Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (October 2017)
25 slides from Capricor Therapeutics's investor presentation, Oct 3, 2017
Slide 1: Capricor Therapeutics
NASDAQ: CAPR October 2017 Capricor Therapeutics
Slide 2: Forward-Looking Statements
Forward - Looking Statements 2 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 efforts and clinical trials; the pace of enrollment of clinical trials; plans regar
Slide 3: Investment Highlights
Investment Highlights ‒ Pipeline focused on rare pediatric disorders for which current options are inadequate ‒ Clinical proof - of - concept demonstrated in lead indication ▪ Potential registration trial planned to initiate in 1Q18 * ▪ $1B+ U.S. sales opportunity ▪ Capricor holds worldwide IP right
Slide 4: Capricor's Product Pipeline
Capricor’s Product Pipeline 4 Candidate Indication Development Phase Status Preclinical Clinical Market CAP - 1002 (allogeneic CDCs) Duchenne Muscular Dystrophy ▪ Improvement in skeletal and cardiac muscle function shown in randomized clinical trial in advanced DMD ▪ Plan to initiate potential regis
Slide 5: Evolution of Capricor's Science and Clinical Development
Evolution of Capricor’s Science and Clinical Development 5
Slide 6: Capricor's Technology
Capricor’s Technology 6 ‒ Capricor’s core technology = cardiosphere - derived cells (CDCs) ▪ Unique population of cells which include cardiac progenitor cells with a distinct surface marker profile ▪ Subject of over 100 peer - reviewed scientific publications ▪ CAP - 1002 (allogeneic CDCs) has been
Slide 7: Exerts its Actions via Extracellular Vesicles (EVs)
CAP - 1002 Exerts its Actions via Extracellular Vesicles (EVs) 7 — Exosomes are nano - scale extracellular vesicles released by most cells ▪ Charged with a variety of biomolecules, including RNAs and proteins ▪ Emerging as major players in cell - to - cell communication cardiosphere - derived cells
Slide 8: Duchenne Muscular Dystrophy
CAP - 1002 for Duchenne Muscular Dystrophy 8 ‒ CAP - 1002’s initial market opportunity is for the treatment of DMD ▪ Rare pediatric disorder – WW incidence ~1 / 3,600 male births ▪ Progressive muscle weakness with eventual loss of function starting in early childhood – Affects skeletal, cardiac, and
Slide 9: Progressive Muscle Wasting Leads to Weakness, Loss of Motor Function and Early Death
Progressive Muscle Wasting Leads to Weakness, Loss of Motor Function and Early Death 9
Slide 10: Treatment and Management Options are Limited
Treatment and Management Options are Limited 10 Glucocorticosteroids ▪ Anti - inflammatory ▪ Slow muscle degeneration ▪ Delay progression ▪ Onerous, permanent side effects Exondys 51 ( eteplirsen ) ▪ “Exon - skipping drug” approved in 2016 ▪ Dystrophin expression via read - through ▪ Addresses only
Slide 11: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes Muscle to Damage 11 • X - linked genetic disorder • Nearly all affected are male • Dystrophin is a structural protein in muscle • Acts both as a cushion and a kind of glue • Without dystrophin, muscles are unable to function properly, suffer progressive damage and even
Slide 12: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model 12 ‒ The mdx mouse is a standard experimental model of DMD ▪ Unable to express dystrophin protein due to genetic mutation, the same causative defect in human DMD ▪ Exhibits a DMD - like phenotype ‒ Following a single administration of CDC or vehicle to mdx mice: ▪
Slide 13: Physiological Effects of CDC in mdx Model
Physiological Effects of CDC in mdx Model 13 Oxidative Stress Fibrosis Inflammation Mitochondrial Integrity Muscle Cell Generation phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2 GCLC cat. sub. phospho - IkB p65 (nuclear) MCP1 CD68+ macrophages CD3+ T
Slide 14: Phase I/II HOPE-Duchenne Clinical Trial
Phase I / II HOPE - Duchenne Clinical Trial 14 CAP - 1002 (1 x 75M cells) Usual Care n=12 25 patients Ages 12 and older DMD heart disease n=13 6 — Trial population characterized by advanced disease: CAP - 1002 given by intracoronary infusion ▪ all are receiving steroids ▪ majority are non - ambulant
Slide 15: PUL Results Indicated Skeletal Muscle Benefit
PUL Results Indicated Skeletal Muscle Benefit 15 ‒ The Performance of the Upper Limb (PUL) test was designed specifically for use in the DMD setting ‒ Individual tasks represent real - world activities of daily living (ns) improvement Thought leaders in DMD with whom we have shared these data view t
Slide 16: Cardiac Functional Measures Improved in Patients
Cardiac Functional Measures Improved in CAP - 1002 Patients 16 improvement improvement Spontaneous scar reduction is not normally observed in DMD Systolic thickening is believed to be a principal mechanism of cardiac output generation in DMD Month 6 (ns) (ns) (ns) (ns)
Slide 17: Cardiac and Skeletal Muscle Effects Seen in the Same Patients
Cardiac and Skeletal Muscle Effects Seen in the Same Patients 17 Inferior Wall Thickening + Middle - Level PUL Scar Reduction + Middle - Level PUL * Concordance of improvements support a treatment effect p - values:
Slide 18: Key Conclusions from Six-Month HOPE Data
Key Conclusions from Six - Month HOPE Data — Results support the potential benefit of CAP - 1002 to boys and young men with advanced Duchenne muscular dystrophy — Signal of skeletal muscle improvement (PUL) — Signal of cardiac muscle improvement (wall thickening) — Approx. three month duration of ef
Slide 19: Plan to Initiate HOPE-2 Clinical Trial in 1Q18
▪ Randomized, double - blind, placebo - controlled ▪ Peripheral intravenous delivery – supported by preclinical studies ▪ Repeat - dose design – potential to achieve sustained benefit ▪ Primary efficacy endpoint to be based on the PUL test at six months ▪ Principal Investigator – Craig M. McDonald,
Slide 20: Manufacturing
Manufacturing 20 ‒ CAP - 1002 is manufactured from donor hearts via a proprietary process ‒ Clinical trial material currently produced at Capricor facility ‒ High - yield process in advanced development ‒ Selection of CMO in process for anticipated early - stage commercial manufacture CSps CDCs Wash
Slide 21: Commercial Outlook in DMD
Commercial Outlook for CAP - 1002 in DMD 21 — DMD prevalence: U.S. ~15,000 Europe ~20,000 ▪ WW incidence ~ one per 3,600 live male births — CAP - 1002 positioned to have broad treatment potential ▪ Acts upon multiple pathways that contribute to DMD disease process ▪ Lack of mutation dependence due t
Slide 22: Capricor has Assembled a World-Class DMD Advisory Board
Capricor has Assembled a World - Class DMD Advisory Board 22 Barry Byrne, M.D., Ph.D. University of Florida (USA) Michelle Eagle, Ph.D., M.Sc., MCSP Atom International Ltd (UK) Richard Finkel , M.D. Nemours Children's Hospital (USA) Pat Furlong Parent Project Muscular Dystrophy (USA) Kan Hor , M.D.
Slide 23: Relationships with Key DMD Advocacy Organizations
Relationships with Key DMD Advocacy Organizations 23
Slide 24: Current Resources Expected to Fund Operations Through 2Q18
Current Resources Expected to Fund Operations Through 2Q18 24 Capricor has received over $30 million in competitive grants and a loan award from: California Institute of Regenerative Medicine National Institutes of Health U.S. Department of Defense Cash and cash equivalents $12.3 million (as of 6/30
Slide 25: Recent and Upcoming Milestones in Duchenne Muscular Dystrophy
Recent and Upcoming Milestones 25 CAP - 1002 in Duchenne Muscular Dystrophy x April 2017: Reported positive top - line six - month results of HOPE trial x July 2017: Granted Rare Pediatric Disease Designation x July 2017: Announced results of FDA meeting x September 2017: Announced Craig McDonald, M
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