Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (October 2017)
25 slides from Capricor Therapeutics's investor presentation, October 2017
Slide 1: Capricor Therapeutics
Capricor Therapeutics NASDAQ: CAPR October 2017
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 discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding
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 rights ‒
Slide 4: Capricor's Product Pipeline
Capricor’s Product Pipeline Development Phase Candidate Indication Status Preclinical Clinical Market ▪ Improvement in skeletal and cardiac muscle function shown in randomized clinical trial in advanced DMD CAP-1002 Duchenne Muscular (allogeneic CDCs) Dystrophy ▪ Plan to initiate potential registrat
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 ‒ 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 evaluate
Slide 7: CAP-1002 Exerts its Actions via Extracellular Vesicles (EVs)
CAP-1002 Exerts its Actions via Extracellular Vesicles (EVs) — 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 Skeletal muscle: Exercise tolerance
Slide 8: CAP-1002 for Duchenne Muscular Dystrophy
CAP-1002 for Duchenne Muscular Dystrophy ‒ 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 respi
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 Glucocorticosteroids ▪ Anti-inflammatory ▪ Slow muscle degeneration Medication ▪ Delay progression ▪ Onerous, permanent side effects Physical Assistive Exondys 51 (eteplirsen) ▪ “Exon-skipping drug” approved in 2016 Therapy Devices ▪ Dystrophin expression
Slide 11: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes Muscle to Damage • 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 eventually die • X-linked genetic disorder • Nearly all affected are
Slide 12: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model ‒ 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: ▪ Exerc
Slide 13: Physiological Effects of CDC in mdx Model
Physiological Effects of CDC in mdx Model phospho-Akt HO-1 GCLC cat. sub. Oxidative Nrf2 (cytoplasmic) Stress catalase SOD-2 Nrf2 (nuclear) phospho-IkB CD68+ macrophages Inflammation NF-kB p65 (nuclear) MCP1 CD3+ T cells collagen I Fibrosis collagen III Mitochondrial
Slide 14: Phase I / II HOPE-Duchenne Clinical Trial
Phase I / II HOPE-Duchenne Clinical Trial CAP-1002 (1 x 75M cells) 25 patients n=13 Ages 12 and older 6 12 DMD heart disease Usual Care top-line interim data n=12 reported in April 2017 CAP-1002 given by intracoronary infusion — Trial population characterized by advanced disease: ▪ all are receiving
Slide 15: PUL Results Indicated Skeletal Muscle Benefit
PUL Results Indicated Skeletal Muscle Benefit ‒ 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 improvement Thought leaders in DMD with whom we have shared these data view them as ind
Slide 16: Cardiac Functional Measures Improved in CAP-1002 Patients
Cardiac Functional Measures Improved in CAP-1002 Patients improvement improvement (ns) (ns) (ns) (ns) Month 6 Spontaneous scar reduction is Systolic thickening is believed to be a principal not normally observed in DMD mechanism of cardiac output generation in DMD 16
Slide 17: Cardiac and Skeletal Muscle Effects Seen in the Same Patients
Cardiac and Skeletal Muscle Effects Seen in the Same Patients p-values: Concordance of improvements support a treatment effect Inferior Wall Thickening Scar Reduction + Middle-Level PUL + Middle-Level PUL* 17
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 effect
Slide 19: Plan to Initiate HOPE-2 Clinical Trial of CAP-1002 in 1Q18
Plan to Initiate HOPE-2 Clinical Trial of CAP-1002 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
Slide 20: Manufacturing
Manufacturing CSps CDCs Wash Formulate Fill CAP-1002 ‒ 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 commerc
Slide 21: Commercial Outlook for CAP-1002 in DMD
Commercial Outlook for CAP-1002 in DMD — 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 to downs
Slide 22: Capricor has Assembled a World-Class DMD Advisory Board
Capricor has Assembled a World-Class DMD Advisory Board 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. Nationw
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 Cash and cash equivalents $12.3 million (as of 6/30/17) Shares outstanding 23.5 million (as of August 10, 2017) Capricor has received over $30 million in competitive grants and a loan award from: California Institute of Regenerative Medicine
Slide 25: Recent and Upcoming Milestones
Recent and Upcoming Milestones CAP-1002 in Duchenne Muscular Dystrophy ✓ April 2017: Reported positive top-line six-month results of HOPE trial ✓ July 2017: Granted Rare Pediatric Disease Designation ✓ July 2017: Announced results of FDA meeting ✓ September 2017: Announced Craig McDonald, M.D. to le
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