Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (September 2024)
16 slides from Capricor Therapeutics's investor presentation, Sep 24, 2024
Slide 1: Capricor Therapeutics, Inc. Corporate Presentation
Linda Marbán, Ph.D. Chief Executive Officer Capricor Therapeutics, Inc. NASDAQ: CAPR September 24, 2024
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 discovery e
Slide 3: Committed to transformative treatments for patients in need
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3 At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for patients in need
Slide 4: Deramiocel for the Treatment of DMD: Pathway to BLA
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel for the Treatment of DMD Pathway to BLA ➢Capricor intends to submit a BLA for full approval ➢BLA filing supported with Capricor’s existing cardiac and available natural history data ➢Submission will seek
Slide 5: Key Takeaways from Pre-BLA Meeting
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 5 Key Takeaways from Pre-BLA Meeting ➢ Strength of Capricor’s long-term cardiac data ➢ Emergence of natural history cardiac dataset ➢ FDA’s continued commitment to rare diseases ➢ No approved therapies for DMD-card
Slide 6: Cardiomyopathy in DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Cardiomyopathy in DMD F rontiers | Ec hoc ardiogra phic Im ag e Q ual ity Deteri ora tes wi th A ge in Children a nd Young Adul ts with Duchenne M us cular D ystrophy (frontiers in.org) Steady course of decline in
Slide 7: Deramiocel Cell Therapy Overview
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 7 • Deramiocel(CAP-1002): biologic consisting of allogeneic cardiosphere-derived cells (CDCs) • Multiple-modalities • Immunomodulatory • Anti-inflammatory • Anti-fibrotic • Pro-angiogenic • Investigated in over 200
Slide 8: HOPE-2: 21/22 Cardiac Outcomes Favored Deramiocel Treatment over Placebo
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 8 HOPE-2: 21/22 Cardiac Outcomes Favored Deramiocel Treatment over Placebo Statistically significant treatment effect in critical measures of cardiac function Results published in The Lancet, March 2022.
Slide 9: Natural History Data Summary
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 9 Natural History Data Summary † “This study aims to focus on cardiomyopathy (heart muscle disease), which is the leading cause of death in Duchenne muscular dystrophy. The study will combine genetic differences wi
Slide 10: HOPE-2 OLE: 3-Year Cardiac Results, Ejection Fraction
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3.0 -5.0 3.1 -6 -5 -4 -3 -2 -1 0 1 2 3 4 Median Changes in Left Ventricular Ejection Fraction (cMRI) Median LVEF=54% (Range = 47% – 60%) n=8 Deramiocel treated Deramiocel treated HOPE-2 OLE: 3-Year Cardiac Results
Slide 11: HOPE-2 OLE: 3-Year Cardiac Results, End Systolic Volumes
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside -5.1 5.0 -6.0 -10 -8 -6 -4 -2 0 2 4 6 Median Changes in End Systolic Volumes-Indexed in cMRI Median LVEF =57% (IQR = 49% – 60%) n=37 Median LVEF=54% (Range = 47% – 60%) n=8 HOPE-2 OLE: 3-Year Cardiac Results End Sy
Slide 12: Deramiocel BLA Supporting Data To Support Full Approval
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel BLA Supporting Data To Support Full Approval 12 BLA Submission HOPE-2 Phase II HOPE-2 Open Label Extension Natural history data Vanderbilt University Medical Center (funded by OOPD) and Cincinnati Childr
Slide 13: Deramiocel has the Potential to Redefine the Standard of Care for DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Deramiocel has the Potential to Redefine the Standard of Care for DMD 13 Deramiocel First-in-class therapy for DMD-cardiomyopathy GENE THERAPIES Elevidys EXON SKIPPING THERAPIES Viltepso® Exondys 51 , Amondys 45 ,
Slide 14: Potential Expansion of Deramiocel
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Potential Expansion of Deramiocel Becker cardiomyopathy develops with a similar progression to DMD-cardiomyopathy Duchenne muscular dystrophy Becker muscular dystrophy Other orphan cardiomyopathies 14 Label expansi
Slide 15: Near-Term Value Driving Catalysts
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Near-Term Value Driving Catalysts 15 Aim to complete BLA submission ~Q4 2024 Key Regulatory & Clinical-Based Milestones Pre-BLA meeting Q3 2024 Plan to commence rolling BLA submission October 2024 Key U.S. Approval
Slide 16: Thank you, Q&A
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 16 Capricor Therapeutics, Inc. 10865 Road to the Cure, Suite 150 San Diego, California 92121 e: w: info@capricor.com www.capricor.com Nasdaq: CAPR Thank you Q&A
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