Hepion Pharmaceuticals, Inc. · OTC:HEPA
Hepion Pharmaceuticals — Investor Presentation (November 2022)
34 slides from Hepion Pharmaceuticals's investor presentation, November 2022
Slide 1: Rencofilstat (CRV431): A Novel Drug Candidate for Fibrotic Diseases
N A S D A Q : H E PA Rencofilstat (CRV431): A Novel Drug Candidate for Fibrotic Diseases, including NASH and HCC Creating a Therapeutic Ecosystem November 2022 1 1
Slide 2: Forward-Looking Statements
Forward-Looking Statements 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,”
Slide 3: Rencofilstat Highlights
Rencofilstat Highlights • Novel mechanism - cyclophilin inhibition • Once-daily, oral medication – soft gel capsules Rencofilstat • Collagen-specific anti-fibrotic Anti-Fibrotic Drug • Targets key pathologies including fibrosis, inflammation, Candidate cell injury • All clinical trials to date show
Slide 4: Landscape of Clinical Antifibrotic Drug Development
Landscape of Clinical Antifibrotic Drug Development Opportunity for Collagen-Targeting Therapeutics • Despite ≈300 clinical trials, the only approved antifibrotic drugs are pirfenidone and nintedanib • Most anti-fibrotic agents have targeted upstream drivers of fibrogenesis (e.g., TGFβ) or disease-s
Slide 5: Excessive Collagen Production: Universal to Every Fibrotic Disease
Excessive Collagen Production: Universal to Every Fibrotic Disease Supports a Unique Poly-Indication Drug Development Strategy • Fibrosis is an exaggerated “scarring” of tissue in response to many types of cellular injuries, chronic inflammation, & other tissue insults • Fibrosis impairs normal orga
Slide 6: Cyclophilin B Participant in Fibrotic Collagen Production
Fibrotic Collagen Production Cyclophilin B (Cyp B; PPIB) – Participant in several stages of collagen synthesis and fibrotic scar formation Denotes CypB participation 6 Am J Respir Cell Mol Biol 2022 Apr;66(4):363-381. Fighting the Fiber: Targeting Collagen in Lung Fibrosis. Claudia A Staab-Weijnitz
Slide 7: Rencofilstat Inhibits Three Primary Cyclophilins
Rencofilstat Inhibits Three Primary Cyclophilins Hitting the Right Targets – Fibrosis, Inflammation, and Cell Injury/Death Rencofilstat blocks Cyclophilin B binding to pro-collagen fibrils ANTI-FIBROTIC and thereby decreases formation and secretion of fibrotic collagen Rencofilstat blocks Cyclophili
Slide 8: Antifibrotic Activity Observed in Preclinical Models
Preclinical Models Consistent Antifibrotic Findings Liver • 8 NASH-related models • Human liver slices Lung Antifibrotic • Chronic fibrosis Activity • Acute injury • Human lung slices Observed in Every Preclinical Kidney • Acute injury Model Heart Skin 8
Slide 9: Summary of Rencofilstat Programs – Pipeline within a Product
Summary of Rencofilstat Programs – ‘Pipeline within a Product’ Program Pre-Clinical Phase 1* Phase 2 Phase 3 Biopsy NASH Fast Track Designation Liver Function Hepatocellular Carcinoma Orphan Drug Designation Multiple Myeloma Idiopathic Pulmonary Fibrosis Renal Fibrosis Cardiac Fibrosis 9
Slide 10: NASH Fibrotic Liver Disease
NASH Fibrotic Liver Disease 10
Slide 11: The Need and Opportunity: NASH is Driving a Healthcare Crisis
The Need and Opportunity NASH is Driving a Healthcare Crisis NAFLD NASH HCC non-alcoholic fatty liver disease non-alcoholic steatohepatitis hepatocellular carcinoma “Fatty liver” disease A more severe form of NAFLD, Most prevalent type (90%) of liver associated with obesity, with inflammation and li
Slide 12: Challenges to Achieve Development and Commercial Success
Challenges to Achieve Development and Commercial Success A Comprehensive Approach to Provide Solutions 1 Need for Disease Modifying Drugs • Many molecules in development, majority targeting metabolic disease (liver fat) NASH • Need to address advanced fibrosis • No FDA drugs approved 2 Need for Comp
Slide 13: Overview of Phase 1 Studies
Overview of Phase 1 Studies (completed) 13
Slide 14: Phase 1 Studies Completed – Safe and Well Tolerated
Phase 1 Studies Completed – Safe and Well Tolerated Key Findings • No serious adverse events • No adverse events with dose response • Effective t1/2 ~ 30 hours Single Multiple Drug-Drug • Tmaxss ~ 1 – 2 hours Ascending Ascending Interaction Food Effect Dose Dose (midazolam & (high fat meal) • Ketoco
Slide 15: Overview of Phase 2a AMBITION NASH Trial
Overview of Phase 2a ‘AMBITION’ NASH Trial (completed) 15
Slide 16: Phase 2a AMBITION NASH Study: Safety, Tolerability, and Pharmacokinetics
Phase 2a ‘AMBITION’ NASH Study Safety, Tolerability, and Pharmacokinetics AMBITION: A Phase 2a, Multi-center, Single-Blind, Placebo-Controlled, Proof of Concept Study to Evaluate the Safety & Tolerability of Rencofilstat Dosed Once Daily in NASH Induced F2 & F3 Subjects Placebo (n=6) OFF-TREATMENT P
Slide 17: Phase 2a AMBITION NASH Study: All Primary Endpoints Met
Phase 2a ‘AMBITION’ NASH Study All Primary Endpoints Met Rencofilstat is safe and well-tolerated Efficacy signals were observed in only 28 days including: • Reduction in ALT (marker of inflammation & fibrosis) • Reduction in Pro-C3 (marker of fibrosis) • Downregulation of collagen genes • Upregulati
Slide 18: Hepion's Proprietary Artificial Intelligence
Hepion’s Proprietary Artificial Intelligence 18
Slide 19: Hepion's Proprietary State-of-the-Art Artificial Intelligence
Hepion’s Proprietary State-of-the-Art Artificial Intelligence Developing a Panel for Clinical Development and Commercialization MACHINE LEARNING Facilitates: • Improved drug target selection AI-POWR™ • Clinical study design ARTIFICIAL INTELLIGENCE • a priori responder analysis Designed to elucidate
Slide 20: AI-POWR Applied to the Phase 2a Program
AI-POWRTM Applied to the Phase 2a Program Guiding Hepion’s Future Development ALT A1c AST Chol WBC Sex Age BMI Using machine learning on data Baso Trigs from the Phase 2a, Hepion was CPK Glu Conc able to identify subjects more likely For future Phase 3 trials Creat PLT to respond to rencofilstat wit
Slide 21: Fibrosis-Associated Gene Network Observed in Phase 2a Blood Samples
Fibrosis-Associated Gene Network Observed in Phase 2a Blood Samples Supports Rencofilstat Antifibrotic Efficacy in 28-Day Study MM P23 B A1B G SDC 2 Rencofilstat impacted gene COCH FBN SERP 2 expression related to ACHE EMID1 F5 LOXL1 INB8 biosynthesis, remodeling, and BCHE N1D1 ICAM1 COL9A2 degradat
Slide 22: Phase 2b ASCEND-NASH Trial
Phase 2b ‘ASCEND-NASH’ Trial (in progress) 22
Slide 23: Phase 2b ASCEND-NASH (Biopsy Trial)
Phase 2b ASCEND-NASH (Biopsy Trial) Primary Objective: Evaluate the efficacy and safety of once-daily 75mg, 150 mg, and 225 mg doses of Rencofilstat compared to placebo in subjects with biopsy proven NASH and stage 2 liver fibrosis (F2) / stage 3 liver fibrosis (F3) Rencofilstat 75 mg qd (n=84) OFF-
Slide 24: Phase 2b ASCEND-NASH Endpoints
Phase 2b ASCEND-NASH Primary Efficacy Endpoint: Superiority of rencofilstat compared to placebo on liver histology at month 12 relative to the screening biopsy, by assessing the proportion of subjects with improvement in fibrosis by at least 1 stage (NASH CRN system) OR NASH resolution without worse
Slide 25: Phase 2 ALTITUDE-NASH Trial
Phase 2 ‘ALTITUDE-NASH’ Trial (in progress) 25
Slide 26: Phase 2 ALTITUDE-NASH (Liver Function Trial)
Phase 2 ‘ALTITUDE-NASH’ (Liver Function Trial) Primary Objective: Evaluate the change in hepatic function with once daily (QD) 75 mg, 150 mg, and 225 mg doses of rencofilstat in subjects with NASH F3 fibrosis using the HepQuant SHUNT test. Rencofilstat 75 mg qd (n=20) OFF- Endpoints: RANDOMIZED 1:1:
Slide 27: Phase 2 ALTITUDE-NASH Endpoints
Phase 2 ‘ALTITUDE-NASH’ (Liver Function Trial) Primary Efficacy Endpoint: Change from baseline in DSI score of subjects taking rencofilstat using HepQuant SHUNT Test, on Day -1, Day 60, and Day 120 Secondary Efficacy Endpoints: • Percent of subjects with a reduction in DSI score of >2 • Change from
Slide 28: Phase 2a HCC Trial
Phase 2a HCC Trial (upcoming) 28
Slide 29: Phase 2a: Advanced Hepatocellular Carcinoma (HCC)
PHASE 2a: Advanced Hepatocellular Carcinoma (HCC) Safety, Tolerability, Pharmacokinetics, and Efficacy A Phase 2a, Open-Label, Multi-Center, Simon 2-Stage Study to Assess Preliminary Efficacy, Safety, and Pharmacokinetics of 2 Dosage Levels in Advanced Metastatic Resistant or Refractory HCC Subjects
Slide 30: Summary
26 PIEPIEN Summary
Slide 31: Hepion's Approach to Developing an Ecosystem
Hepion’s Approach to Developing an Ecosystem Creating a Bundle to Support Rencofilstat’s Success Disease Modifying Drug Strategy: • Rencofilstat (disease modifying), efficacious, safe and well-tolerated • Companion Point-of-Care Diagnostic (e.g., Blood Panel) • A.I. and Multiomic Analyses Identify R
Slide 32: Two Value Drivers
Tackling Fibrotic Diseases Two Value Drivers • Rencofilstat, once-daily oral, targeting key drivers of pathology, tested in over 200 subjects • Two Phase 2 NASH trials underway • Upcoming Phase 2 for HCC • Developing companion A.I. for clinical development and commercialization strategy • Core scien
Slide 33: Experienced Team
Experienced Team Core R&D team has collectively > 120 yrs of experience with cyclophilin inhibition drug development, most notably voclosporin (Lupkynis®) for lupus nephritis while at Isotechnika (Aurinia, NASDAQ:AUPH) Launa Aspeslet, PhD Robert Foster, PharmD, PhD COO CEO Formerly COO of Isotechnik
Slide 34: Contact Us
CONTACT US Hepion Pharmaceuticals Inc. 399 Thornall Street, First Floor Edison, New Jersey, USA, 08837 Email: info@hepionpharma.com Phone: 732-902-4000 www.hepionpharma.com
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