Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (December 2017)
26 slides from Capricor Therapeutics's investor presentation, Dec 19, 2017
Slide 1: Capricor Therapeutics, December 2017
Feapricor herapeutics™ NASDAQ: CAPR December 2017
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: Investment Highlights
Investment Highlights ‒ Pipeline focused on rare pediatric disorders for which current options are inadequate ‒ Clinical proof - of - concept in lead indication – Duchenne muscular dystrophy ▪ To initiate potential registration trial in 1Q18 ▪ $1B+ U.S. sales opportunity ▪ Capricor holds worldwide I
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 ▪ Expect to initiate potential registration trial in 1Q18 ▪ Improvement in skeletal and cardiac muscle function seen in randomized clinical
Slide 5: DMD Progression is Sequential, Non-Linear and Irreversible
DMD Progression is Sequential, Non - Linear and Irreversible 5
Slide 6: Cardiomyopathy is the #1 Cause of Death in DMD
Cardiomyopathy is the #1 Cause of Death in DMD 6 ‒ DMD results from mutation in dystrophin gene, ~1 per 3,600 male births ‒ 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 as
Slide 7: Capricor's Technology
‒ CAP - 1002 is a biologic product 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 ▪ Acts by releasing extracellular vesicles, or exosomes » Contain non - coding RNAs and
Slide 8: Targets Multiple Disease Processes in DMD
damage to cell membrane CAP - 1002 Targets Multiple Disease Processes in DMD 8 CAP - 1002 dystrophin deficiency c ellular [Ca 2+ ] myocyte loss nitrosoactive stress inflammation mitochondrial inefficiency fibrosis microvascular ischemia
Slide 9: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model 9 ‒ Following a single administration of CDC or vehicle to mdx mice: Aminzadeh et al, 2017 ( http://biorxiv.org/content/early/2017/04/20/128900 ). Improved cardiac function Increased exercise capacity ▪ Left ventricular ejection fraction markedly improved vs. contr
Slide 10: Effects of CDCs in mdx Mouse Model
Effects of CDCs in mdx Mouse Model 10 ‒ Following a single administration of CDC or vehicle to mdx mice: Aminzadeh et al, 2017 ( http://biorxiv.org/content/early/2017/04/20/128900 ). Enhanced skeletal muscle function ▪ Twitch force, tetanic force, and fibrosis in soleus (slow - twitch) and extensor
Slide 11: Physiological Effects of CDC in mdx Model
Physiological Effects of CDC in mdx Model 11 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 cells collagen I collage
Slide 12: Phase I/II HOPE-Duchenne Clinical Trial
Phase I / II HOPE - Duchenne Clinical Trial 12 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 13: Baseline Characteristics
Baseline Characteristics 13 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 14: Heart Muscle: Reduced Myocardial Scarring
Heart Muscle: Reduced Myocardial Scarring 14 ‒ 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 15: Heart Muscle: Increased Regional Systolic Wall Thickening
Heart Muscle: Increased Regional Systolic Wall Thickening 15 ‒ Greatest evidence of improvement seen in inferior wall ‒ Similar trend in anterior wall ‒ Lesser trends in lateral and septal walls ‒ Consistent with natural history of scar progression in DMD ▪ Inferior → Anterior → Lateral → Septal ‒ N
Slide 16: Measuring DMD Progression Requires Multiple Outcome Measures
Measuring DMD Progression Requires Use of Multiple Outcome Measures 16 Muscle Function ▪ 6 - minute walk test ▪ 4 - stair climb ▪ 4 - stair descend ▪ 10m walk / run Disease Progression ▪ North Star Ambulatory Assessment (NSAA) ▪ Loss of Ambulation Upper Limb Impairment ▪ Brooke / EK Scales ▪ Perform
Slide 17: Skeletal Muscle: PUL Results Indicate Functional Benefit
17 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 18: Skeletal Muscle: PUL Results Indicate Functional Benefit
18 Skeletal Muscle: PUL Results Indicate Functional Benefit Patients (%) ‒ Middle + Distal PUL scores ‒ Subgroup of patients with more advanced disease (baseline middle + distal PUL score < 55 of 58 max) ‒ Responder defined as: ≥ 10% improvement from baseline or max possible improvement Post Hoc Res
Slide 19: Key Conclusions from HOPE Trial Results
Key Conclusions from HOPE Trial Results — Early clinical data consistent with preclinical studies showing CAP - 1002 benefits both cardiac (scar & thickening) and skeletal (PUL) muscle in DMD — CAP - 1002 (75M cells) generally safe and well - tolerated ▪ Adverse events consistent with an intracorona
Slide 20: HOPE-2 Clinical Trial in DMD
— Potential registration trial to initiate in 1Q 2018 ▪ 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 ▪ Pri
Slide 21: IV Delivery Studies in mdx Mouse
21 IV Delivery Studies in mdx Mouse Exercise capacity of mdx mice given CDCs by two IV administration routes. (n=4) (n=4) (n=6) Exercise capacity of mdx mice given escalating doses of CDCs by IV administration. (n=4) (n=8) (n=10) (n=4) Results presented at poster session at the Cell and Gene Meeting
Slide 22: Manufacturing
22 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
Slide 23: Capricor has Assembled a World-Class DMD Advisory Board
Capricor has Assembled a World - Class DMD Advisory Board 23 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 24: Relationships with Key DMD Advocacy Organizations
Relationships with Key DMD Advocacy Organizations 24
Slide 25: Resources Expected to Fund Operations Through 3Q18
Resources Expected to Fund Operations Through 3Q18 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 $13.9 million (as of Sept. 30, 20 17)
Slide 26: Recent and Upcoming Milestones
Recent and Upcoming Milestones 26 4Q 2017 Presented six - month HOPE results at World Muscle Society Congress 4Q 2017 Presented 12 - month HOPE results at AHA Late - Breaking Session 4Q 2017 Announced IND clearance to conduct HOPE - 2 Trial 1Q 2018 Expect to submit request for RMAT designation for C
Capricor Therapeutics — Investor Presentation (December 2017) on 6ix