Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (August 2026)
38 slides from Capricor Therapeutics's investor presentation, August 2026
Slide 1: Corporate Presentation
Corporate Presentation Capricor Therapeutics, Inc. Nasdaq: CAPR August 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: Committed to advancing transformative treatments for patients
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 01 Capricor Therapeutics Overview 02 Deramiocel DMD Program Overview 03 StealthX Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 4
Slide 5: Capricor's History & Evolution
Capricor’s History & Evolution Discovery to Clinical Validation 2018: Published foundational preclinical 2012: Initial publication Duchenne muscular dystrophy (DMD) demonstrating early clinical study in Stem Cell Reports Q3 2026: Published positive benefits of CDCs1 2022: Published positive results
Slide 6: Scientific Foundation & Lead Program
Scientific Foundation & Lead Program Lead Program in Rare Disease Duchenne Muscular Dystrophy Scientific Foundation Pipeline Development Cardiology and Cell Biology StealthXTM Exosome Platform Core technology: developed at Lead indication - DMD: a rare, Targeted natural drug Johns Hopkins Univ
Slide 7: 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 Deramiocel Duchenne muscular allogeneic CDCs dystrophy (DMD) Becker muscular Deramiocel dystrophy (BMD) StealthXTM Infectious di
Slide 8: Deramiocel Duchenne Program: Regulatory Update
Deramiocel Duchenne Program Regulatory Update PDUFA Extension Phase 3 HOPE-3 Data Path Forward N E W PDUFA TA RG E T DAT E PUL BE N E F I T VS. PLAC E BO RE F I N E D TA RG E T I N D I C AT I O N Nov. 22, 2026 4.55% Upper limb impairment (>9 years of age) • Extended from Aug. 22, • Primary endpoint
Slide 9: Capricor: Financial Overview
Capricor: Financial Overview Runway and Potential Cash Infusions CASH BALANCE CURRENT RUNWAY OUTSTANDING EMPLOYEES SHARES $238 Through ~350 FTEs 58.1 million¹ million¹ 2027* *Excludes any potential revenue from product sales and the potential monetization of a PRV ELIGIBLE FOR PRIORITY REVIEW VOUCHE
Slide 10: Table of Contents
Table of Contents 01 Capricor Therapeutics Overview 02 Deramiocel DMD Program Overview 03 StealthX Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 10
Slide 11: Deramiocel: Cellular Therapy
Deramiocel: Cellular Therapy Comprised of Human Allogeneic Cardiosphere-Derived Cells (CDCs) CDCs are derived from Peer-reviewed scientific cells of transplant publications on CDCs qualified human hearts; >250 worldwide1 they are not stem cells Patients administered Intravenous doses >300 CDCs acros
Slide 12: Deramiocel's Multi-Modal Mechanism
Deramiocel’s Multi-Modal Mechanism CDCs (Deramiocel) 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 Transfo
Slide 13: 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 doses
Slide 14: Capricor's GMP Manufacturing Facility
Capricor’s GMP Manufacturing Facility San Diego, California FDA Pre-License Inspection successfully completed in 2025 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 14
Slide 15: 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 16: 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 17: Performance of Upper Limb: PUL
Performance of Upper Limb: PUL Assessment to Evaluate Upper-Limb Function & Impact on Daily Living Proximal (shoulder) domain Evaluates high-level movements, PUL 2.0 scores such as lifting arms above head decline Middle (elbow) domain indicating Measures functional actions using the elbow, such as
Slide 18: 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 19: 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 DMD Orphan Drug Designation (FDA and EMA) Regenerative Medicine Advanced Ther
Slide 20: Deramiocel's Clinical Development
Deramiocel’s Clinical Development 3 Independent Trials in DMD HOPE-DUCHENNE1 HOPE-2 HOPE-2 OLE HOPE-3 Phase Phase Phase Phase 1 2 2 3 N=25 N=20 N=13 N=106 • Randomized, placebo- • 5-year extension study • Randomized, placebo- • Statistically significant controlled trial controlled trial • PUL total:
Slide 21: HOPE-3 Pivotal Phase 3 Trial
HOPE-3 Pivotal Phase 3 Trial Overview Deramiocel Design & Endpoints S R 150 million cells Phase 3: randomized (1:1), double-blind, placebo- C A IV infusion (quarterly) controlled study R N E D N = 106 subjects randomized 12 O Baseline E 1:1 (DERAMIOCEL TO PLACEBO) months Conducted in the U.S.
Slide 22: 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 23: 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 24: HOPE-3: Key Efficacy Results
HOPE-3: Key Efficacy Results Skeletal Muscle (PUL 2.0) and Cardiac Function (LVEF) n=54 n=52 n=32 n=32 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside **McDonald, et al. The Lancet, 2026 24
Slide 25: Compelling Efficacy Data
Compelling Efficacy Data Across Two Randomized Placebo-Controlled Trials LS-Means Difference Favors Placebo Favors Deramiocel (95% CI) p-value HOPE-3 (Phase 3) PUL 2.0 Total Score (Primary) 4.55 (0.47, 8.63) 0.0290 LVEF (%) (Key secondary) 9.51 (-1.64, 20.65) 0.0935 PUL 2.0 Mid-level Score* 8.12 (2.
Slide 26: DMD: Large Commercial Opportunity
DMD: Large Commercial Opportunity PREVALENCE¹ LIFE EXPECTANCY 15,000 DMD patients in United States ~25-30 years 150,000-200,000 DMD patients worldwide MARKET SIZE² COMMERCIAL OPPORTUNITY ~$27 Billion Target reimbursement price Aim to be similar or higher than approved Global market size estimated by
Slide 27: 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 Infrastructure Potential Capricor is Patient support Pricing e
Slide 28: Key Duchenne Advocacy Relationships
Key Duchenne Advocacy Relationships Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 28
Slide 29: 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) Michelle Eagle, Ph.D., Eugenio Mercuri, M.D., Kan Ho
Slide 30: 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 31: Table of Contents
Table of Contents 01 Capricor Therapeutics Overview 02 Deramiocel DMD Program Overview 03 StealthX Exosomes Platform Overview Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 31
Slide 32: 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 33: 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 34: 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 35: 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 36: StealthX Exosome Platform: Building a New Class of Medicines
StealthXTM Exosome Platform Building a New Potential 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 partn
Slide 37: Experienced Leadership Team
Experienced Leadership Team Extensive Scientific and Operational Experience Across Pharma & Biotech Linda Marbán, Ph.D. AJ Bergmann, M.B.A Kristi Elliott, Ph.D. Chief Executive Officer Chief Financial Officer Chief Operating & Science Officer Prior experience: Excigen, Johns Hopkins University Prior
Slide 38: 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 38
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