Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Q1 2026 Earnings Presentation
41 slides from Capricor Therapeutics's earnings presentation, May 12, 2026
Slide 1: Corporate Presentation
Corporate Presentation Capricor Therapeutics, Inc. Nasdaq: CAPR February 2026 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 clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, f
Slide 3: At Capricor, we are committed to advancing transformative treatments
At Capricor, we are committed to advancing transformative treatments and delivering meaningful outcomes for patients in need. 3
Slide 4: Table of Contents
Table of Contents Capricor Therapeutics (CAPR) Overview Deramiocel DMD Program Overview StealthXTM Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 4
Slide 5: Capricor's History & Evolution
Capricor’s History & Evolution From Discovery to Late-Stage Clinical Validation and Platform Expansion 2018: Published foundational preclinical 2022-2023: Established strategic 2012: Initial publication Duchenne muscular dystrophy (DMD) Present: Active FDA commercial partnerships in the demonstratin
Slide 6: Capricor's Strategic Priorities
Capricor’s Strategic Priorities Advance Deramiocel toward approval for Duchenne muscular dystrophy Expand pipeline into additional Disciplined indications Long Term Vision corporate and beyond DMD and Lead the next financial pursuing global generation of cell and execution expansion exosome-based me
Slide 7: Groundbreaking Science
Groundbreaking Science Developing First-in-Class Therapeutics Lead Program in Rare Disease Duchenne Muscular Dystrophy Scientific Foundation Pipeline Development Cardiology and Cell Biology StealthXTM Exosome Platform Initial Technology: developed at Indication: Duchenne muscular Targeted natu
Slide 8: Capricor's Product Pipeline
Capricor’s Product Pipeline Advancing Transformative Therapies for Rare Diseases & Beyond Development Phase Product Indication Discovery Phase 1 Phase 2 Phase 3 Commercial Partner Deramiocel Duchenne muscular (allogeneic CDCs) dystrophy Deramiocel Becker muscular (allogeneic CDCs) dystrophy StealthX
Slide 9: Deramiocel Duchenne Program
Deramiocel Duchenne Program Pathway to Approval & Next Steps Pre-license inspection (PLI) completed and CMC alignment CRL issued requesting additional clinical evidence; Type A FDA meeting confirmed HOPE-3 Phase 3 should serve as the required additional study Positive HOPE-3 Phase 3 topline da
Slide 10: Capricor: Financial Overview
Capricor: Financial Overview Runway and Potential Cash Infusions Proforma cash Employees: ~250 FTEs $335 million1 balance ~2-3 years Current runway Based on current operating plan2 Outstanding 55.3 million1 shares Milestone payments U.S. approval: $80 million to Capricor Sales-based milestones: up t
Slide 11: Table of Contents
Table of Contents Capricor Therapeutics (CAPR) Overview Deramiocel DMD Program Overview StealthXTM Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 11
Slide 12: Deramiocel: Cellular Therapy
Deramiocel: Cellular Therapy Comprised of Human Allogeneic Cardiosphere-Derived Cells (CDCs) CDCs are derived from cells of transplant Peer-reviewed scientific qualified human hearts; >250 publications worldwide1 they are not stem cells Patients administered Doses of intravenous >250 CDCs across mul
Slide 13: Deramiocel's Multi-Modal Mechanism
Deramiocel’s Multi-Modal Mechanism CDCs Exosomes and growth factors Anti-fibrotic1 Anti-inflammatory 120 Immunomodulatory1 100 100 8 Protective 80 80 6 MΦ Phenotype CDC 60 Collagen 60 cxcl8 4 40 40 Toxic 20 2 20 0 0 M1 M2 MCDC 0 Control CDC Capricor Therapeutics, Inc. Developing Transformative Thera
Slide 14: Deramiocel Manufacturing
Deramiocel Manufacturing Novel Process Enables a Multi-Dose Allogeneic Product RNA profiling and Capricor receives potency assessment transplant qualified human heart EDC expansion & Cardiosphere Formation CDC Expansion Deramiocel MCB formulation Explants derived from cardiac tissue Deramiocel Deram
Slide 15: Capricor's GMP Manufacturing Facility
Capricor’s GMP Manufacturing Facility San Diego, California FDA Pre-License Inspection (PLI) successfully completed in June 2025 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 15
Slide 16: DMD: A Devastating Rare Disease
DMD: A Devastating Rare Disease High Unmet Needs Across the Entire Disease Trajectory Dystrophin is a key structural protein at the muscle cell membrane that maintains muscle integrity and protects against damage; acts both as a cushion and glue Much of the muscle injury that occurs in DMD is attrib
Slide 17: Approaching Loss of Ambulation
Approaching Loss of Ambulation Late-Ambulatory Patients with DMD Performance of Upper Limb (PUL) entry 2 to 6 (target population) 0 1 2 3 4 5 6 10 sec 10 MWR 100% lose ambulation within 24 months 10 sec 10 MWR indicates onset of upper extremity loss of function (Time to change from PUL entry 6 to PU
Slide 18: Performance of Upper Limb (PUL)
Performance of Upper Limb (PUL) Validated Tool to Assess Skeletal Muscle Function Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside *Mayhew et al, 2019; Pane et al, 2018. 18
Slide 19: Duchenne Cardiomyopathy
Duchenne Cardiomyopathy “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: H
Slide 20: Deramiocel has the Potential to Redefine the Standard of Care
Deramiocel has the Potential to Redefine the Standard of Care for Duchenne Deramiocel can be used in combination with existing therapeutics Deramiocel GENE THERAPIES CORTICOSTEROIDS First-in-class potential therapy for Duchenne muscular dystrophy CARDIAC AND SKELETAL MUSCLE Orphan Drug Designation
Slide 21: Deramiocel's Clinical Development
Deramiocel’s Clinical Development A Decade of Development in Duchenne HOPE-DUCHENNE1 HOPE-DUCHENNE1 HOPE-2 HOPE-2 OLE HOPE-3 Phase 1 Open label extension (OLE) Phase 2 (Ongoing) Phase 3 N = 25 N=8 N = 20 N = 13 N = 106 IC infusion IV infusion IV infusion IV infusion IV infusion • Improved • First st
Slide 22: HOPE-3 Pivotal Phase 3 Trial
HOPE-3 Pivotal Phase 3 Trial Overview Design & Endpoints Deramiocel S R Phase 3: randomized (1:1), double-blind, placebo- 150 million cells C A IV infusion (quarterly) controlled study R N N = 106 subjects randomized E D Baseline 12 Conducted in the United States at 20 clinical sites E O 1:1 (
Slide 23: HOPE-3: Study Demographics
HOPE-3: Study Demographics Placebo Deramiocel Overall Baseline Demographics 1 (n=52) (n=54) (n=106) Age (years) N 52 54 106 Mean (SD) 14.6 (2.95) 15.4 (3.10) 15.0 (3.04) Median 14 15 15 Min, Max 10, 22 10, 22 10, 22 PUL v2.0 entry item score 2,3 23 (44.2) 25 (46.3) 48 (45.3) 4,5,6 29 (55.8) 29 (53.7
Slide 24: HOPE-3: Safety Profile Results
HOPE-3: Safety Profile Results Placebo Deramiocel Overall Overview (n=52), n (%) (n=53), n (%) (n=1051), n (%) Any TEAEs 43 (82.7) 50 (94.3) 93 (88.6) TEAEs related to IP or administration procedure 19 (36.5) 44 (83.0) 63 (60.0) TEAEs related to IP 16 (30.8) 44 (83.0) 60 (57.1) TEAEs related to admi
Slide 25: HOPE-3: Topline Efficacy Results
HOPE-3: Topline Efficacy Results Primary Endpoint Met with Statistical Significance Achieved in All Type-1 Error Controlled Secondary Endpoints n=54 n=52 *LS-Mean difference = 4.55 percentage point (1.2 -point difference on the PUL scale) ** Based on prespecified repeated measures model using percen
Slide 26: HOPE-3: Topline Cardiac Efficacy Results
HOPE-3: Topline Cardiac Efficacy Results Left Ventricular Ejection Fraction n=39 n=44 n=32 n=32 * LS-mean difference = 11.65 ranks (2.4 percentage point difference in LVEF) * LS-mean difference = 12.36 ranks (3.3 percentage point difference in LVEF) ** Based on prespecified rank ANCOVA model ** Base
Slide 27: DMD: Large Commercial Opportunity
DMD: Large Commercial Opportunity Prevalence1 ~15,000 ~150,000-200,000 DMD patients in United States DMD patients worldwide Deramiocel Life Expectancy ~25-30 years Potential to be first-in-class Disease High unmet clinical need Patients experience high symptom burden including muscle weakness, loss
Slide 28: Deramiocel: Strong Commercial Profile
Deramiocel: Strong Commercial Profile Strong Clinical Profile Significant Potential Commercial Reach First-in-Class Slows Disease Sustained Safety Patient Large Commercial Therapy for DMD Progression Benefit Profile Support Reimbursement Partnership Potential Alliance with an Patient support With sm
Slide 29: Partnership with Nippon Shinyaku
Partnership with Nippon Shinyaku Commercial Distribution of Deramiocel for DMD Capricor responsible for product manufacturing and clinical development necessary for potential approvals in select territories Nippon Shinyaku and NS Pharma teams to support broad commercialization efforts United Sta
Slide 30: Key Duchenne Advocacy Relationships
Key Duchenne Advocacy Relationships Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 30
Slide 31: World-Class Duchenne Advisory Board
World-Class Duchenne Advisory Board Craig McDonald, Jonathan Soslow, M.D. Chet Villa, M.D. M.D. (National PI) MSCI Cincinnati Children’s Hospital University of California, Medical Center (USA) Davis (USA) Vanderbilt University Medical Center (USA) Timothy Franson, M.D., Michelle Eagle, Ph.D., Eugeni
Slide 32: Potential Indication Expansion of Deramiocel
Potential Indication Expansion of Deramiocel DUCHENNE MUSCULAR DYSTROPHY BECKER MUSCULAR DYSTROPHY Becker cardiomyopathy has similar progression to DMD-cardiomyopathy OTHER POTENTIAL DISEASES STATES -Cardiomyopathies -Dystrophinopathies -Muscular dystrophies Capricor Therapeutics, Inc. Developing Tr
Slide 33: Table of Contents
Table of Contents Capricor Therapeutics (CAPR) Overview Deramiocel DMD Program Overview StealthXTM Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 33
Slide 34: Exosomes are Nature's Delivery Tool
Exosomes are Nature’s Delivery Tool Natural Drug Delivery Platform • ~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 R
Slide 35: Potential Benefits: Exosomes vs. LNPs
Potential Benefits: Exosomes vs. LNPs Natural Exosomes Synthetic LNPs Commercial manufacturing + +++ Therapeutic loading ++ ++ Therapeutic release +++ + Cellular uptake +++ + Targeting +++ + Low immunogenicity +++ + Safety (expected) (+++) + Clinical trials + +++ Capricor Therapeutics, Inc. Developi
Slide 36: StealthX Exosome Platform
StealthXTM Exosome Platform 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 RNA (
Slide 37: Exosomes: Scalable Production
Exosomes: Scalable Production Producer Cell Cell Exosome Exosome Exosome Exosome Drug Line Supernatant Concentration Purification Characterization Product Capricor has developed a scalable, reproducible process for exosome purification • Producer cell line is widely used for production in other ap
Slide 38: Active NIH Collaboration
Active NIH Collaboration Exosome-Based Multivalent Vaccine Currently in Phase 1 Capricor’s StealthX vaccine was selected by Project NextGen • Aim is to advance a pipeline of innovative vaccines which may provide broader and more durable protection against COVID-19 and other potential infectious th
Slide 39: StealthX Exosome Platform
StealthXTM Exosome Platform 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 through
Slide 40: Experienced Leadership Team
Experienced Leadership Team Extensive Scientific and Operational Experience Across Pharma & Biotech Linda Marbán, Ph.D. AJ Bergmann, M.B.A Michael Binks, M.D. Chief Executive Officer Chief Financial Officer Chief Medical Officer Prior experience: Excigen, Johns Hopkins University Prior experience: G
Slide 41: 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 41
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