Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (February 2017)
31 slides from Capricor Therapeutics's investor presentation, Feb 15, 2017
Slide 1: Corporate Overview February 2017
Corporate Overview February 2017 A Translational Medicine Company NASDAQ: CAPR
Slide 2: Forward-Looking Statements
2 This presentation contains forward - looking statements and information that are based on the beliefs of the management of Capricor Therapeutics, Inc . (Capricor) as well as assumptions made by and information currently available to Capricor . All statements other than statements of historical fac
Slide 3: Transformative Therapies from Bench to Bedside
3 Transformative Therapies from Bench to Bedside Capricor’s mission is to serve patients through products that promote the body’s own repair systems to address cardiac and other medical conditions
Slide 4: Investment Highlights
4 Investment Highlights Innovative, Proprietary Therapeutic Platforms Capital Efficiency Strong Scientific Foundation & Leadership Team • First - in - class biologics with potential to reverse “irreversible ” damage to the heart and other organs • Product candidates based on cells and exosomes • Dev
Slide 5: Capricor's Innovative Product Pipeline
5 Capricor’s Innovative Product Pipeline Product Candidate Indication/ Population Development Phase Status Preclinical I II III CAP - 1002 ( allogeneic CDCs) Duchenne Muscular Dystrophy ▪ To report to p - line six - month results from Phase I/II HOPE clinical trial in early 2Q 2017 Post - Myocardial
Slide 6: Estimated Global Patient Populations for CAP-1002
6 Estimated Global Patient Populations for CAP - 1002 Population US 15 K Europe 20 K WW incidence ~ 1 per 3,600 male births Population US 6 M Europe 15 M Japan 1 M Duchenne Muscular Dystrophy Heart Failure CAP - 1002 has FDA orphan drug designation for the treatment of DMD.
Slide 7: The Power of Capricor's Technology
7 The Power of Capricor’s Technology Scar formation results from heart muscle death, and the amount of scar correlates with outcomes. Neither time nor current treatment options will lead to the replacement of scar tissue with muscle. Capricor’s technology offers transformative treatment potential th
Slide 8: Clinical Validation Provided by the CADUCEUS Trial
8 Clinical Validation Provided by the CADUCEUS Trial – Randomized trial in patients who had experienced a large heart attack (N=25; two centers) – One - time intracoronary delivery of autologous CDCs vs. usual care controls First in - human evidence for therapeutic heart regeneration Lancet , 2012,
Slide 9: Scar Size Predicts Outcomes Following MI
9 Scar Size Predicts Outcomes Following MI CADUCEUS Trial Results Scar Size (as mean p ercent of LV) (SD) CDCs Control Baseline 23.8 % (9.9) 22.4% (7.9) 12 Months 12.9% (6.1) 20.3% (7.5) The size of the infarct or residual scar following MI is a predictor of outcomes, e.g.: – re - infarction – heart
Slide 10: Cell Therapy Programs for Adult Heart Disease
10 Cell Therapy Programs for Adult Heart Disease
Slide 11: ALLSTAR & DYNAMIC Trials with Off-the-Shelf CAP-1002
11 Phase I/II ALLSTAR trial in post - myocardial infarction Phase I DYNAMIC trial in advanced heart failure ALLSTAR & DYNAMIC Trials with “Off - the - Shelf” CAP - 1002 Clinical trials of CAP - 1002 in prevalent cardiovascular disorders: – Well - tolerated in completed studies; no immune - related s
Slide 12: Enrollment in Phase II ALLSTAR Trial has Completed
12 Enrollment in Phase II ALLSTAR Trial has Completed – Similar to CADUCEUS trial, but using CAP - 1002 (allogeneic) – Randomized, double - blind , placebo - controlled Phase II trial of CAP - 1002 134 patients who had experienced a large heart attack were enrolled Each received a one - time infusio
Slide 13: Positive Results with CAP-1002 in Advanced Heart Failure
13 0 20 40 60 80 100 Baseline 6 Months 12 Months % of Subjects (N=12) NYHA Class by Study Visit I II III IV Positive Results with CAP - 1002 in Advanced Heart Failure – DYNAMIC demonstrated an efficacy signal despite its small sample size ( N=14). – Concordant improvements from baseline in functiona
Slide 14: Janssen Holds a License Option on CAP-1002
14 – In 2014, Capricor granted Janssen Biotech an exclusive option to enter into an exclusive license agreement for worldwide rights to CAP - 1002 for certain CV indications – Option period to end 60 days following Capricor’s delivery of interim six - month ALLSTAR data to Janssen – Under a potentia
Slide 15: Manufacturing Process Development with Janssen
15 Manufacturing Process Development with Janssen We expect to be able to generate thousands of doses of CAP - 1002 from each donor heart via commercial process in development
Slide 16: Cell Therapy Program for Duchenne Muscular Dystrophy
16 Cell Therapy Program for Duchenne Muscular Dystrophy
Slide 17: Heart Failure is #1 Cause of Death in DMD
17 Heart Failure is #1 Cause of Death in DMD – DMD results from mutation in dystrophin gene – By early adulthood, nearly all people with DMD have clinical manifestations of cardiac disease – No approved therapies for the heart disease associated with DMD
Slide 18: Heart Failure is the End Result of Progressive Scarring in DMD
18 Menon et al, Pediatr Cardiol 2014 ; 35 :1279 - 85; Tandon et al, J Am Heart Assoc 2015 ; 4 :e001338 . Heart Failure is the End Result of Progressive Scarring in DMD scarring in heart muscle increases linearly with age and strongly correlates with LV ejection fraction m ay be clinically silent due
Slide 19: Concept of Using CAP-1002 to Treat DMD Heart Disease
19 Concept of Using CAP - 1002 to Treat DMD Heart Disease What happens in DMD CDCs’ Actions Oxidative/ nitrosative stress Anti - oxidative Inflammation Anti - inflammatory Apoptosis Anti - apoptotic Remodeling Anti - remodeling Loss of myocytes Regenerative Diverse effects of CDCs support their pote
Slide 20: CDCs Improve Function and Increase Survival in Mdx Mice
20 CDCs Improve Function and Increase Survival in Mdx Mice Improved cardiac function Increased exercise capacity Increased survival
Slide 21: Randomized Phase I/II HOPE-Duchenne Trial of CAP-1002
21 Randomized Phase I / II HOPE - Duchenne Trial of CAP - 1002 Being conducted in 25 boys and young men with DMD - associated cardiomyopathy Ambulatory and non - ambulatory patients are eligible Single infusion of CAP - 1002 or usual care only Exploratory efficacy assessments to be conducted at six
Slide 22: HOPE Features a Variety of Exploratory Efficacy Analyses
22 HOPE Features a Variety of Exploratory Efficacy Analyses Week 6 Month 3 Month 6 Month 12 Imaging Cardiac magnetic resonance ( cMRI ) Ɣ Ɣ Functional Tests Performance of the Upper Limb (PUL) Ɣ Ɣ Ɣ Ɣ 6 - Minute Walk Test Ɣ Ɣ Ɣ Ɣ Spirometry Ɣ Ɣ Ɣ Ɣ Quality of Life Pediatric QL Inventory Ɣ Ɣ Ɣ Ɣ PODC
Slide 23: Cardiac MRI to Assess Several Parameters
23 Cardiac MRI to Assess Several Parameters Left Ventricular Month 6 Month 12 Myocardial composition Scar ( late gadolinium enhancement) Ɣ Ɣ Viable mass Ɣ Ɣ Dynamics Ejection fraction Ɣ Ɣ Regional wall motion Ɣ Ɣ Circumferential strain Ɣ Ɣ Dimensions End - diastolic volume Ɣ Ɣ End - systolic volume
Slide 24: Initial HOPE Data to be Reported in Early 2Q 2017
24 Initial HOPE Data to be Reported in Early 2Q 2017 ► Expected to be reported: – Safety and tolerability – Exploratory efficacy measures (no powering for statistical significance) ► Independent Data Safety Monitoring Board (DSMB) has met four times during study per a predefined schedule – Trial was
Slide 25: Exosome Program
25 Exosome Program “Next - Generation” Class of Biological Therapies
Slide 26: Exosomes – a Cell-Free Regenerative Medicine Platform
26 Exosomes – a Cell - Free Regenerative Medicine Platform Gallet et al, Eur Heart J 2016 ; 38 :201 - 211.
Slide 27: Exosomes – a Cell-Free Regenerative Medicine Platform
27 Exosomes – a Cell - Free Regenerative Medicine Platform Gallet et al, Eur Heart J 2016 ; 38 :201 - 211. also active in non - cardiac inflammatory disease models
Slide 28: Cash Runway Through Key 2017 Events
28 Cash Runway Through Key 2017 Events Capricor has received over $30 million in competitive grant and loan awards Cash and cash equivalents $20.5 million (as of 9/30/16) Net cash used in operations $11.6 million (nine months ended 9/30/16) Shares outstanding 21.4 million (February 13, 2017 )
Slide 29: Cash Runway Through Key 2017 Events
29 Cash Runway Through Key 2017 Events Capricor has received over $30 million in competitive grant and loan awards Cash and cash equivalents $20.5 million (as of 9/30/16) Net cash used in operations $11.6 million (nine months ended 9/30/16) Shares outstanding 21.4 million (February 13, 2017 )
Slide 30: Recent and Upcoming Milestones
30 Recent and Upcoming Milestones Clinical Programs CAP - 1002 in Duchenne Heart Disease x 3Q 2016: Announced completion of enrollment in HOPE trial Early 2Q 2017: To report six - month top - line results of HOPE trial CAP - 1002 in Adult Heart Disease x 4Q 2016: Announced completion of ALLSTAR enro
Slide 31: www.capricor.com
www.capricor.com A Translational Medicine Company NASDAQ: CAPR
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