related genes • decrease cyclophilin B-dependent collagen synthesis and

  11. Slide 11: Summary of Nonclinical Anti-fibrotic Activities

    Summary of Nonclinical Anti-fibrotic Activities 11 Treatment Fibrosis Reduction Species Model Location Other CRV431 Effects Duration (% Sirius Red) Friedman NASH model Mice Scripps (USA) 6 weeks 82%  Weight gain  (CCl4 + Western diet) STAM NASH model Mice Stelic (Japan) 3 weeks 57%  none (strepto

  12. Slide 12: Clinical Pharmacology: Single Ascending Dose Study

    Clinical Pharmacology 12 Single Ascending Dose (SAD) Study: (CTRV-CRV431-101) → TO EVALUATE THE SAFETY AND → RANDOMIZED, PARTIALLY TOLERABILITY OF SINGLE ORAL DOSES OF BLINDED, PLACEBO-CONTROLLED, OBJECTIVES CRV431 AT INCREASING DOSE LEVELS DESIGN SEQUENTIAL SAD STUDY IN HEALTHY VOLUNTEERS → TO EVAL

  13. Slide 13: Clinical Pharmacology: CRV431

    Clinical Pharmacology: CRV431 13 Mean Pharmacokinetic Parameters: Non-Compartment Model AUC0-inf, Tmax, h Cmax, ng/mL t½, h Dose (range) (SD) ng*h/mL (SD) (SD) 75 mg 4 (2-10) 334±106 20,917±3,780 73.6±15.2 225 mg 1.3 (1-2) 1,368±221 84,422±32,373 97.3±18.4 375 mg 1.5 (1-3) 1,488±176 103,833±30,916 1

  14. Slide 14: Clinical Pharmacology: Safety Profile and Conclusions

    Clinical Pharmacology 14 Safety Profile and Conclusions: SAD Study → NO SAEs WERE REPORTED IN THE SAD STUDY SAFETY → AEs FROM THE SAD STUDY HAVE BEEN MILD TO MODERATE AND MOSTLY UNRELATED TO PROFILE STUDY DRUG → THERE WERE NO GRADE 3 OR GRADE 4 LABORATORY ABNORMALITIES → VITAL SIGNS AND ECGS WERE UN

  15. Slide 15: Clinical Pharmacology: Multiple Ascending Dose Study

    Clinical Pharmacology 15 Multiple Ascending Dose (MAD) Study (CRV431-101) → TO EVALUATE THE SAFETY AND TOLERABILITY OF MULTIPLE ORAL DOSES OF CRV431 AT → RANDOMIZED, INCREASING DOSE LEVELS OVER 28 DAYS SEQUENTIAL MAD OBJECTIVES DESIGN → TO EVALUATE MAXIMUM TOLERATED DOSE STUDY IN HEALTHY VOLUNTEERS

  16. Slide 16: Phase 2A Proof-of-Concept AMBITION Trial

    Clinical Pharmacology 16 Phase 2A Proof-of-Concept (CRV431-201, ‘AMBITION’ Trial) OBJECTIVES  TO EVALUATE THE SAFETY AND TOLERABILITY OF ONCE DAILY (QD) 150 MG DOSE OF CRV431 IN PRESUMED NONALCOHOLIC STEATOHEPATITIS (NASH) FIBROSIS STAGE 2 (F2)/FIBROSIS STAGE 3 (F3) SUBJECTS COMPARED TO PLACEBO CON

  17. Slide 17: Clinical Pharmacology: Where does the Drug Work?

    C l i n i c a l P h a r m a c o l o g y : W h e re d o e s t h e D r u g Wo r k ? 17 Physiological Based PK Modeling Bioinformatics: Gene-Gene Interactions  EXTENSIVE LIVER PARTITIONING  FAVORS THE USE OF CRV431 IN LIVER DISEASES  CONFIRMS ONCE DAILY DOSING AT DRUG TARGET SITES CRV431 Receptor Ge

  18. Slide 18: Bioinformatics: AI Predicting Clinical Outcomes

    Bioinformatics: Artificial Intelligence (AI) Predicting Clinical Outcomes 18 NASH Clinical Outcome: AI STEP 1: SHALLOW NEURAL NET: A SIMPLE EXAMPLE STEP 2: DEEP NEURAL NET: IN-TRAINING WITH ENHANCED PROPRIETARY METHODS NEURAL NETWORK ARCHITECTURE B1 B2 Drug I1  Black lines indicate positive weights

  19. Slide 19: Clinical Timelines

    19 H1 2020 • DATA FROM CLINICAL 28-DAY STUDY, ORAL CRV431 MULTIPLE ASCENDING DOSE (MAD), ONCE DAILY • INITIATE PHASE 2 NASH BIOMARKER PILOT STUDY, 1-MONTH CRV431 REPEAT DOSE Q3 CLINICAL • DATA FROM PHASE 2 NASH PILOT TRIAL TIMELINES (‘AMBITION’), 1 MONTH, CRV431 REPEAT DOSE • BIOINFORMATIC BIOMARKER

  20. Slide 20: Non-Clinical Events

    20 2020 Q1 DATA FROM PRECISION CUT LIVER SLICES, UK • INITIATE CHRONIC TOXICOLOGY IN RAT AND MONKEY • CONTINUE FIBROSIS STUDIES Q2 NON-CLINICAL • DATA FROM KIDNEY FIBROSIS MODEL • BIOINFORMATIC ANALYSES OF PREVIOUS EVENTS ANIMAL MODELS H2 • DATA FROM CHRONIC TOXICOLOGY, RAT AND MONKEY • DATA FROM D

  21. Slide 21: Thank You

    21 THANK YOU Corporate Contact: Investor Relations: Robert Foster Stephen Kilmer rfoster@hepionpharma.com skilmer@hepionpharma.com Tel: 646.274.3580 Nasdaq: HEPA

Hepion Pharmaceuticals — Investor Presentation (March 2020) on 6ix