Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (May 2018)
26 slides from Capricor Therapeutics's investor presentation, May 4, 2018
Slide 1: Capricor Therapeutics, May 2018
Feapricor herapeutics™ May 2018
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 re
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 Innovative, Proprietary Therapeutic Platforms Capital Efficiency Strong Scientific Foundation & Leadership Team ▪ First - in - class biologics with potential to improve cardiac and skeletal muscle ▪ Product candidates based on cells and extracellular vesicles (exosomes) ▪ Enroll
Slide 5: Capricor's Product Pipeline
Capricor’s Product Pipeline 5 Candidate Indication Development Phase Status Preclinical Clinical Market CAP - 1002 (allogeneic CDCs) Duchenne Muscular Dystrophy ▪ HOPE - 2 trial enrolling ▪ Improvement in skeletal and cardiac muscle function seen in randomized clinical trial in advanced DMD ▪ Orphan
Slide 6: 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 CAP - 1002: Duchenne Muscular Dystrophy Program 6
Slide 7: Lack of Dystrophin Predisposes Muscle to Damage
Lack of Dystrophin Predisposes Muscle to Damage 7 — 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 dystroph
Slide 8: Treatment Options for DMD are Limited
Treatment Options for DMD are Limited 8 Challenges ▪ EXONDYS 51 – 13% of DMD population ▪ Gene therapy not always effective due to antibody response ▪ Steroids have adverse long term side - effects ▪ NF - kB inhibition may not be enough to mediate immune response ▪ Exon Skipping ▪ Gene therapy ▪ Utr
Slide 9: 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 » Internalized by target ce
Slide 10: Cardiomyopathy is the #1 Cause of Death in DMD
Cardiomyopathy is the #1 Cause of Death in DMD 10 ‒ Lack of functional dystrophin in heart leads to: ‒ Hearts become dilated and non - compliant, and eventually fail ‒ No therapies have been shown to address the heart disease associated with DMD inflammation cardiomyocyte death progressive cardiac f
Slide 11: Effects of CDCs in mdx Mouse Model: Cardiac Function
Effects of CDCs in mdx Mouse Model 11 ‒ Following a single administration of CDC or vehicle to mdx mice: Aminzadeh et al, Stem Cell Reports 2018 ( http://www.cell.com/stem - cell - reports/fulltext/S2213 - 6711(18)30049 - 3 ). Improved cardiac function Increased exercise capacity ▪ Left ventricular
Slide 12: Effects of CDCs in mdx Mouse Model: Skeletal Muscle
Effects of CDCs in mdx Mouse Model 12 ‒ 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
Slide 13: Mechanism of Action Defined in Stem Cell Reports
Mechanism of Action Defined in “Stem Cell Reports” 13 Oxidative Stress Fibrosis Inflammation Cellular Energy 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 EF > 35% Stable steroids n=13 6 CAP - 1002 given by intracoronary infusion 12 Data presented at AHA 2017 late - breaking session ▪ One - time, multi - vessel, int
Slide 15: Baseline Characteristics
Baseline Characteristics 15 Usual Care (n=12) CAP - 1002 (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
Slide 16: Heart Muscle: Reduced Myocardial Scarring
Heart Muscle: Reduced Myocardial Scarring 16 ‒ Assessed by cardiac MRI with blinded analysis by core lab ‒ Scar increased in the Usual Care group, but decreased in the CAP - 1002 group ▪ 11.9% group difference in change score at Month 12 (p=0.03) ‒ Decreased scar is counter to the natural history of
Slide 17: Heart Muscle: Increased Regional Systolic Wall Thickening
Heart Muscle: Increased Regional Systolic Wall Thickening 17 ‒ Measurement of cardiac function by MRI allows focus on treated areas ‒ Magnitude of scar reduction is consistent with natural history of scar progression in DMD ▪ Inferior → Anterior → Lateral → Septal ‒ Measure is important indicator of
Slide 18: Skeletal Muscle: PUL Results Indicate Functional Benefit
18 Skeletal Muscle: PUL Results Indicate Functional Benefit ‒ 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 observed throughout follow - up Middle + Distal PUL Score %
Slide 19: 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 benefit ▪ Primary efficacy endpoin
Slide 20: Manufacturing
20 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 f
Slide 21: Exosomes (CAP-2003): A New Regenerative Medicine Platform
Exosomes (CAP - 2003) A New Regenerative Medicine Platform 21 Potential Benefits of Exosomes vs. Cells ▪ Extended shelf life (lyophilize) ▪ Penetrate cells and tissues not readily accessed by cells ▪ Reduced manufacturing costs
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 bro
Slide 23: Senior Management
Senior Management AJ Bergmann, MBA Chief Financial Officer Deborah Ascheim, MD Chief Medical Officer Karen Krasney, JD EVP & General Counsel Rachel Smith, PhD VP of Research & Development Luis Rodriguez - Borlado, PhD VP of Regenerative Therapies Linda Marbán, PhD Chief Executive Officer, Co - found
Slide 24: World-Class DMD Advisory Board
Capricor has Assembled a World - Class DMD Advisory Board 24 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. Na
Slide 25: Current Resources Expected to Fund Operations into 1Q19
Current Resources Expected to Fund Operations into 1Q19 25 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 equivalents $14.1 million (as of Dec. 31, 201
Slide 26: Anticipated Milestones
Anticipated Milestones Recent Accomplishments 26 Potential Near Term Catalysts Clinical Development ▪ Announced positive 6 and 12 month data from HOPE - Duchenne ▪ HOPE - 2 IND cleared Regulatory ▪ Granted orphan drug designation for DMD ▪ Granted rare pediatric designation for DMD ▪ Granted RMAT de
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