Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Corporate Update (December 2025)
18 slides from Capricor Therapeutics's corporate update, Dec 3, 2025
Slide 1: HOPE-3 Phase 3 Study Topline Data Call
1 Capricor Therapeutics, Inc. Nasdaq: CAPR December 3, 2025 HOPE-3 Phase 3 Study Topline Data Call
Slide 2: Forward Looking Statements
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 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 tr
Slide 3: Attendees
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Attendees Linda Marbán, Ph.D. Chief Executive Officer, Capricor Therapeutics AJ Bergmann, M.B.A. Chief Financial Officer, Capricor Therapeutics Michael Binks, M.D. Chief Medical Officer, Capricor Therapeutics Mark
Slide 4: Agenda
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Agenda Introduction Deramiocel Program Overview HOPE-3 Phase 3 Trial Overview HOPE-3 Patient Demographics HOPE-3 Safety Profile Overview HOPE-3 Efficacy Overview Conclusions and Next Steps Q&A 4
Slide 5: DMD: A Devastating Rare Disease
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside DMD: A Devastating Rare Disease High Unmet Needs Across the Entire Disease Trajectory Whole Muscle Tissue Dystrophin Muscle-Fiber Membrane Dystrophin is a structural protein located within the muscle fiber membrane
Slide 6: Deramiocel's Clinical Development
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel’s Clinical Development 10 Years of Development in DMD 6 HOPE-DUCHENNE1 Phase 1 HOPE-DUCHENNE1 Open label extension (OLE) HOPE-2 Phase 2 HOPE-2 OLE (Ongoing) HOPE-3 Phase 3 N = 25 IC infusion N = 8 IV inf
Slide 7: Deramiocel's Multi-Modal Mechanism
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel’s Multi-Modal Mechanism Anti-fibrotic1 Anti-inflammatory Immunomodulatory1 1de Couto et al., 2015 CDCs Exosomes and growth factors 0 20 40 60 80 100 Control CDC cxcl8 0 20 40 60 80 100 120 Collagen CDC 0
Slide 8: HOPE-3 Pivotal Phase Trial
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE-3 Pivotal Phase 3 Trial S C R E E N I N G R A N D O M I Z E Deramiocel 150 million cells IV infusion (quarterly) 1:1 (DERAMIOCEL TO PLACEBO) 12 months Placebo IV infusion (quarterly) Baseline ≤ 2d to D1 Design
Slide 9: Approaching Loss of Ambulation
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Approaching Loss of Ambulation Late-ambulatory patients with DMD and 10MWR > 10 sec 9 0 1 2 3 4 5 6 Performance of Upper Limb (PUL) Entry 2 to 6 (Target Population) 10 sec 10 MWR 100% lose ambulation within 24 mont
Slide 10: HOPE-3 Baseline Demographics
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Baseline Demographics Placebo (n=52) Deramiocel (n=54) Overall (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 2.0 Entry Item Score 2,
Slide 11: HOPE-3 Safety Profile Results
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Overview Placebo (n=52) n (%) Deramiocel (n=53) n (%) Overall (n=105) 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
Slide 12: HOPE-3: Primary Endpoint (PUL v2.0)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE-3: Primary Endpoint (PUL v2.0) Month 12 12 (n=53) (n=52)
Slide 13: HOPE-3: Key Secondary Endpoint (LVEF)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE-3: Key Secondary Endpoint (LVEF) Month 12 13 (n=39) (n=44)
Slide 14: HOPE-3 Topline Efficacy Results
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE-3 Topline Efficacy Results 14
Slide 15: Deramiocel has the Potential to Redefine the Standard of Care for Duchenne
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel has the Potential to Redefine the Standard of Care for Duchenne 15 Deramiocel Potential first-in-class therapy for DMD CARDIAC AND SKELETAL MUSCLE GENE THERAPIES EXON SKIPPING THERAPIES CORTICOSTEROIDS S
Slide 16: Conclusions and Next Steps
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Conclusions and Next Steps 16 • Pivotal Phase 3 study met the primary endpoint (PUL v2.0) and the key secondary cardiac endpoint (LVEF), both achieving statistical significance (p=0.03 and p=0.04) • Statistical sig
Slide 17: Acknowledgments
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Acknowledgments All patients and their families who participated in the HOPE studies • Craig McDonald, M.D. (UC Davis Health), HOPE-3 National PI • Jonathan Soslow, M.D., MSCI, (Vanderbilt University Medical Center
Slide 18: Thank you Questions
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Thank you Questions 18
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