Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (June 2017)
25 slides from Capricor Therapeutics's investor presentation, Jun 7, 2017
Slide 1: A Translational Medicine Company
NASDAQ: CAPR June 2017 a translational m edicine c ompany
Slide 2: 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 regarding regulatory filings, f
Slide 3: Capricor's Innovative Product Pipeline
3 Capricor’s Innovative Product Pipeline Product Candidate Indication Development Phase Status Preclinical I II III CAP - 1002 ( allogeneic CDC) Duchenne Muscular Dystrophy ▪ Positive six - month results from randomized clinical trial of single - dose, intra - coronary CAP - 1002 recently reported ▪
Slide 4: Clinical Data Support Opportunity in Heart Failure
4 Clinical Data Support CAP - 1002’s Opportunity in Heart Failure — Recent interim analysis of Phase II ALLSTAR trial in people who had suffered a myocardial infarction (“heart attack”) showed low probability of CAP - 1002 achieving primary endpoint of scar size reduction, however: Scar data in pati
Slide 5: Cell Therapy Program for Duchenne Muscular Dystrophy
5 Cell Therapy Program for Duchenne Muscular Dystrophy
Slide 6: Duchenne Muscular Dystrophy (DMD)
6 Duchenne Muscular Dystrophy (DMD ) Functional decline of ambulation in childhood Loss of walking ability by early teenage years Functional decline of arm function in adolescence Ventilatory support required by late teens Average life expectancy of less than 30 years DMD is a rare genetic disorder
Slide 7: DMD is an Ultra-Orphan Market Opportunity
7 DMD is an Ultra - Orphan Market Opportunity — Annual WW incidence estimated to be one per 3,500 – 5,000 male births ▪ Estimated 15,000 – 20,000 cases in U.S. — FDA - approved drugs limited to corticosteroids and eteplirsen ▪ Steroids uniquely shown to have some ability to improve function, but are
Slide 8: Capricor is Developing CAP-1002 for the Treatment of DMD
8 Capricor is Developing CAP - 1002 for the Treatment of DMD — CAP - 1002 is an “off the shelf” cell therapeutic candidate ▪ CDCs have been the subject of >100 peer - reviewed scientific reports ▪ E xerts potent anti - inflammatory, anti - fibrotic, and anti - apoptotic activities ▪ Supports the abi
Slide 9: Phase I/II HOPE-Duchenne Clinical Trial – Ongoing
9 Phase I / II HOPE - Duchenne Clinical Trial – Ongoing CAP - 1002 (1 x 75M cells) Usual Care n=12 25 patients Ages 12 and older DMD heart disease n=13 6 12 — Trial population characterized by advanced disease: CAP - 1002 given by intracoronary infusion ▪ a ll are receiving steroids ▪ majority are n
Slide 10: Safety and Tolerability
10 Safety and Tolerability — CAP - 1002 was generally safe and well - tolerated — N o early study discontinuations due to adverse events — M ajority of treatment - emergent adverse events rated as mild or moderate — No clinical events associated with peri - procedural cardiac enzyme elevations
Slide 11: Exploratory Efficacy Endpoints
11 Exploratory Efficacy Endpoints Baseline Week 6 Month 3 Month 6 Month 12 Imaging Cardiac magnetic resonance ( cMRI ) ● ● ○ Functional Tests Performance of the Upper Limb (PUL) ● ● ● ● ○ 6 - Minute Walk Test ● ● ● ● ○ Spirometry ● ● ● ● ○ Quality of Life Pediatric QL Inventory ● ● ● ● ○ PODCI Adole
Slide 12: Reduced Cardiac Scar in Treated Patients
12 Reduced Cardiac Scar in CAP - 1002 Treated Patients Spontaneous scar reduction is not observed in DMD improvement
Slide 13: Improved Systolic Wall Thickening in Treated Patients
13 Improved Systolic Wall Thickening in CAP - 1002 Treated Patients Systolic thickening is believed to be a principal mechanism of cardiac output generation in DMD improvement (ns) (ns)
Slide 14: Results of Ejection Fraction and Cardiac Volumes
14 Results of Ejection Fraction and Cardiac Volumes Patients had near - normal ejection fraction and low volumes at baseline improvement improvement improvement (ns) (ns) (ns)
Slide 15: Performance of the Upper Limb (PUL) Test
15 Performance of the Upper Limb (PUL ) Test
Slide 16: Mid- and Distal-Level PUL Results
16 Mid - and Distal - Level PUL Results improvement (ns) (ns) (ns)
Slide 17: Responder Analysis on Mid- and Distal-Level PUL
17 Responder Analysis on Mid - and Distal - Level PUL P otential functional improvement in skeletal muscle improvement Responder defined as ≥ 10% or max possible improvement (ns) (ns)
Slide 18: Cardiac and Skeletal Muscle Effects Seen in the Same Patients
18 Cardiac and Skeletal Muscle Effects Seen in the Same Patients Inferior Wall Thickening + Middle - Level PUL Scar Reduction + Middle - Level PUL Supportive of a treatment effect p - values: * Analysis excluded one subject due to uninterpretable imaging data. *
Slide 19: Key Conclusions from Six-Month HOPE Data
19 Key Conclusions from Six - Month HOPE Data — Data support the potential benefit of CAP - 1002 to boys and young men with advanced Duchenne muscular dystrophy — CAP - 1002 was generally safe and well - tolerated — Limited duration of effect following a single dose indicates that sustained benefit
Slide 20: Next Steps for Capricor's Duchenne Muscular Dystrophy Program
20 Next Steps for Capricor’s Duchenne Muscular Dystrophy Program — FDA meeting set for late June to review HOPE data and discuss development toward potential registration — Plan to apply for Regenerative Medicine Advanced Therapy (RMAT) designation — Repeat dosing via intravenous delivery anticipate
Slide 21: Manufacturing
21 CAP - 1002 Manufacturing — “Off - the - shelf” product — Expect to be able to generate thousands of doses per donor heart via commercial process in development with Janssen Biotech — 3 - year storage life CSps CDCs Wash Formulate Fill CAP - 1002
Slide 22: Exosome Program: Next-Gen Class of Biological Therapies
22 Exosome Program “Next - Gen” Class of Biological Therapies
Slide 23: Exosomes – a Cell-Free Therapeutic Technology
23 Exosomes – a Cell - Free Therapeutic Technology e ndothelial cells fibroblasts cardiomyocytes leukocytes CDC CDC exosomes
Slide 24: Financial Summary
24 Financial Summary Capricor has received over $30 million in competitive grant and loan awards from: California Institute of Regenerative Medicine National Institutes of Health U.S. Department of Defense Cash and cash equivalents * $11.7 million (as of 3/31/17) Shares outstanding 22.6 million (as
Slide 25: Recent and Upcoming Milestones
25 Clinical Programs CAP - 1002 in Duchenne Muscular Dystrophy x April 2017: Reported top - line six - month results of HOPE trial Late June 2017: Type B meeting with FDA scheduled 2H 2017: Plan to initiate clinical trial of repeat - dose, intravenous CAP - 1002 * CAP - 1002 in Adult Heart Disease x
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