Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (May 2022)
43 slides from Capricor Therapeutics's investor presentation, May 2022
Slide 1: Corporate & Investor Presentation
CORPORATE & INVESTOR PRESENTATION NASDAQ: CAPR
Slide 2: Forward Looking Statements
Forward Looking Statements Statements in this presentation regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding
Slide 3: Capricor Therapeutics Company Highlights
Capricor Therapeutics Company Highlights NASDAQ: CAPR Cell and Exosome-Based Platform Therapeutics Company Commencing Phase 3 in Duchenne Muscular Dystrophy • Strategic partnership: $705 million in potential milestones • Market potential over $1 billion annual sales • Positive phase 2 data published
Slide 4: Capricor's Recent Achievements
Capricor’s Recent Achievments Cell Therapy Program in Duchenne Muscular Dystrophy • Entered exclusive partnership with Nippon Shinyaku, Co. for U.S. distribution rights • Received $30 million, additional $705 million in potential milestones plus revenue share • Phase 2 data published in The Lancet •
Slide 5: Capricor's Product Pipeline
Capricor’s Product Pipeline Target Development Phase Candidate Indications Discovery Preclinical Phase I Phase II Phase III Status CAP-1002 Duchenne Muscular Phase III initiating Q2 2022 (allogeneic CDCs) Dystrophy Exosome-Based Vaccine Infectious diseases Preclinical (Multivalent design) Engineered
Slide 6: Capricor's CAP-1002 Cell Therapy Technology
Capricor’s CAP-1002 Cell Therapy Technology Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 6
Slide 7: Capricor's Cell Therapy Technology
Capricor’s Cell Therapy Technology CAP-1002 CAP-1002: biologic consisting Manufactured of allogeneic cardiosphere- from donated heart muscle derived cells (CDCs) Does not act by «stemness» Has been investigated in the cells do not engraft into host tissue multiple independent clinical trials and app
Slide 8: CAP-1002 Mechanism of Action: Defined
CAP-1002 Mechanism of Action: Defined phospho-Akt HO-1 GCLC cat. sub. Oxidative Nrf2 (cytoplasmic) Stress catalase SOD-2 Nrf2 (nuclear) phospho-IkB CD68+ macrophages Inflammation NF-kB p65 (nuclear) MCP1 CD3+ T cells collagen I Fibrosis collagen III mitochondrial DNA
Slide 9: CAP-1002 Supporting the Mechanism of Action
CAP-1002 Supporting the Mechanism of Action Purity: miR-#1 Identity: miR-#2 Potency miR-#3 Non-Potent Potent Non-Potent Potent MSC-EXO CDC-EXO MSC-XO CDC-EXO Non-Potent Potent CAP-1002’s purity, identity and potency can be defined by specific miRNAs Capricor Therapeutics, Inc. Developing Transformat
Slide 10: Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy CAP-1002 Cell Therapy Technology Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 10
Slide 11: Duchenne Muscular Dystrophy Market Opportunity
Duchenne Muscular Dystrophy Market Opportunity Duchenne Muscular Dystrophy: the most severe form of muscular dystrophy • X-linked genetic disorder • Unable to produce dystrophin: essential protein for muscle formation and growth • Deficiency leads to loss of ambulation; severe cardiac and respirator
Slide 12: Lack of Dystrophin Predisposes Muscle to Damage
Duchenne Muscular Dystrophy Lack of Dystrophin Predisposes Muscle to Damage Dystrophin is a structural protein located within the muscle fiber membrane Acts both as a cushion and glue Without dystrophin, muscles are unable to Dystrophin function properly, suffer progressive damage and eventually die
Slide 13: Capricor's Addressable DMD Population
Capricor’s Addressable DMD Population Capricor’s Targeted Patient Population Estimated that over 50% of DMD patients in U.S. are non-ambulant Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 13
Slide 14: Trajectory of CAP-1002 in DMD: Preclinical Data
Trajectory of CAP-1002 in DMD Preclinical Data HYPOTHESES CDCs to treat CDCs to improve CDCs to improve CDCs to treat cardiomyopathy exercise capacity skeletal muscle diaphragm Left ventricular ejection Exercise performance function muscle fraction markedly approximately doubled Twitch force, tetani
Slide 15: Competitive Landscape for DMD
Competitive Landscape for DMD 01 02 03 Exon Gene Steroids Options Skipping therapy 01 02 03 CAP-1002 Benefits Exon Skipping Gene therapy Steroids Challenges treats a small potential have adverse portion of the safety risks side-effects DMD population • Immunomodulatory • Anti-fibrotic CAP-1002 • Pro
Slide 16: Performance of Upper Limb (PUL Test)
Performance of Upper Limb (PUL Test) To Assess Skeletal Muscle Function PUL v.2.0: • 3-point response scale - more robust and reproducible than v1.2 • Compensatory strategies allowed to achieve tasks (not allowed in v1.2) • v2.0: better able to detect change at 12 months at all levels of ability*
Slide 17: Capricor's Regulatory Designations
Capricor’s Regulatory Designations Duchenne Muscular Dystrophy Products may also be eligible for accelerated approval • RMAT provides benefits that include more frequent 01 meetings with FDA to discuss the development plan for the product candidate RMAT GOAL OF FDA’S • Eligibility for rolling review
Slide 18: HOPE-Duchenne Phase I/II Overview
HOPE-Duchenne Phase I/II Overview • Phase I/II study: 25 patients, randomized and open-label • One-time, multi-vessel, intracoronary delivery of 75M cells • HOPE population were all on stable corticosteroids • Very limited options for this patient population RESULTS • Reduction in cardiac scar at 6
Slide 19: HOPE-Duchenne Results
HOPE-Duchenne Results Reduced Cardiac Scar and Improved PUL Scar Size Mid+distal PUL Scar R.G. Victor et al., AHA LBCT 2017; M. Taylor et al., submitted Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in Neurology, 2019. 19
Slide 20: HOPE-2 Phase II Design
HOPE-2 Phase II Design • Design: Phase II, randomized, double-blind, placebo-controlled trial in participants with DMD and reduced skeletal muscle function • Objective: Evaluate safety and efficacy of CAP-1002 • Dosing Regimen: 150M cells delivered intravenously every 3 months • Sites: 9 sites (USA)
Slide 21: HOPE-2 Phase 2 Results
HOPE-2 Phase 2 Results Published in The Lancet, March 2022 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 21
Slide 22: HOPE-2 Upper Limb Improvements
HOPE-2 Upper Limb Improvements Primary Efficacy Endpoint: PUL 1.2 Mid Level Mid-level PUL 1.2 p=0.01 improvement 71% slowing of disease (PUL) Δ2.6 points in CAP-1002 vs. placebo at 12-months Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline
Slide 23: HOPE-2 Upper Limb Improvements (PUL 2.0)
HOPE-2 Upper Limb Improvements PUL 2.0 Full Full PUL 2.0 p=0.04 improvement Δ1.8 points in CAP-1002 vs. placebo at 12-months Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as dependent variable and percentile ranked baseline score, treatm
Slide 24: HOPE-2 Cardiac Improvements
HOPE-2 Cardiac Improvements Ejection Fraction and CKMB LV Ejection Fraction Creatine Kinase MB/ Total Creatine Kinase (%) improvement p=0.002 p=0.02 Δ=4% in LVEF by cardiac MRI Biomarker to assess cardiac muscle damage Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile
Slide 25: HOPE-2 Safety Results
HOPE-2 Safety Results 69total infusions Generally safe and well tolerated throughout the study With the exception of three were performed in HOPE-2 hypersensitivity reactions; no safety signals were identified Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Resu
Slide 26: CAP-1002 Proteomic Biomarkers in DMD
CAP-1002 Proteomic Biomarkers in DMD A B Treated vs. C Placebo (mons) Blood Protein Detection Function and Canonical pathways 3 6 component conc. method Binding of mononuclear leukocytes Cell movement of natural killer cells Mobilization of blood cells Resident Mitra blood device LC-MS Dual Plasma L
Slide 27: Phase III Pivotal Trial
Phase III Pivotal Trial Initiating in Q2 2022 • HOPE-3: Phase III, randomized, double-blind, placebo-controlled trial to be conducted in the United States • Target Enrollment: 70 patients • Number of estimated sites: 20-30 • Patient Population: Non-ambulant DMD patients • Primary Endpoint: PUL 2.0 a
Slide 28: Partnership with Nippon Shinyaku
Partnership with Nippon Shinyaku Exclusive Distribution and Commercialization Commercial Rights: CAP-1002 for the treatment of DMD • US rights only • Nippon to appoint NS Pharma, its US subsidiary, as exclusive sales and distribution partner Deal Valued at up to $735M • $30M upfront • Up to $705M in
Slide 29: World-Class Duchenne Advisory Board
World-Class Duchenne Advisory Board Craig McDonald, Pat Furlong Kan Hor, M.D. Thomas Voit, M.D. M.D. (National PI) Parent Project Muscular Nationwide Children's Hospital University College University of California Dystrophy (USA) (USA) London (UK) at Davis (USA) Michelle Eagle, Oscar Henry Mayer, Eu
Slide 30: Capricor's Exosome Platform
Capricor’s Exosome Platform Therapeutics and Vaccines Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 30
Slide 31: RNA Therapeutics
RNA Therapeutics A New Class of Medicines Broad Market Opportunities Capital Efficiency for Development Rapid Innovation Possible Proof of Concept Established in Vaccines Barrier to Success: Effective Delivery Systems of Nucleic Acids Capricor Therapeutics, Inc. Developing Transformative Therapies f
Slide 32: Exosomes are Nature's Delivery Tool
Exosomes are Nature’s Delivery Tool Drug Delivery System • ~100 nanometer vesicles • Made by nearly all cells • Abundant in blood and all biofluids • Transfers signals and molecules to other cells • Decades of transfusion and transplantation medicine demonstrates safety • Can be used to deliver RNAs
Slide 33: Exosomes Platform Development Tools
Exosomes Platform Development Tools Production Loading Targeting Delivery • Scalable production • Evaluate loading • Unmodified for local • Exosome targeting, processes in requirements and size injection cargo delivery, dosing, development constraints • Cell-specific targeting safety, distribution •
Slide 34: Exosomes Potential Solutions to Drug Delivery Challenges
Exosomes Potential Solutions to Drug Delivery Challenges Problems Possible Solutions • Gene therapy using viral delivery (AAV) Exosomes: • Immune response • low immunogenicity • Delivery of RNAs • can deliver contents to the cell • Uptake to render biologic relevance without integration • Therapeuti
Slide 35: Targeted Exosome Platform
Targeted Exosome Platform Allows for Broad Applicability SURFACE-MODIFIED EXOSOMES Specific Tropism • Modify exosome membrane • Targeted delivery CDC-EXOSOMES Regenerative Medicine 2.0 • Immunomodulation • Tissue regeneration ENGINEERED EXOSOMES Loaded Exosomes • Exosomes loaded with therapeutic car
Slide 36: Exosomes as a Delivery Vehicle
Exosomes as a Delivery Vehicle Vaccine: Proof of Concept Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 36
Slide 37: Novel Exosome-Based Vaccine Platform
Novel Exosome-Based Vaccine Platform Display and mRNA Technologies Exosome-based approach • Natural intracellular delivery particle • Allows for specificity in cellular targeting • Innovative technology, yet is well characterized Unique multi-antigen approach • Potentially confers greater immunity b
Slide 38: Coronavirus Structure
Coronavirus Structure Unique Protein Visualization Spike Protein generates antibody response* Other vaccines Spike in development are targeting the spike “S” protein solely (S) Capricor’s exosome vaccine Addresses both “S” and “N” proteins Nucleocapsid (N) Unique vaccine approach utilizing exosomes
Slide 39: Exosome: Display Vaccine Approach
Exosome: Display Vaccine Approach Broad Potential Use in Infectious and Other Diseases 01 02 03 04 05 Create vectors Transfect Grow the cells, Purify the exosomes: Immunize that express into 293 cells which produce which carry the S and N with purified VLPs, the SARS-CoV-2 SARS-CoV-2 VLPs proteins i
Slide 40: Exosome mRNA Vaccine
Exosome mRNA Vaccine Preclinical Results: Cellular and Humoral Response Study key findings: • Drives cell and antibody immunity to nucleocapsid protein (SARS-CoV-2) • Drives cell and antibody immunity to spike protein (SARS-CoV-2) • Confirms multiplexed mRNA approach • Unique antigen design Capricor
Slide 41: Exosomes Platform Goals
Exosomes Platform Goals Building a New Class of Medicines Monogenic Diseases RNA and protein therapeutics Oncology Vaccines and targeted delivery therapeutics Inflammatory & Vascular Biologics Exosomes Drug Payload Infectious Diseases Vaccines Cell Drive research Expand and exploit Scale Goals and p
Slide 42: Recent and Targeted Milestones
Recent and Targeted Milestones Cash Runway CAP-1002 for Duchenne Muscular Dystrophy Reported 1-year positive results from HOPE-2 at World Muscle Society Announced FDA acceptance of Phase 3 trial protocol and design Announced partnership with Nippon Shinyaku for US distribution rights Published Phase
Slide 43: Senior Leadership Team
Senior Leadership Team Linda Marbán, Ph.D. AJ Bergmann, M.B.A. Karen Krasney, J.D. Daniel Paulson, M.D. Kristi Elliott, Ph.D. Yasmine Shad Stephen Gould, Ph.D. Chief Executive Officer, Chief Financial Officer Executive Vice President Vice President of Vice President of R&D Vice President of Quality
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