Hepion Pharmaceuticals, Inc. · OTC:HEPA
Hepion Pharmaceuticals — Investor Presentation (October 2023)
36 slides from Hepion Pharmaceuticals's investor presentation, October 2023
Slide 1: Rencofilstat (CRV431): A Novel Drug Candidate for NASH, Fibrosis, and HCC
N A S D A Q : HE PA Rencofilstat (CRV431): A Novel Drug Candidate for NASH, Fibrosis, and HCC Creating a Therapeutic Ecosystem October 2023 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: NASH
NASH Fibrotic Liver Disease Leading Indication for Rencofilstat 4
Slide 5: 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 Prostate Cancer Idiopathic Pulmonary Fibrosis Renal Fibrosis Cardiac Fibrosi
Slide 6: 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 7: Why Develop a Drug for NASH?
Why Develop a Drug for NASH? • No currently approved drugs for the treatment of NASH • Market for NASH is extremely large (estimated as ~20 million adults in U.S. alone) • NAFLD/NASH may be asymptomatic with no convenient diagnostic to identify subjects early in disease progression • Symptoms may on
Slide 8: NASH Therapeutic Strategies
NASH Therapeutic Strategies Multiple therapeutic agents targeting multiple stages of disease likely required WHY? • Generally poor response rates with drugs in development • Many pathologic mechanisms contribute to disease normal liver cells Most failed drugs and drugs in development are "metabolic"
Slide 9: NASH Drug Development Landscape Phase 2b and 3
NASH Drug Development Landscape Phase 2b and 3 9
Slide 10: Rencofilstat Mode of Action
Rencofilstat Mode of Action 10
Slide 11: Collagen - Core To Fibrotic Diseases
Collagen - Core To Fibrotic Diseases Excessive collagen production – universal to every fibrotic disease Fibrosis is an exaggerated “scarring” of tissue in response to many types of cellular injuries, chronic inflammation, & other tissue insults Fibrosis impairs normal organ structure & function
Slide 12: Rencofilstat Attenuates Collagen Synthesis by Inhibiting Cyclophilin B
Rencofilstat Attenuates Collagen Synthesis by Inhibiting Cyclophilin B Enzyme Activity - "normalizing" disease-associated fibrosis Cyclophilin B is one of the most active enzymes involved in the proper folding, synthesis, secretion, and eventual crosslinking of collagen that forms the fibrotic matri
Slide 13: Antifibrotic Activity Observed in Preclinical Models: Proof of Concept
Preclinical Models: Proof of Concept Cyclophilin Inhibition Produces Consistent Antifibrotic Effects Liver: 8 NASH-related models Human liver slices Antifibrotic Lung Chronic fibrosis Activity Acute injury Observed in Human lung slices Every Preclinical Kidney Model Acute injury Heart Skin
Slide 14: 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 15: Overview of Phase 1 Studies (Completed)
Overview of Phase 1 Studies (Completed) 15
Slide 16: Phase 1 Studies Completed – Demonstrated a Favorable Safety Profile
Phase 1 Studies Completed – Demonstrated a Favorable Safety Profile 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 fa
Slide 17: Overview of Phase 2a 'AMBITION' NASH Trial (Completed)
Overview of Phase 2a ‘AMBITION’ NASH Trial (Completed) 17
Slide 18: Phase 2a 'AMBITION' NASH Study: Safety, Tolerability, and Pharmacokinetics
Phase 2a ‘AMBITION’ NASH Study Safety, Tolerability, and Pharmacokinetics 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 All Endpoints Were Met Study Design • F
Slide 19: Phase 2 'ALTITUDE-NASH' Trial (Clinically Complete)
Phase 2 ‘ALTITUDE-NASH’ Trial (Clinically Complete) 19
Slide 20: Phase 2 'ALTITUDE-NASH' Safety and Liver Function
Phase 2 ‘ALTITUDE-NASH’ (Liver Function Trial) Safety and Liver Function A Phase 2, Randomized, Multi-Center, Open-Label Study to Evaluate the Safety & Efficacy of Rencofilstat in Adult Subjects with NASH Stage 3 Fibrosis Baseline Characteristics Study Design 75 mg 150 mg 225 mg rencofilstat rencofi
Slide 21: Phase 2 'ALTITUDE-NASH' HepQuant SHUNT Test Results
Phase 2 ‘ALTITUDE-NASH’ HepQuant SHUNT Test Results Functionally Impaired Subjects# All Subjects with Paired Data (n=61) with Paired Data (n=34) All Doses 75 mg 150 mg 225 mg All Doses 75 mg 150 mg 225 mg % of Subjects with a greater than 2-point Decrease in DSI % of Subjects with a greater than 2-p
Slide 22: Phase 2 'ALTITUDE-NASH' Key NASH Non-Invasive Markers Results
Phase 2 ‘ALTITUDE-NASH’ Key NASH Non-Invasive Markers (NIMs) Results All Subjects Subjects with Baseline ProC3 ≥ 37.5 ng/ml % Change From Baseline % Change From Baseline 75 mg 150 mg 225 mg 75 mg 150 mg 225 mg rencofilstat rencofilstat rencofilstat rencofilstat rencofilstat rencofilstat n=23 n=21 n=
Slide 23: Phase 2b 'ASCEND-NASH' Trial (Enrolling)
Phase 2b ‘ASCEND-NASH’ Trial (Enrolling) 23
Slide 24: Phase 2b ASCEND-NASH Biopsy Trial
Phase 2b ASCEND-NASH Biopsy Trial A Phase 2b, Randomized, Multi-Center, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy And Safety of Rencofilstat in Adult Subjects with HASH and Advanced Liver Fibrosis Study Design Efficacy Endpoints Rencofilstat 75 mg qd (n=84) Primary: RANDOMIZED
Slide 25: Hepion's Proprietary Artificial Intelligence
Hepion’s Proprietary Artificial Intelligence 25
Slide 26: The Hepion AI-POWR Proprietary Clinical Process
The Hepion AI-POWR Proprietary Clinical Process Right Patient All Data Right Dose Generative Neural Net INPUT OUTPUT Deep Convoluted Neural Net 26
Slide 27: The Hepion AI-POWR Proprietary Clinical Process
The Hepion AI-POWR Proprietary Clinical Process AI-POWR • Biomarkers • Responder Identification PKPD/QSP/PBPK • Digital Biomarkers • Patient Selection • Phase III Enrichment + Multi-omics Pre-Clinical Data • Eliminate Biopsy + QoL • Simulation • Predict Clinical Outcome Informs AI/ML • Patient Spe
Slide 28: Illustration of Hepion's AI-POWR for Rencofilstat (RCF)
Illustration of Hepion’s AI-POWR™ for Rencofilstat (RCF) *Genes (number) Predictive Genes (number) AUROC Comment 1733 Statistically Highly Predictive for 25 0.97 Significant ProC3 Response *Key genes identified demonstrate RCF – CypA and B interaction in NASH subjects Lipids (number) Predictive Lipi
Slide 29: Phase 2a HCC Trial (Pending)
Phase 2a HCC Trial (Pending) 29
Slide 30: 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 31: Intellectual Property
2° VEPION Intellectual Property
Slide 32: Intellectual Property Position
Intellectual Property Position Long Patent Life with Patent Term Extensions (PTE) Family Status Notes December 2031 Expiry Composition of Matter 54 US & International Issued Patents (December 2036 Expiry with PTE) Assuming 2028 and 2029 NDA submission and approval Composition of Matter Provisional F
Slide 33: Summary
2° VEPION Summary
Slide 34: Two Value Drivers; Financials
Two Tackling Fibrotic Diseases Value Drivers • Rencofilstat, once-daily oral, targeting key drivers of pathology, tested in over 425 subjects • Two Phase 2 NASH trials: • ALTITUDE-NASH – Clinically Complete • ASCEND-NASH Phase 2b – Recruiting • Upcoming Phase 2 for HCC • Developing companion A.I. fo
Slide 35: 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) Robert Foster, PharmD, PhD Daren Ure, PhD CEO CSO Founded Isotechnika (Aurinia) i
Slide 36: 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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