Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (October 2016)
24 slides from Capricor Therapeutics's investor presentation, October 2016
Slide 1: Investor Presentation
A Translational Medicine Company Investor Presentation NASDAQ: CAPR October 2016 www.capricor.com
Slide 2: Forward-Looking Statements
2 Forward-Looking Statements 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 state
Slide 3: Pipeline Summary
3 Pipeline Summary Product Indication Preclinical Phase 1/2 Phase 2 Phase 3 Status & Upcoming Milestones Candidate Duchenne Enrollment completed Muscular To report top-line six-month HOPE results Dystrophy in Q1:17 Advanced To present 12-month DYNAMIC results at CAP-1002 Heart Failure TCT annu
Slide 4: Clinical Validation Provided by the CADUCEUS Trial
4 Clinical Validation Provided by the CADUCEUS Trial Lancet, 2012, 21(6): 1121-1135. Showed for the first time that heart regeneration is therapeutically possible Provides hope for a curative approach vs. stabilization, palliation – Randomized trial in 25 patients with reduced ejection fraction foll
Slide 5: Infarct (Scar) Size Significantly Impacts Survival
5 Infarct (Scar) Size Significantly Impacts Survival Kaplan-Meier curve for Major Adverse Cardiac Events (MACE) following acute ST-segment elevation MI, as stratified by infarct size. MACE = death myocardial infarction heart failure admission Wu et al, Heart 2008;94:730-6.
Slide 6: Janssen has Option to License CAP-1002
6 Janssen has Option to License CAP-1002 – CADUCEUS data attracted the interest of big pharma – Capricor granted Janssen Biotech an exclusive option to enter into exclusive license agreement for worldwide rights to CAP-1002 for certain CV indications – Expect Janssen’s decision on license option by
Slide 7: Heart Cells for Heart Disease
7 Heart Cells for Heart Disease Capricor’s core technology is the cardiosphere-derived cell (CDC): – Does not act by ‘stemness’ – does not engraft into myocardium – Exerts favorable and durable effects on target cells via exosome signalling – cardiomyogenic, anti-apoptotic, angiogenic, anti-fibrotic
Slide 8: Capricor – Portfolio at a Glance
Capricor – Portfolio at a Glance 8 PROGRAM Adult Phase II Cardiac Conditions CAP-1002 CAP-2003 Natriuretic Peptides
Slide 9: Enrollment in Phase II ALLSTAR Trial has Completed
9 Enrollment in Phase II ALLSTAR Trial has Completed – Similar patient population as CADUCEUS, but using allogeneic cells – Phase II: randomized, double-blind, placebo-controlled Patients enrolled at 30 centers in U.S. & Canada 134 subjects with recent (30 – 90 d) or chronic (91 – 365 d) STEMI or NS
Slide 10: ALLSTAR Phase I Efficacy (Phase II-Equivalent Population)
10 ALLSTAR Phase I Efficacy (Phase II-Equivalent Population) Received high dose CAP-1002 and lacked donor-specific antibodies (DSAs) p < 0.05 * 15% reduction in scar size at one year 4 percentage-point improvement in ejection fraction at one year
Slide 11: DYNAMIC Clinical Trial of CAP-1002 in Advanced HF
11 DYNAMIC Clinical Trial of CAP-1002 in Advanced HF – Phase IIa, open-label, dose-escalation clinical trial in heart failure patients Conducted at Cedars-Sinai Medical Center – Enrollment was open to subjects with dilated cardiomyopathy NYHA Class III or ambulatory Class IV Ischemic or non-ischemic
Slide 12: Six-Month DYNAMIC Results on CAP-1002 For Advanced HF
12 Six-Month DYNAMIC Results on CAP-1002 For Advanced HF Presented at the American Heart Association Annual Meeting, November 2015* NYHA Class Physical Activity I No limitation II Slight limitation III Marked limitation IV Severe limitation – Of the 12 evaluable Class III patients at six months, 11
Slide 13: Six-Month DYNAMIC Results on CAP-1002 For Advanced HF
13 Six-Month DYNAMIC Results on CAP-1002 For Advanced HF Presented at the American Heart Association Annual Meeting, November 2015* Left Ventricular Dynamics & Dimensions less is better * N=14; two subjects lost to follow-up; measurements assessed by echocardiography. – Efficacy signal continued to
Slide 14: Capricor – Portfolio at a Glance
Capricor – Portfolio at a Glance 14 PROGRAM CAP-1002 Duchenne Muscular Dystrophy Phase I / II CAP-2003 Natriuretic Peptides
Slide 15: Cardiomyopathy is #1 Cause of Death in DMD
15 Cardiomyopathy is #1 Cause of Death in DMD – DMD results from mutation in dystrophin gene – 1 per 3,600 male births – Lack of functional dystrophin in heart leads to: inflammation cardiomyocyte death progressive cardiac fibrosis – Hearts become dilated and non-compliant, and eventually fail – No
Slide 16: CAP-1002 Improves Cardiac Function and Exercise Capacity in DMD Mouse Model
16 CAP-1002 Improves Cardiac Function and Exercise Capacity in DMD Mouse Model Repeat Dosing 80 1st injection 2nd injection * 800 70 * *** 700 CTL * 60 * 600 EF (%) Meters 500 Mdx + CAP-1002 50 400 300 40 Mdx + CAP-1002 Mdx + vehicle 200 Mdx + Vehicle 30 100 3 4 5 6 Week *** p < 0.001 *p < 0.05 Mdx
Slide 17: Enrollment in HOPE-Duchenne Trial Has Completed
17 Enrollment in HOPE-Duchenne Trial Has Completed – Randomized Phase I/II trial at three U.S. centers 25 boys with DMD-associated cardiomyopathy 13 in active arm – CAP-1002 triple-coronary infusion (single procedure) 12 in comparator arm – usual care only CAP-1002 Infusion 30 d Screen Randomize W2
Slide 18: Capricor – Portfolio at a Glance
Capricor – Portfolio at a Glance 18 PROGRAM CAP-1002 Diseases of Inflammation CAP-2003 Preclinical and Fibrosis Natriuretic Peptides
Slide 19: Exosomes: Cell Free Regenerative Medicine Platform
19 Exosomes: Cell Free Regenerative Medicine Platform – CDC Exosomes are virus-sized vesicles that can reduce inflammation and regenerate damaged tissue – CAP-2003 (CDC Exosomes) may be used in applications for which CDCs may not be well-suited – Exclusive WW license from Cedars-Sinai Medical Center
Slide 20: Ocular GVHD – First Clinical Development Opportunity
Ocular GVHD – First Clinical Development Opportunity 20 – Graft-vs-host disease (GVHD) A common complication of bone marrow transplantation (BMT) Chronic rejection of new cells – Ocular GVHD is a common, debilitating form of chronic GVHD and is an unmet medical need Burning, irritation, photophobia,
Slide 21: CAP-2003 Demonstrated Significant Improvement in Alkali Burn Injury Model
21 CAP-2003 Demonstrated Significant Improvement in Alkali Burn Injury Model Clinical score Epithelial defect area (Fluorescein staining) 21 15 Vehicle Topical Subcon. Corneal Injury Area (mm2) 16 10 Score sum 11 5 Vehicle Topical Subcon 6 0 DAY 0 DAY 4 DAY 7 DAY 14 DAY 21 DAY 0 DAY 4 DAY 7 DAY 14 D
Slide 22: Cash Runway Through Key 2017 Milestones
22 Cash Runway Through Key 2017 Milestones Cash, cash equivalents, and marketable securities $11.4 million reported at June 30, 2016 Net cash used in operations in H1:16 $9.1 million Shares outstanding 21.4 million* * Includes approx. 3.4 million shares sold in September 2016 offering – Raised appro
Slide 23: Recent and Upcoming Milestones
23 Recent and Upcoming Milestones CAP-1002 in Duchenne Heart Disease Announced completion of HOPE enrollment in September 2016 Expect to report six-month top-line results of HOPE in Q1:17 CAP-1002 in Adult Heart Disease Announced completion of ALLSTAR enrollment in October 2016 To present 12
Slide 24: A Translational Medicine Company
A Translational Medicine Company 8840 Wilshire Boulevard – 2nd floor NASDAQ: CAPR Beverly Hills, CA 90211 www.capricor.com (310) 358-3200
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