Hepion Pharmaceuticals, Inc. · OTC:HEPA
Hepion Pharmaceuticals — Investor Presentation (December 2020)
23 slides from Hepion Pharmaceuticals's investor presentation, December 2020
Slide 1: AI-Driven Bioinformatics Platform to Accelerate Development of a Novel NASH Therapeutic
Our AI-Driven Bioinformatics Platform is Expected to Accelerate the Development of a Novel NASH Therapeutic and Create Pathways to Optimize Efficacy HEPA December 2020 Investor Presentation
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: The cost to develop one new drug is $2.6 Billion
The cost to develop one New Drug is $2.6 Billion: the approval FDA Rejects NASH Therapeutic, June 2020 rate for Drugs Entering clinical development is less than 12% Fails Phase 3 Trial, May 2020 Tufts Center for the Study of Drug Development, 2019 AI-driven bioinformatics has the potential to transf
Slide 4: Highlights
HIGHLIGHTS ▪ Clinical phase 2a NASH program currently underway TWO VALUE DRIVERS ▪ Artificial Intelligence and Smart Algorithms Platform (AI- POWR™) will help to identify and optimize responder AI-Driven, A Therapy for NASH with outcomes indications for several Bioinformatic Platform other condition
Slide 5: Pipeline
PIPELINE NDA THERAPY PROGRAM PRE-CLINICAL Phase 1* Phase 2 Phase 3 SUBMISSION NASH IND 142904 CRV431 205 COVID-19 pIND 151344 CRV431 206 HBV IND 1376787 Open to Partnerships *Separate Phase 1 programs were not conducted for each separate indication. The Phase 1 program was comprised of Single and Mu
Slide 6: About Us
ABOUT US We Have Developed an AI-Driven Bioinformatics Platform Which is Expected to Accelerate Cost-Effective Clinical Trials Study Design to Increase Response Rates p. 6
Slide 7: Bioinformatics Accelerates the Development of Our Own Therapies and Can Be Used as a Platform
Bioinformatics ACCELERATES the Development of Our OWN THERAPIES and can be used as a PLATFORM AI P O W R Supervised and unsupervised Precision medicine based on Omics, including genomics, proteomics, World, accessing world Response and clinical AI-enabled machine learning genetics, environment, and
Slide 8: Identifying Responders Increases Signal-to-Noise
Identifying Responders Increases “Signal-to-Noise” Supervised and Unsupervised Machine Learning Algorithms Model Inputs and Run Scenarios to Constantly Increase Response Rates AI-POWR™ Produces a Concentrated Set of Responders Which we Believe Will: ▪ Optimize to identify responders a priori ▪ Reduc
Slide 9: Components of AI-POWR
Components AI-POWR™ ACCESSING DATA Inputs AI DATA-PROCESSING DEFENDING OUTCOMES Are All Publicly Available, for everyone Is the Tough, and Proprietary part Are the Ultimate Goals of Bioinformatic AI Platforms. Big Data Access Scripting Algorithms / AI Demonstrate Improved Response We all have access
Slide 10: The Net Benefits of AI-Driven, Bioinformatic Trials and Study Design
THE NET BENEFITS of AI-Driven, Bioinformatic Trials and Study Design Anticipated Increase Trial Success Rates Shorten Trial Timelines Decrease Costs Benefits of AI- Phase I-III selection and biomarker Modeling results, adjusting variables Shortening intervals between trials verification reduce varia
Slide 11: A cyclophilin inhibitor in accelerated development using AI-POWR
a cyclophilin inhibitor in accelerated development using AI-POWRTM to produce anticipated market-leading efficacy rates to treat NASH
Slide 12: CRV431 for NASH
CRV431 for NASH THERAPY DISCOVERY PRE-CLINICAL Phase 1 Phase 2 Phase 3 NDA SUBMISSION Synthesized and Projected screened NDA Filing ~200 molecules 2025 Identified CRV431 as Single and Phase 2a lead molecule Multiple 'AMBITION' Clinical Ascending Trial Ongoing Dosing Completed Efficacy Models:
Slide 13: The Genesis of CRV431 / Why Target Cyclophilins
THE GENESIS OF CRV431 Theory Prediction and Experimentation Findings Modifying Cyclosporine A Modifications of Cyclosporine A can either eliminate or enhance Inhibition of each cyclophilin can greatly increase its immunosuppression. Our team’s past discovery, voclosporin, isoform produces distinct i
Slide 14: Patent Portfolio
Patent Portfolio Composition of Matter CRV431 Formulation Family Family International PCT Application 1 47 2 US Patent Application Issued Patents Nov 2019 Treatment of Fibrotic Disease in Anti-Fibrotic and Anti-Cancer Multiple Organs Activities Family Family US Provisional Patent Application 4 US Pr
Slide 15: CRV431 Mode of Action
CRV431 MODE OF ACTION Cyclophilin enzymes regulate the structure and activity of many proteins throughout the body Cyclophilin A (cytosol and secreted) Secreted from injured cells and acts as proinflammatory cytokine by binding to CD147. Binds to Cyclophilin A • Hijacked by viruses to assist in vira
Slide 16: The Development of CRV431 Using AI-POWR
The Development of CRV431 Using AI-POWR™
Slide 17: Preclinical Antifibrotic Efficacy
Preclinical Antifibrotic Efficacy FIBROSIS – a response to chronic injury – is a major cause of organ dysfunction and its reduction is the primary goal in the treatment of NASH Human Cell Cultures CRV431 Effects Hepatic stellate cells, fibroblasts TGFβ or endogenous stimulation ▼fibrotic gene expres
Slide 18: Phase 1 Studies – Safety, Tolerability and Pharmacokinetics
PHASE 1 STUDIES – Safety, tolerability and pharmacokinetics (PK) CRV431 in Healthy Subjects Single Ascending Dose (SAD) Multiple Ascending Dose (MAD) Drug-Drug Interaction (DDI) ✓ N = 32 (24 CRV431; 8 Placebo). ✓ N = 25 (All CRV431). ✓ N= 18 ✓ Doses: 75 mg, 225 mg, 375mg, ✓ Doses: 75 mg, 150 mg, 225
Slide 19: Example of AI-POWR Utility with CRV431 in NASH/NAFLD Liver Slices
Example of AI-POWR™ Utility with CRV431 in NASH/NAFLD Liver Slices AI-POWR™ Rapid and Early Identification of Key NASH-Related Genes Altered by 3-Days CRV431 Gene Implications for NASH Fold p-value (Down-regulation, loss-of-function, Change ▪ NASH/NAFLD is heterogenous polymorphism) TM6SF2 Linked to
Slide 20: Ongoing Phase 2a AI-Powered Pilot Study
ONGOING PHASE 2a AI-POWERED PILOT STUDY OBJECTIVES ▪ Evaluate the safety and tolerability of once daily (qd) 75 mg and 225 mg dose of CRV431 in presumed nonalcoholic steatohepatitis (NASH) fibrosis stage 2 (F2)/fibrosis stage 3 (F3) patients compared to placebo control over 28 days of dosing ▪ Measu
Slide 21: Phase 2b AI-POWR NASH Registration Trial
PHASE 2b AI-POWR NASH REGISTRATION TRIAL OBJECTIVES: • Evaluate the efficacy of once daily (qd) 75 mg and 225 mg dose of CRV431 in biopsy proven NASH fibrosis stages F2 and F3 patients compared to placebo over 6 months of dosing. • Measure antifibrotic activity of CRV431 • Validate antifibrotic biom
Slide 22: Anticipated Events
ANTICIPATED EVENTS CRV431 ▪ Complete Ongoing Phase 2A NASH program (first dosing (75 mg) cohort completed November 2020) ✓ Enrolled the last patient in the 75 mg cohort ✓ DSMB recommended the study continue with 225 mg dose cohort ✓ Clinically significant reductions observed in the liver safety para
Slide 23: Contact Us
HEPA CONTACT US Robert T. Foster, PharmD, PhD Chief Executive Officer HEPION PHARMACEUTICALS INC. 399 Thornall Street, First Floor Edison, New Jersey, USA, 08837 www.hepionpharma.com Email: rfoster@hepionpharma.com p. 23
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