Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — PPMD Webinar (October 2026)
27 slides from Capricor Therapeutics's conference presentation, Oct 7, 2026
Slide 1: Deramiocel Program Update
Deramiocel Program Update Capricor Therapeutics, Inc. · Nasdaq: CAPR October 7, 2026 · PPMD Webinar Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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: Call Attendees
Call Attendees CAPRICOR THERAPEUTICS CLINICIANS/KOLS Linda Marbán, Ph.D. Craig McDonald, M.D. Chief Executive Officer National Principal Investigator, HOPE-2 and HOPE-3 Professor of Pediatrics; Chair, Department of Physical Medicine and Rehabilitation; Director of Rehabilitation Services and of the
Slide 4: Deramiocel Regulatory Update
Deramiocel Regulatory Update PHASE 3 HOPE-3 RESULTS NEW FDA DECISION DATE (PDUFA) 4.55% better preserved arm function (PUL 2.0) vs. placebo at 12 months Nov. 22, 2026 ✓ Primary endpoint met (p=0.029) One of the largest DMD datasets Extended 3 months from Aug. 22, 2026 after FDA classified our submis
Slide 5: Deramiocel BLA Amendment Data Package
Deramiocel BLA Amendment Data Package How our application changed, and the new data FDA is now reviewing ORIGINAL FILING NEW DATA ADDED DMD-associated REFINED INDICATION cardiomyopathy HOPE-3 Robustness and sensitivity analyses FILED WITH DMD upper limb HOPE-2 (Phase 2) RCT 12-month results impairme
Slide 6: HOPE-3: 1-Year Results Published in The Lancet (2026)
HOPE-3: 1-Year Results Published in The Lancet (2026) McDonald, et al. The Lancet, July 2026 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 6
Slide 7: Consistent Benefits Shown Across the HOPE Studies
Consistent Benefits Shown Across the HOPE Studies ~1 point per year PUL 2.0 treatment benefit, consistent in every HOPE study HOPE-3 HOPE-3 OLE HOPE-2 HOPE-2 OLE ISE Duration 12 months 12 months 12 months 36 months 12 months Deramiocel Delayed-start Deramiocel Deramiocel Deramiocel Comparison vs. pl
Slide 8: HOPE-3 OLE: 2-Year Results
HOPE-3 OLE: 2-Year Results Craig McDonald, M.D. National Principal Investigator, UC Davis Health HOPE-3 OLE Overview HOPE-3 OLE Efficacy Results HOPE-3 OLE Safety Summary Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 8
Slide 9: PUL 2.0: 1-Point Change is Clinically Meaningful
PUL 2.0: 1-Point Change is Clinically Meaningful Bringing Hand to Mouth Bringing Hand to Mouth PUL 2.0 Score = 2 PUL 2.0 Score = 1 Disease Progression (Normal) (Difficulty/Compensation) PUL 2.0 Score 2 Normal Ability With difficulty / 1 compensation 0 Unable to perform Capricor Therapeutics, Inc. De
Slide 10: HOPE-3 OLE: Study Design
HOPE-3 OLE: Study Design Year 1 compared Deramiocel with placebo. In Year 2, all patients received Deramiocel YEAR 1 · DOUBLE-BLIND YEAR 2 · OPEN-LABEL EXTENSION Early start Deramiocel arm Deramiocel Deramiocel 54 randomized · 49 in Year 2 Delayed start Placebo arm Placebo Deramiocel 52 randomized ·
Slide 11: HOPE-3 OLE: Baseline Characteristics
HOPE-3 OLE: Baseline Characteristics A later-stage, mostly non-ambulatory population; groups were similar at the start Deramiocel Placebo Characteristic (early start, N=49) (delayed start, N=49) Age at randomization, years, mean (SD) 15.3 (3.11) 14.4 (2.93) ~15 years Age at DMD diagnosis, years, mea
Slide 12: HOPE-3 Primary Endpoint: PUL 2.0 Total Score
HOPE-3 Primary Endpoint: PUL 2.0 Total Score Patients on Deramiocel lost less arm function over 12 months than those on placebo PUL 2.0 Total Score, ITT (raw means) PRIMARY ENDPOINT SENSITIVITY ANALYSIS 4.55% 1.1 points Less decline vs. placebo Less decline vs. placebo (percent change) (absolute cha
Slide 13: HOPE-3 OLE: PUL 2.0 Results
HOPE-3 OLE: PUL 2.0 Results The benefit follows the treatment, not the passage of time Delayed start: placebo year vs. first treated year Early start: treated year 1 vs. treated year 2 Difference at 12 mo Difference at 12 mo +1.56 +0.05 (95% CI 0.47 to 2.64) (95% CI −1.03 to 1.14) ✓ Treated year bet
Slide 14: HOPE-3 OLE: Two Years vs. Expected Decline
HOPE-3 OLE: Two Years vs. Expected Decline At 2 years, patients on Deramiocel declined less than models and natural history predicted KEY TAKEAWAYS • Early start patients • −3.42 vs. −5.88 points after 2 years on Deramiocel • Delayed start patients: • −3.76 vs. −5.35 points after switching at Month
Slide 15: HOPE-3: Heart Function (LVEF: 1-Year Results)
HOPE-3: Heart Function (LVEF: 1-Year Results) PATIENTS WITH CARDIOMYOPATHY Heart pumping function held steady on Deramiocel and declined on placebo Change in LVEF from baseline (raw means, ±SE) Deramiocel DIFFERENCE VS. PLACEBO 2.8% n=32 LVEF change (%) in LVEF at 12 months (n=64) p = 0.017 ranked A
Slide 16: HOPE-3 OLE: LVEF vs. Matched Natural History
HOPE-3 OLE: LVEF vs. Matched Natural History First year on Deramiocel (both arms) Second year (early start only) Matched external natural history ITT Cardiomyopathy (sub-population) Treated n=73 · External n=103 Treated n=56 · External n=100 Month 12 Month 12 +2.4 pp +2.7 pp (0.9, 3.9) (1.1, 4.4) Mo
Slide 17: Safety Summary
Safety Summary WHAT AN INFUSION INVOLVES 1 Before 2 During 3 After Oral prednisone and IV infusion, about 60 minutes Approximately 2 hours of antihistamines to help prevent observation by the care team reactions following infusion Generally well-tolerated No known long-term safety Side effects were
Slide 18: HOPE-3: Robustness & Sensitivity Analyses
HOPE-3: Robustness & Sensitivity Analyses Nathaniel Hogan, Ph.D. Biostatistics, Capricor Therapeutics 600+ robustness analyses Integrated HOPE-2 & HOPE-3 efficacy (ISE) Within-patient comparisons Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 18
Slide 19: Robustness & Sensitivity Analyses
Robustness & Sensitivity Analyses Two Additional Deliverables Provided to the FDA Robustness Report Integrated Summary of Efficacy (HOPE-3) (HOPE-2 & HOPE-3) • Aim to address FDA concerns regarding • Efficacy results of combined HOPE-2 and fragility HOPE-3 data • Aim to address issues related to ana
Slide 20: HOPE-3: Robustness of the Primary Endpoint
HOPE-3: Robustness of the Primary Endpoint 600+ analyses ~1.0 point benefit Real effect performed to test the robustness of consistent treatment effect across Shows lack of fragility: every analysis the primary result analyses favored Deramiocel Each marker is one analysis result; marker size reflec
Slide 21: Integrated Summary of Efficacy (ISE)
Integrated Summary of Efficacy (ISE) HOPE-2 and HOPE-3 combined into one integrated analysis Both placebo-controlled trials used the same dosing, administration and assessments One integrated analysis HOPE-2 HOPE-3 Deramiocel vs placebo Deramiocel vs placebo Upper-limb function PUL 2.0 total score C
Slide 22: ISE: PUL 2.0 Total Score at Month 12
ISE: PUL 2.0 Total Score at Month 12 PUL 2.0 Total, LS mean change from baseline (points, ±SE) Month 12 treatment difference Deramiocel vs placebo, pooled HOPE-2 & HOPE-3 ABSOLUTE CHANGE 1.29 PUL points p = 0.0087 · 95% CI 0.33 to 2.25 PERCENT CHANGE 5.27 percentage points p = 0.0051 · 95% CI 1.61 t
Slide 23: ISE: PUL 2.0 at Month 12, Sensitivity Analyses
ISE: PUL 2.0 at Month 12, Sensitivity Analyses Sensitivity analyses aimed to address AdCom concerns point estimate range 1.11–1.31 across all 7 analyses (primary 1.29) 8 of 8 95% confidence intervals exclude zero p < 0.02 for every analysis Analysis 1.0 = clinically meaningful SE LSMD (95% CI) p val
Slide 24: ISE: LVEF at Month 12
ISE: LVEF at Month 12 ITT population Cardiomyopathy population Mean change in LVEF (percentage points), ±SE Mean change in LVEF (percentage points), ±SE percentage points percentage points (p = 0.XX) +2.21 LS-mean difference at Month 12, Deramiocel vs placebo (p = 0.05) +3.13 Observed difference at
Slide 25: Data Summary & Conclusions
Data Summary & Conclusions The Deramiocel treatment effect is robust, not fragile Robustness Report Integrated Summary of Efficacy (ISE) (HOPE-3) (HOPE-2 & HOPE-3) • 600+ alternative analyses, same ~1-point • More patients: 1.29-point PUL benefit, PUL benefit p=0.0087 • Effect does not shift with an
Slide 26: Acknowledgements
Acknowledgements We are grateful to the patients and families who took part in the HOPE studies THEIR COMMITMENT MADE THIS RESEARCH POSSIBLE Investigators and study teams Patient advocacy and scientific community World Muscle Society (WMS) HOPE-3 investigators, coordinators and staff at 20 sites Mus
Slide 27: Q&A
Q&A Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 27