Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Q4 2025 Earnings Presentation
20 slides from Capricor Therapeutics's earnings presentation, Mar 12, 2026
Slide 1: Confirmation of musculoskeletal and cardiac benefit in DMD from deramiocel in HOPE-3
Poster Number: 479 LBT Confirmation of musculoskeletal and cardiac benefit in DMD from deramiocel, an allogeneic cell therapy, in the Phase 3 HOPE-3 study Craig McDonald M.D. Chair, Department of Physical Medicine & Rehabilitation Distinguished Professor PM&R and Pediatrics Director Neuromuscular Di
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: Disclosures
Disclosures • Dr. McDonald has received consulting income and clinical trial research grants from Capricor Therapeutics CAPRICOR THERAPEUTICS, INC. 3
Slide 4: Regulatory Update: Target PDUFA Date August 2026
Regulatory Update Target PDUFA Date 22 August 2026 CAPRICOR THERAPEUTICS, INC. 4
Slide 5: Introduction to Deramiocel
Introduction to Deramiocel • Cardiosphere derived cells (CDCs) are cardiac tissue derived cells which: • Have anti-fibrotic, immunomodulatory and anti-inflammatory activities in human in vitro assays1 • In murine mdx models improve both skeletal and cardiac muscle function and reduce cardiac fibrosi
Slide 6: HOPE-3 Pivotal Phase 3 Trial Study Design
HOPE-3 Pivotal Phase 3 Trial Study Design Design & Endpoints Phase 3: randomized (1:1), double-blind, placebo- controlled study N = 106 subjects randomized Conducted in the United States at 20 clinical sites Primary efficacy endpoint1: PUL v2.0 skeletal muscle assessment Key Entry Criteria
Slide 7: HOPE-3 Patient Disposition
HOPE-3 Pivotal Phase 3 Trial Patient disposition 133 Patients Screened 106 pts Randomized 52 pts Allocated to 54 pts Allocated to Placebo Deramiocel 1 discontinuation due to AE 1 did not receive treatment 1 withdrew consent 3 withdrew consent OUTCOME MEASURES Primary PUL 2.0 (ITT=106) 52 Patients 54
Slide 8: Population Demographics
Population Demographics Well balanced treatment groups 8 1One subject enrolled but dropped out prior to baseline assessment (n=105) 2Updated as of Feb. 2026; subgroup: 64 of 79 patients with centrally reviewed 8 CAPRICOR THERAPEUTICS, INC. Devloping Transformative Therapies from Bench to Bedside and
Slide 9: Safety Results
Safety Results Deramiocel profile is comparable to placebo (which includes DMSO) 9 CAPRICOR THERAPEUTICS, INC. Developing Transformative Terapies from Bench to Bedside 1 Safety population (n=105) 9
Slide 10: Overview of All Type-I Error-Controlled Endpoints
Overview of All Type-I Error-Controlled Endpoints CAPRICOR THERAPEUTICS, INC. 10
Slide 11: Primary Endpoint: PUL 2.0 at 12 Months
Primary Endpoint: PUL 2.0 at 12 Months Deramiocel treatment difference = 1.2 points (54% slowing); p=0.029 11 *LS-Mean difference = 4.55 percentage point (1.2 -point difference on the PUL scale) ** Based on prespecified repeated measures model using percent change from BL CAPRICOR THERAPEUTICS, INC.
Slide 12: Key Secondary Endpoint: LVEF at 12 Months
Key Secondary Endpoint: LVEF at 12 Months Deramiocel treatment difference = 2.4% pts on LVEF (91% slowing); p=0.041 for ranked comparison 12 * LS-mean difference = 11.65 ranks (2.4 percentage point difference in LVEF) ** Based on prespecified rank ANCOVA model LVEF: n reflects the number of patients
Slide 13: Secondary Endpoint: LVEF at 12 Months in Cardiomyopathy Patients
Secondary Endpoint1: LVEF at 12 Months Patients with Cardiomyopathy2 at Baseline Deramiocel treatment difference = 3.3% pts on LVEF (>100% slowing); p=0.017 for ranked comparison 13 n=32 * LS-mean difference = 12.36 ranks (3.3 percentage point difference in LVEF) 1 – Prespecified endpoint not TYPE-I
Slide 14: Secondary Endpoint: Late Gadolinium Enhancement
Secondary Endpoint: Late Gadolinium Enhancement Deramiocel treatment difference = ∆ 3 Segments; p=0.022; LS Mean Change at 12 Months Deramiocel Placebo LGE stabilization with treatment of Deramiocel LGE progression in non- treated patients Baseline Month 12 n=9 n=13 n reflects the number of patients
Slide 15: Secondary Endpoint: Global Statistical Test
Secondary Endpoint: Global Statistical Test Deramiocel treatment difference = ∆ 0.28; p=0.017; LS Mean Change at 12 Months GST is a patient-Level composite outcome to incorporate clinically relevant components of: • Performance of Upper Limb 2.0 Total Score (PUL 2.0) • Left Ventricular Ejection Frac
Slide 16: Disease Progression: Mid-Level PUL 2.0 vs. DVA Eat-10 Bites
Mid-Level PUL 2.0 vs. DVA Eat-10 Bites Similar Statistically Significant Benefits in Hand-to-Mouth Function across multiple analyses Disease Progression CAPRICOR THERAPEUTICS, INC. 16
Slide 17: Conclusions - 1
Conclusions - 1 • Deramiocel is an allogeneic cell therapy delivered intravenously every 3 months and has an acceptable tolerability and safety profile • The HOPE-3 Phase 3 study met all its primary and secondary (type-1 error controlled) endpoints Upper Limb Function – deramiocel slowed disease pro
Slide 18: Conclusions - 2
Conclusions - 2 Cardiac Function – deramiocel preserved LVEF and slowed LGE (cardiac fibrosis) 1) LVEF slowing of 91% in all evaluable patients 2) LVEF slowing of >100% in known cardiomyopathy patients 3) Reduced rate of LGE segment progression Preservation of cardiac function is likely to transla
Slide 19: Acknowledgements
Acknowledgements A Huge Thank You! To all the patients and families who participated in the HOPE-3 Study and… • Muscular Dystrophy Association • Parent Project Muscular Dystrophy • Cure Duchenne • Jonathan Soslow, M.D. (Vanderbilt University 19 • Chet Villa, M.D. (Cincinnati Childrens) • Michael Tay
Slide 20: Questions
Questions 20 CAPRICOR THERAPEUTICS, INC. 20
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