Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (May 2018)
26 slides from Capricor Therapeutics's investor presentation, May 2018
Slide 1: Capricor Therapeutics - NASDAQ: CAPR May 2018
Feapricor herapeutics”
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: Capricor's Mission
Capricor’s Mission Capricor is focused on the discovery, development and commercialization of innovative cell and exosome based therapies for patients with immune-inflammatory rare diseases with a focus on Duchenne muscular dystrophy. 3
Slide 4: Corporate Highlights
Corporate Highlights First-in-class biologics with potential to improve cardiac Innovative, Proprietary and skeletal muscle Therapeutic Platforms Product candidates based on cells and extracellular vesicles (exosomes) Enrolling HOPE-2, potential registration trial in DMD Advanced Pipeline Co
Slide 5: Capricor's Product Pipeline
Capricor’s Product Pipeline Development Phase Candidate Indication Status Preclinical Clinical Market HOPE-2 trial enrolling Improvement in skeletal and cardiac CAP-1002 Duchenne muscle function seen in randomized (allogeneic CDCs) Muscular Dystrophy clinical trial in advanced DMD Orphan Drug,
Slide 6: CAP-1002: Duchenne Muscular Dystrophy Program
CAP-1002: Duchenne Muscular Dystrophy Program ― X-linked genetic disorder ― Incidence: ~1 per 3,600 male births ― Prevalence: US: ~15,000-20,000, WW: ~200,000 6
Slide 7: 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 — Much of the muscle injury that occurs in dystrophin
Slide 8: Treatment Options for DMD are Limited
Treatment Options for DMD are Limited Challenges Exon Skipping Immunomodulatory EXONDYS 51 –13% of DMD population Gene therapy Gene therapy not always effective Anti-fibrotic due to antibody response Utrophin Steroids have adverse long term Pro-angiogenic side-effects NF-kB NF-kB i
Slide 9: Capricor's CAP-1002 Technology
Capricor’s CAP-1002 Technology ‒ CAP-1002 is a biologic consisting of allogeneic cardiosphere-derived cells (CDCs) Manufactured from donated heart muscle Does not act by “stemness” – the cells do not engraft into host tissue MOA: cells secrete exosomes Contain non-coding RNAs and proteins
Slide 10: Cardiomyopathy is the #1 Cause of Death in DMD
Cardiomyopathy is the #1 Cause of Death in DMD ‒ Lack of functional dystrophin in heart leads to: inflammation cardiomyocyte death progressive cardiac fibrosis ‒ Hearts become dilated and non-compliant, and eventually fail ‒ No therapies have been shown to address the heart disease associated with D
Slide 11: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model ‒ Following a single administration of CDC or vehicle to mdx mice: Improved cardiac function Increased exercise capacity Left ventricular ejection fraction Exercise performance markedly improved vs. control approximately doubled vs. control (p<0.05 at all timep
Slide 12: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model ‒ Following a single administration of CDC or vehicle to mdx mice: Enhanced skeletal muscle function Twitch force, tetanic force, and fibrosis in soleus (slow-twitch) and extensor digitorum longus (fast-twitch) muscles significantly improved vs. control (p<0.05;
Slide 13: Mechanism of Action Defined in Stem Cell Reports
Mechanism of Action Defined in “Stem Cell Reports” 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 mitoc
Slide 14: Phase I / II HOPE-Duchenne Clinical Trial
Phase I / II HOPE-Duchenne Clinical Trial CAP-1002 25 patients (1 x 75M cells) Ages 12 and older n=13 DMD heart disease 6 12 EF > 35% Stable steroids Usual Care n=12 Data presented at AHA 2017 late-breaking session CAP-1002 given by intracoronary infusion One-time, multi-vessel, intracoronary deli
Slide 15: Baseline Characteristics
Baseline Characteristics Usual Care CAP-1002 (n=12) (n=13) Age, median years (range) 17.5 (12–20) 18 (14–25) Wheelchair Use Always (%) 7 (58) 10 (77) Cardiac Scar Size, mean % (SD) 21.4 (10.8) 17.6 (6.8) LV Ejection Fraction, mean % (SD) 48.4 (7.5) 49.6 (6.7) Intracoronary Dose, M cells (SD) n/a 73.
Slide 16: Heart Muscle: Reduced Myocardial Scarring
Heart Muscle: Reduced Myocardial Scarring ‒ Assessed by cardiac MRI with blinded analysis by core lab ‒ Scar increased in the Usual Care group, but decreased in the CAP-1002 group IMPROVEMENT 11.9% group difference in change score at Month 12 (p=0.03) p=0.09 p=0.03 ‒ Decreased scar is counter to t
Slide 17: Heart Muscle: Increased Regional Systolic Wall Thickening
Heart Muscle: Increased Regional Systolic Wall Thickening Measurement of cardiac function by MRI INFERIOR WALL ‒ IMPROVEMENT allows focus on treated areas ‒ Magnitude of scar reduction is consistent with natural history of scar progression in p=0.04 p=0.09 DMD ANTERIOR Inferior → Anterior → Latera
Slide 18: Skeletal Muscle: PUL Results Indicate Functional Benefit
Skeletal Muscle: PUL Results Indicate Functional Benefit Middle + Distal PUL Score % change from baseline IMPROVEMENT ‒ Performance of the Upper Limb (PUL) test is a validated instrument in DMD Relates to patients’ ability to perform common activities of daily living ‒ Trends towards improvement o
Slide 19: HOPE-2 Clinical Trial of CAP-1002 in DMD
HOPE-2 Clinical Trial of CAP-1002 in DMD — HOPE-2 – currently enrolling Randomized, double-blind, placebo-controlled Target enrollment of 84 patients with advanced disease Peripheral intravenous delivery – supported by preclinical studies Repeat-dose design – potential to achieve sustained b
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 ‒ Previous 3-year collaboration with Janssen Biotech focused on c
Slide 21: Exosomes (CAP-2003) A New Regenerative Medicine Platform
Exosomes (CAP-2003) A New Regenerative Medicine Platform Potential Benefits of Exosomes vs. Cells Extended shelf life (lyophilize) Penetrate cells and tissues not readily accessed by cells Reduced manufacturing costs 21
Slide 22: Extracellular Vesicles (Exosomes): Cell Free Regenerative Medicine
Extracellular Vesicles (Exosomes): Cell Free Regenerative Medicine – Extracellular vesicles - term for cell-derived vesicles, includes exosomes – Nanometer-sized lipid-bilayer vesicles – Rich in RNAs and proteins – Secreted by nearly all cell types – Cell signaling modality – Potential for broad the
Slide 23: Senior Management
Senior Management Linda Marbán, PhD AJ Bergmann, MBA Chief Executive Officer, Co-founder and Director Chief Financial Officer Under her direction, Capricor has secured over $45 million in non-dilutive funding and over $50 million in equity capital. Earlier in her career, Dr. Marbán was with Mr. Berg
Slide 24: 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 25: Current Resources Expected to Fund Operations into 1Q19
Current Resources Expected to Fund Operations into 1Q19 Cash and equivalents $14.1 million (as of Dec. 31, 2017) Shares outstanding 27.7 million (as of Mar. 20, 2018) Capricor reported the above information in its most recent Annual Report on Form 10-K filed with the SEC on March 22, 2018. Capricor
Slide 26: Anticipated Milestones
Anticipated Milestones Recent Accomplishments Potential Near Term Catalysts Clinical Development Pipeline Updates Announced positive 6 and 12 month data from Currently enrolling HOPE-2 trial HOPE-Duchenne Planning to publish HOPE-Duchenne study HOPE-2 IND cleared Planning to file IND for e
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