Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Q4 2024 Earnings Presentation
57 slides from Capricor Therapeutics's earnings presentation, Mar 19, 2025
Slide 1: Corporate Presentation
Corporate Presentation Capricor Therapeutics, Inc. Nasdaq: CAPR March 2025 1
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: Committed to pushing the boundaries of possibility
At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for patients in need. 3
Slide 4: Table of Contents
Table of Contents 1 Capricor Overview 2 Deramiocel DMD Program 3 StealthXTM Exosomes Platform 4 Appendix Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 4
Slide 5: Capricor's History & Evolution
Capricor’s History & Evolution 2012: Groundbreaking publication in The Lancet 2018: Stem Cell Reports 2004: Foundational showing CDCs benefits1 preclinical study in DMD Today: Capricor’s BLA discovery of CDCs accepted for review Today: Global partnerships and elucidation of seeking approval of with
Slide 6: Groundbreaking Science to First-in-Class Product
Groundbreaking Science to First-in-Class Product Pioneering Foundation Core Program Research and Discovery Cardiology and Cell Biology Deramiocel (CDCs) StealthXTM Exosome Platform Cardiosphere-derived cells (CDCs) Optimized allogeneic product Opportunity for a new biological being developed for com
Slide 7: Capricor's Product Pipeline
Capricor’s Product Pipeline Development Phase Product Candidate Indication Preclinical Phase 1 Phase 2 Phase 3 Commercial Partner Deramiocel Duchenne muscular dystrophy (allogeneic CDCs) Exosome-Based Vaccine Infectious diseases (Multivalent design) (SARS-CoV-2) Engineered Exosomes (RNA, protein and
Slide 8: Near-Term Value Driving Catalysts
Near-Term Value Driving Catalysts Deramiocel for the Treatment of DMD Regulatory Milestones Approval-Based Potential Milestones & Milestones PRV Sale Pre-BLA meeting Priority review granted Milestones payable to Capricor August 2024 March 2025 through U.S. approval $80 million Initiated rolling BLA
Slide 9: Pathway to Approval
Deramiocel for the Treatment of DMD Pathway to Approval FDA has accepted Capricor’s BLA seeking full approval for deramiocel in the U.S. BLA supported with Capricor’s existing cardiac and natural history data Submission seeking broad DMD-cardiomyopathy label If approved, this would serve to
Slide 10: Timeline to Potential Launch
Timeline to Potential Launch FDA Designations BLA filing Target PDUFA • RMAT BLA accepted for PDUFA action designation review (March 2025) August 31, 2025 • Orphan drug BLA submitted for designation full approval • Rare pediatric Application granted designation priority review Capricor The
Slide 11: Table of Contents
Table of Contents 1 Capricor Overview 2 Deramiocel DMD Program 3 StealthXTM Exosomes Platform 4 Appendix Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 11
Slide 12: Deramiocel Overview
Deramiocel Overview • Deramiocel: biologic consisting of allogeneic cardiosphere-derived cells (CDCs) • Mechanism of action: • Immunomodulatory • Anti-inflammatory • Anti-fibrotic • Pro-angiogenic • Investigated in over 200 patients • Potency assays accepted by FDA which support MOA • DMD regulatory
Slide 13: Deramiocel Mechanism of Action
Deramiocel Mechanism of Action Foundational Scientific Support 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
Slide 14: Deramiocel Manufacturing
Deramiocel Manufacturing Novel Process Enables a Multi-dose Allogeneic Product RNA characterization Capricor receives qualified human heart EDC expansion & Cardiosphere Formation CDC Expansion Deramiocel MCB formulation Explants derived from cardiac tissue Deramiocel thawed and Doses of deramiocel s
Slide 15: Deramiocel Manufacturing Facility
Deramiocel Manufacturing Facility San Diego, California Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 15
Slide 16: DMD: Lack of Dystrophin Predisposes Muscle to Damage
DMD: 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 (cardiac and Dystrophin skeletal) are unable to function properly, suffer progressive damage and eventually die
Slide 17: Deramiocel has the Potential to Redefine the Standard of Care for DMD
Deramiocel has the Potential to Redefine the Standard of Care for DMD Deramiocel can be used in combination with existing therapeutics Deramiocel GENE THERAPIES CORTICOSTEROIDS Elevidys Emflaza®, Agamree® First-in-class therapy for DMD-cardiomyopathy EXON SKIPPING THERAPIES OTHER THERAPEUTICS Viltep
Slide 18: DMD: Large Commercial Opportunity
DMD: Large Commercial Opportunity Prevalence1 ~15,000-20,000 ~200,000 DMD patients in United States DMD patients worldwide Deramiocel Life Expectancy 25-30 years Potential to be High unmet clinical need Disease the first-in-class Burden Patients experience highly burdensome symptoms, including progr
Slide 19: Cardiomyopathy in DMD
Cardiomyopathy in DMD “Cardiopulmonary failure is the leading cause of mortality in DMD in the current era…Unfortunately, standard heart failure therapies are not DMD-specific and have limited efficacy….For maximal efficacy, most therapies should begin early in the disease process…” Circulation: Hea
Slide 20: Duchenne Disease Trajectory
Duchenne Disease Trajectory SEVERITY Impaired ability to run, jump and rise Loss of stair climb Cardiac dysfunction Loss of stair descend Loss of ambulation Loss of upper limb Ventilation Late-stage disease 0 5 10 15 20 25 30 AGE Capricor Therapeutics, Inc. Developing Transformative Therapies from B
Slide 21: HOPE-2: Phase 2 Overview
HOPE-2: Phase 2 Overview • Design: Phase 2, randomized, double-blind, placebo-controlled trial in DMD participants with reduced skeletal muscle function (9 USA sites) • Deramiocel dosing: 150 million cells (i.v.) every 3 months over 1 year • Data: 20 subjects (12 placebo, 8 treated) • Primary endpoi
Slide 22: HOPE-2: Phase 2 Results
HOPE-2: Phase 2 Results Compelling Safety and Efficacy Published in The Lancet Statistically and clinically Statistically and clinically Favorable significant changes in PUL v1.2 significant improvements in LVEF Δ2.6 points in deramiocel treated safety and Δ4% in deramiocel treated p=0.01 tolerabili
Slide 23: HOPE-2: Phase 2 Results
HOPE-2: Phase 2 Results Statistically Significant Skeletal and Cardiac Improvements Mid-level PUL v1.2 LV Ejection Fraction p=0.01 p=0.002 p=0.01 Δ=4% in LVEF by cardiac MRI Percentile ranked change from baseline represents the percentage of all change scores smaller than the given value, where lowe
Slide 24: HOPE-2: 21 of 22 Cardiac Measures
HOPE-2: 21 of 22 Cardiac Measures Favored Deramiocel Treatment over Placebo Statistically significant treatment effect in critical measures of cardiac function Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 24
Slide 25: HOPE-2 Open Label Extension
HOPE-2 Open Label Extension Study Overview OLE Overview: 13 patients • 6 original deramiocel patients; 7 original placebo patients; 1 patient withdrew Objective: Continued evaluation of safety and efficacy of deramiocel Demographics • Current mean age: ~17 years • All patients on stable regimen cort
Slide 26: Natural History Data Summary
Natural History Data Summary Source Principal Investigator Data Status Vanderbilt University Summary Stats from Jonathan Soslow, M.D. Cardiac MRI Medical Center /FDA† publication † “This study aims to focus on cardiomyopathy (heart muscle disease), which is the leading cause of death in Duchenne mus
Slide 27: HOPE-2 OLE: 3-Year Cardiac Results
HOPE-2 OLE: 3-Year Cardiac Results Ejection fraction compared to external comparator End systolic volumes- indexed compared to external comparator improvement Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside *Soslow, et al. Cardiovascular Measures of All-Cause Mo
Slide 28: Large Potential Revenue Opportunity
Large Potential Revenue Opportunity • U.S. DMD prevalence1: ~15,000-20,000 patients • Treatment regimen: 4 doses per year • Potential for multi-year treatment U.S. DMD POPULATION ~15,000-20,0001 • Target reimbursement price: aim to be similar or higher than approved exon skipping therapies TARGET RE
Slide 29: Partnership with Nippon Shinyaku
Partnership with Nippon Shinyaku NS Pharma Role: Development, Role: Exclusive manufacturing and U.S. marketing regulatory activities Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 29
Slide 30: Partnership with Nippon Shinyaku
Partnership with Nippon Shinyaku Commercial rights: Deramiocel for the treatment of DMD • Potential milestones from agreements payable to Capricor total ~$1.5 billion1 • Capricor responsible for product manufacturing and clinical activities necessary for potential approvals in territories • Nippon S
Slide 31: Key Duchenne Advocacy Relationships
Key Duchenne Advocacy Relationships Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 31
Slide 32: World-Class DMD Advisory Board
World-Class DMD Advisory Board Craig McDonald, M.D. (National PI) Pat Furlong Kan Hor, M.D. University of California, Davis Parent Project Muscular Dystrophy (USA) Nationwide Children's Hospital (USA) (USA) Timothy Franson, M.D., Michelle Eagle, Ph.D., Oscar Henry Mayer, M.D. Eugenio Mercuri, M.D.,
Slide 33: Potential Expansion of Deramiocel
Potential Expansion of Deramiocel Duchenne muscular dystrophy Potential label expansion to include skeletal muscle myopathy Becker muscular dystrophy Becker cardiomyopathy has similar progression to DMD-cardiomyopathy Other orphan cardiomyopathies Capricor Therapeutics, Inc. Developing Transformativ
Slide 34: Table of Contents
Table of Contents 1 Capricor Overview 2 Deramiocel DMD Program 3 StealthXTM Exosomes Platform 4 Appendix Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 34
Slide 35: Exosomes are Nature's Delivery Tool
Exosomes are Nature’s Delivery Tool Natural Drug Delivery • ~100 nanometer vesicles • Made by nearly all cells • Abundant in blood and biofluids • Transfers signals and molecules to other cells • Decades of transfusion and transplantation medicine indicates safety • Can be used to deliver RNAs, DNA,
Slide 36: Potential Benefits: Exosomes vs. LNPs
Potential Benefits: Exosomes vs. LNPs Natural Exosomes Synthetic LNPs Commercial Manufacturing + +++ Drug/Therapeutic Loading ++ ++ Drug/Therapeutic Release +++ + Cellular Uptake +++ + Targeting +++ + Low Immunogenicity +++ + Safety (expected) (+++) + Clinical trials + +++ Capricor Therapeutics, Inc
Slide 37: StealthX Platform Overview
StealthXTM Platform Overview StealthXTM technology allows Capricor to present diversified proteins outside of exosomes Soluble proteins (ex. ScFvs) Transmembrane proteins (ex. Receptors) Viral antigens StealthXTM technology allows Capricor to load diversified payloads inside of exosomes siRN
Slide 38: Exosomes: Scalable Production
Exosomes: Scalable Production Producer Cell Cell Exosome Exosome Exosome Exosome Drug Line Supernatant Concentration Purification Characterization (DS/DP) • Capricor has developed a scalable, reproducible process for exosome purification • Producer cell line is widely used for production in other ap
Slide 39: Exosome Loading of Drug Payloads
Exosome Loading of Drug Payloads Endogenous and Exogenous Methods Cell Based Cargo Endogenous Loading Packaging • Proprietary StealthX • Co-incubation engineering • Transfection • StealthX producer cells • Electroporation and exosomes • Extrusion • Surface proteins • Freeze-thaw • Therapeutic cargos
Slide 40: StealthX Exosome Platform Goals
StealthXTM Exosome Platform Goals Building a New Class of Medicines Monogenic Diseases RNA, protein and small molecule therapeutics Infectious Diseases Vaccines Oncology Exosomes Drug Payload Vaccines and targeted delivery therapeutics Cell Drive research Expand and exploit Scale Goals and partner t
Slide 41: Experienced Leadership Team
Experienced Leadership Team Broad Experience in Pharmaceutical & Life Sciences Linda Marbán, Ph.D. AJ Bergmann, M.B.A Karen Krasney, J.D. Chief Executive Officer Chief Financial Officer Executive Vice President and General Counsel Prior experience: Excigen, Johns Hopkins University Prior experience:
Slide 42: Table of Contents
Table of Contents 1 Capricor Overview 2 Deramiocel DMD Program 3 StealthXTM Exosomes Platform 4 Appendix Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 42
Slide 43: DMD: Preclinical and Clinical Data
DMD: Preclinical and Clinical Data Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 43
Slide 44: Trajectory of CDCs in DMD
Trajectory of CDCs 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, tetanic fo
Slide 45: HOPE-Duchenne: Phase I/II Results
HOPE-Duchenne: Phase I/II Results Overview: 25 patients, randomized and open-label Scar Size Dosing: 1-time, multi-vessel, intracoronary delivery of 75 million cells RESULTS • Reduction in cardiac scar at 6 and 12 months measured by MRI • Improvements in cardiac function (systolic wall Mid+distal PU
Slide 46: HOPE-2 OLE: 3-Year Skeletal Muscle Results
HOPE-2 OLE: 3-Year Skeletal Muscle Results PUL v2.0 Total Score Compared to External Comparator PUL v2.0 external comparator* • Data matched by age, range of PUL v2.0 total score and entry item score • All patients on standard of care • Longitudinal data per patient between 2-3 years follow-up Capri
Slide 47: HOPE-3 Trial Overview
HOPE-3 Trial Overview Current aim to support label expansion to skeletal muscle Design & Endpoints • Cohorts A and B: n=~105 R Deramiocel, N=~52 • Evaluating next steps for potential global S 150 million cells A expansion C IV infusion q3m R N • Primary endpoint: PUL v2.0 at 12 months E D • Secondar
Slide 48: Exosomes as a Therapeutic Platform
Exosomes as a Therapeutic Platform Antisense Oligonucleotides (ASO)-Loaded Exosomes Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 48
Slide 49: StealthX Platform for DMD
StealthXTM Platform for DMD StealthX -DMD (STX-DMD) engineered exosomes in development for dystrophin restoration • Muscle-targeting antibodies expressed on the exosome surface • PMOs loaded inside exosomes • Aim: to deliver therapeutic payload to muscle and heart Anti-human receptor antibody Human
Slide 50: StealthX Targeted Delivery of Exosomes
StealthXTM Targeted Delivery of Exosomes Skeletal Muscle and Heart Skeletal Muscle – Lower Limbs Control Control PBS PBS STX-DMD STX-DMD 5 Doses 5 Doses STX-DMD STX-DMD 3 Doses 3 Doses STX-DMD STX-DMD 1 Dose 1 Dose • STX-DMD engineered exosomes target skeletal muscle and heart • Significant STX-DMD
Slide 51: StealthX Efficient Exon Skipping
StealthXTM Efficient Exon Skipping Skeletal Muscle – Lower Limbs Loaded Exosomes PMO Alone Dose Dose Dose Dose Dose Dose • Efficient, dose-dependent 1 1/3 1/10 1 1/3 1/10 exon skipping using STX-DMD engineered exosomes No Skipping • ~2.5X greater exon skipping 86% 44% 22% 32% 19% 0% Exon Skipping us
Slide 52: Exosomes as a Vaccine Platform
Exosomes as a Vaccine Platform Proof of Concept Study Results Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 52
Slide 53: StealthX Rapid Vaccine Platform
StealthXTM Rapid Vaccine Platform Select Antigen of Interest • Rapid development cycle • Versatile for multiplexing Create Producer Cell Line • Native protein expression • Potent, dose dependent response • Elicits strong immunity (B and T cells) Expand Vaccine Production • Potentially safer with no
Slide 54: StealthX STX-Spike & STX-Nucleocapsid
StealthXTM STX-Spike & STX-Nucleocapsid Exosome Characterisitics Expected Exosome Size Distribution Exosome Protein Expression Exosome Expression of S & N Proteins Spike Protein STX-S (Spike) and STX- N (Nucleocapsid) are expressed at high level on the cells and exosomes Nucleocapsid Protein Caprico
Slide 55: StealthX STX-Spike & Nucleocapsid
StealthXTM STX-Spike & Nucleocapsid Antibody, T-Cell and Neutralization Responses IgG ELISA IFN-g Elispot Day 35 Day 35 Delta - B.1.617.2 150 PBS STXS day 14 3E11 • Potent, dose dependent % neutralization 100 STXS day 40 3E11 antibody response 50 STXS day 40 3E10 CoV02-Delta Spike 0 induced by Steal
Slide 56: NIH Vaccine Collaboration
NIH Vaccine Collaboration Exosome-Based Multivalent Vaccine • Capricor’s StealthX multivalent vaccine was selected by Project NextGen • Initiative’s aim is to advance a pipeline of innovative vaccines to potentially provide broader and more durable protection against COVID-19 • The National Institut
Slide 57: Capricor Therapeutics, Inc.
Capricor Therapeutics, Inc. 10865 Road to the Cure, Suite 150 San Diego, California 92121 e: info@capricor.com w: www.capricor.com Nasdaq: CAPR Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 57
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