Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (October 2016)
24 slides from Capricor Therapeutics's investor presentation, Oct 5, 2016
Slide 1: Investor Presentation October 2016
NASDAQ: CAPR A Translational Medicine Company www.capricor.com Investor Presentation October 2016
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: Pipeline Summary
3 Pipeline Summary Product Candidate Indication Preclinical Phase 1/2 Phase 2 Phase 3 Status & Upcoming Milestones CAP - 1002 Duchenne Muscular Dystrophy ▪ Enrollment completed ▪ To report to p - line six - month HOPE results in Q1:17 Advanced Heart Failure ▪ To present 12 - month DYNAMIC results at
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 f
Slide 5: Infarct (Scar) Size Significantly Impacts Survival
5 Infarct (Scar) Size Significantly Impacts Survival Wu et al, Heart 2008 ; 94 :730 - 6. 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
Slide 6: Janssen has Option to License
6 – 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 mid - 2017 License fee and additional
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
Slide 8: Capricor – Portfolio at a Glance: Adult Cardiac Conditions
8 Capricor – Portfolio at a Glance CAP - 1002 CAP - 2003 Natriuretic Peptides Adult Cardiac Conditions P ROGRAM Phase II
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
Slide 10: ALLSTAR Phase I Efficacy (Phase II-Equivalent Population)
10 p < 0.05 * ALLSTAR Phase I Efficacy (Phase II - Equivalent Population) 15 % reduction in scar size at one year 4 percentage - point improvement in ejection fraction at one year Received high dose CAP - 1002 and lacked donor - specific antibodies ( DSAs)
Slide 11: DYNAMIC Clinical Trial 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 C onducted at Cedars - Sinai Medical Center – Enrollment was open to subjects with dilated cardiomyopathy NYHA Class III or ambulatory Class IV I schemic or
Slide 12: Six-Month DYNAMIC Results for Advanced HF
12 NYHA Class Physical Activity I No limitation II Slight limitation III Marked limitation IV Severe limitation * N=14 ; two subjects lost to follow - up. Six - Month DYNAMIC Results on CAP - 1002 For Advanced HF Presented at the American Heart Association Annual Meeting, November 2015* – Of the 12
Slide 13: Left Ventricular Dynamics & Dimensions
13 Left Ventricular Dynamics & Dimensions less is better * N=14; two subjects lost to follow - up; measurements assessed by echocardiography . Six - Month DYNAMIC Results on CAP - 1002 For Advanced HF Presented at the American Heart Association Annual Meeting, November 2015* – Efficacy signal contin
Slide 14: Capricor – Portfolio at a Glance: Duchenne Muscular Dystrophy
14 Capricor – Portfolio at a Glance CAP - 1002 CAP - 2003 Natriuretic Peptides Duchenne Muscular Dystrophy P ROGRAM Phase I / II
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 – N
Slide 16: Improves Cardiac Function and Exercise Capacity in DMD Mouse Model
16 30 40 50 60 70 80 Mdx + CAP-1002 Mdx + Vehicle *** p < 0.001 * p < 0.05 100 200 300 400 500 600 700 800 3 4 5 6 CTL Mdx + CAP - 1002 Mdx + vehicle Week *** 1 st injection EF (%) Repeat Dosing Meters 2 nd injection Mdx mice + CDC, n=12 Mdx mice + vehicle, n=12 CTL (wild - type), n=5 CAP - 1002 Imp
Slide 17: Enrollment in HOPE-Duchenne Trial Has Completed
17 – Successfully completed two interim DSMB safety reviews – Expect to report top - line six - month data in Q1 2017 Structural (cardiac MRI)*, functional, quality - of - life endpoints – Results may enable Capricor to discuss product registration with FDA Randomize Usual Care M12 W2 W6 M3 M6 30 d
Slide 18: Capricor – Portfolio at a Glance: Diseases of Inflammation and Fibrosis
18 Capricor – Portfolio at a Glance Diseases of Inflammation and Fibrosis CAP - 1002 CAP - 2003 Natriuretic Peptides P ROGRAM Preclinical
Slide 19: Exosomes: Cell Free Regenerative Medicine Platform
19 Exosomes: Cell Free Regenerative Medicine Platform Camussi et al, Kidney Int 2010 ; 78 :838 – 848. – 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
Slide 20: Ocular GVHD – First Clinical Development Opportunity
20 Ocular GVHD – First Clinical Development Opportunity – 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, photopho
Slide 21: CAP-2003 Demonstrated Significant Improvement in Alkali Burn Injury Model
21 CAP - 2003 Demonstrated Significant Improvement in Alkali Burn Injury Model 6 11 16 21 DAY 0 DAY 4 DAY 7 DAY 14 DAY 21 S c o r e s u m Clinical score Vehicle Topical Subcon. 0 5 10 15 DAY 0 DAY 4 DAY 7 DAY 14 DAY 21 C o r n e a l I n j u r y A r e a ( m m 2 ) Epithelial defect area (Fluorescein s
Slide 22: Cash Runway Through Key 2017 Milestones
22 Cash, cash equivalents, and marketable securities reported at June 30, 2016 $11.4 million Net cash used in operations in H1:16 $9.1 million Shares outstanding 21.4 million* Cash Runway Through Key 2017 Milestones – Raised approx. $10 million in net proceeds in September 2016 from sale of common s
Slide 23: Recent and Upcoming Milestones
23 Recent and Upcoming Milestones CAP - 1002 in Duchenne Heart Disease x 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 x Announced completion of ALLSTAR enrollment in October 2016 □ To pr
Slide 24: A Translational Medicine Company
NASDAQ: CAPR A Translational Medicine Company www.capricor.com 8840 Wilshire Boulevard – 2 nd floor Beverly Hills, CA 90211 (310) 358 - 3200
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