Slide 1: A Translational Medicine Company
NASDAQ: CAPR A Translational Medicine Company www.capricor.com Investor Presentation June 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: Capricor – Portfolio at a Glance
3 Capricor – Portfolio at a Glance Diseases of Inflammation and Fibrosis Outpatient Management of Heart Failure Cardiosphere - Derived Cells CDC Exosomes Natriuretic Peptides Adult Cardiac Conditions Duchenne Muscular Dystrophy P ROGRAM Internal & External Janssen Option TBD Phase I / II Phase II Pr
Slide 4: Cardiosphere-Derived Cells (CDCs)
4 – Derived from donor human hearts (allogeneic) – Manufactured by Capricor via proprietary process – Record of immunological safety per cumulative c linical experience – Packaged in Cryostor ™, three - year frozen shelf life – IP licensed from Johns Hopkins, U. of Rome, Cedars - Sinai Medical Cntr
Slide 5: CDCs Exert Their Effects Via Paracrine Mechanisms
5 CDCs Exert Their Effects Via Paracrine Mechanisms – Do not act by ‘ stemness ’ – do not engraft – Act as local drug delivery vehicles – Are epigenetic modulators of gene expression and cell function – Release a wide variety of regulatory bio - molecules to effect their actions – RNAs – Proteins –
Slide 6: CDCs: Heart Cells for Heart Disease
6 – A large body of reproducible data shows that CDCs: Induce cardiac myogenesis Prevent cardiomyocyte apoptosis (programmed cell death) Promote new blood vessel formation Exert anti - scarring activity Attract endogenous progenitor cells – Clinical trials of CDCs have shown durable cardiac improvem
Slide 7: Clinical Proof-of-Concept was Provided by CADUCEUS
7 Clinical Proof - of - Concept was Provided by CADUCEUS – Patients with reduced ejection fraction (EF ) following myocardial infarction (MI ) – N=25 (17 active, 8 control ) – One - time intracoronary delivery of autologous CDCs (25 million cells) – Sponsored by Cedars - Sinai Medical Center, with J
Slide 8: Capricor – Detail by Program
8 Capricor – Detail by Program Cardiosphere - Derived Cells CDC Exosomes Natriuretic Peptides Duchenne Muscular Dystrophy P ROGRAM TBD Phase I / II
Slide 9: DMD Cardiomyopathy is #1 Cause of Death in DMD
9 DMD Cardiomyopathy is #1 Cause of Death in DMD – DMD results from mutation of calcium - regulating dystrophin gene – 1 per 3,500 male births – Induces profound, progressive dysfunction of all muscle types – Cardiac involvement is universal and #1 cause of death – DMD cardiomyopathy results in dila
Slide 10: Heart Failure is the End Result of Progressive Scarring
10 Menon et al, Pediatr Cardiol 2014 ; 35 :1279 - 85. Heart Failure is the End Result of Progressive Scarring Tandon et al, J Am Heart Assoc 2015 ; 4 :e001338. – In DMD, myocardial fibrosis ( scar): – Independently predicts adverse cardiac remodeling, ventricular arrhythmia, death – Increases linear
Slide 11: CAP-1002 Improves Cardiac Function and Exercise Capacity in DMD Mouse Model
11 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 12: CAP-1002 in Clinical Development for DMD-Associated Cardiomyopathy
12 – Ongoing HOPE - Duchenne study designed to support registration – CAP - 1002 is distinct from the exon skipping therapies Apparent lack of mutation dependence supports use in any genotype – FDA Orphan Drug Designation for the treatment of DMD CAP - 1002 in Clinical Development for DMD - Associat
Slide 13: HOPE-Duchenne Trial Ongoing
13 – Boys with cardiomyopathy secondary to DMD – N=24, parallel - group study (3 - 4 sites) 1:1 randomization to one - time CAP - 1002 (75 million cells) or ‘usual care’ Randomize Usual Care M12 W2 W6 M3 M6 30 d Randomized Phase I / II HOPE - Duchenne Trial Ongoing Screen * FDA Draft Guidance, June
Slide 14: Capricor – Detail by Program
14 Capricor – Detail by Program Cardiosphere - Derived Cells CDC Exosomes Natriuretic Peptides Adult Cardiac Conditions P ROGRAM Janssen Option Phase II
Slide 15: CAP-1002's Opportunity in Advanced Heart Failure
15 – > 5 million U.S. patients estimated to have heart failure Current treatments largely fail to address the progressive underlying disease – In a preclinical model of non - ischemic dilated cardiomyopathy, CDCs have been shown to: reverse abnormalities in cell signaling prevent adverse remodelling
Slide 16: DYNAMIC Results Support CAP-1002 For Advanced HF
16 NYHA Class Physical Activity I No limitation II Slight limitation III Marked limitation IV Severe limitation * N=14 ; two subjects lost to follow - up. DYNAMIC Results Support CAP - 1002 For Advanced HF Six - Month Data Presented at the American Heart Association Annual Meeting, November 2015* –
Slide 17: DYNAMIC Results Support CAP-1002 For Advanced HF
17 6 Minute Walk Test & VO 2 Max Quality of Life less is better * N=14 ; two subjects lost to follow - up; AHA Annual Meeting, November 2015. DYNAMIC Results Support CAP - 1002 For Advanced HF Six - Month Data Presented at the American Heart Association Annual Meeting, November 2015*
Slide 18: DYNAMIC Results Support CAP-1002 For Advanced HF
18 Left Ventricular Dynamics & Dimensions less is better * N=14; two subjects lost to follow - up; measurements assessed by echocardiography; AHA Annual Meeting, November 2015. DYNAMIC Results Support CAP - 1002 For Advanced HF Six - Month Data Presented at the American Heart Association Annual Meet
Slide 19: Agreement with Janssen Biotech, Inc. on CAP-1002
19 – Janssen has exclusive option to enter into exclusive license agreement for worldwide rights to CAP - 1002 for certain cardiovascular indications – If exercised, Janssen to fund all future development costs and Capricor eligible to receive: License fee and additional milestone payments totaling
Slide 20: ALLSTAR Trial Nearing Enrollment Completion
20 ALLSTAR Trial Nearing Enrollment Completion – Randomized, double - blind, placebo - controlled; 30 - 35 U.S. centers – 120 subjects with recent (30 – 90 d) or chronic (91 – 365 d) STEMI or NSTEMI – Left ventricular scar size ≥15% of LV mass – Left ventricular ejection fraction ≤45% – 25 million C
Slide 21: ALLSTAR Phase I Results: Showed Improvement in Scar Size
21 ALLSTAR Phase I Results: Showed Improvement in Scar Size * by groups t - test # by paired t - test – Results shown are from the “Phase II Equivalent” population Received high dose CAP - 1002 Lacked donor - specific antibodies (DSAs) DSA = donor - specific antibody p < 0.05 # p < 0.05 * n=8 n=8 n=
Slide 22: Capricor – Detail by Program
22 Capricor – Detail by Program Diseases of Inflammation and Fibrosis Cardiosphere - Derived Cells CDC Exosomes Natriuretic Peptides P ROGRAM Internal & External Preclinical
Slide 23: Capricor's CDC Exosomes – CAP-2003
23 Capricor’s CDC Exosomes – CAP - 2003 – Exosomes represent a potential next - generation, cell - free therapeutic platform – Exosomes mediate the regenerative and cardioprotective effects of CDCs Were discovered in the course of elucidating CDCs’ mechanism of action – Like exosomes from other cell
Slide 24: CDC Exosomes Significantly Improve Cardiac Structure and Function in Preclinical Studies
24 Ibrahim et al, Stem Cell Reports 2014 ; 2 :606 - 19 . Effects not observed with exosomes from other cell types CDC Exosomes Significantly Improve Cardiac Structure and Function in Preclinical Studies 0 5 10 15 20 CTRL NHDF-XO MSC-XO CDC-XO Scar Mass (mg ) Day 30 * ** ** 25 30 35 40 45 50 1 15 30
Slide 25: CDC Exosomes Provide Rapid and Dose-Dependent Improvement in Ocular Inflammation Model
25 Control CAP - 2003 (low - dose) CAP - 2003 (high - dose) – Keratoconjunctivitis induced in rabbits (wound followed by LPS application) – After development of severe inflammation, eyes were treated with a single administration of CAP - 2003 or control, then followed for three days – Day three data
Slide 26: CAP-2003 Moving into the Clinic
26 – CDC exosomes have regenerative and cell - modulating capabilities – Broad treatment potential in inflammatory and fibrotic conditions Supported by growing body of preclinical data – Capricor plans to announce the first indication for CAP - 2003 in mid - 2016 CAP - 2003 Moving into the Clinic
Slide 27: Capricor – Detail by Program
27 Capricor – Detail by Program Outpatient Management of Heart Failure Cardiosphere - Derived Cells CDC Exosomes Natriuretic Peptides P ROGRAM Phase II Potential Outlicense
Slide 28: Cenderitide (CD-NP) – For Potential Outlicense
28 Cenderitide (CD - NP) – For Potential Outlicense Dual Natriuretic Peptide Receptor Activator for Cardio - Renal Disease States – Provides a first - in - class product licensing opportunity Positioned to address a large heart failure market segment lacking in therapeutic options – Proof - of - con
Slide 29: Cash Runway Through Early 2017 Data Readouts
29 Cash, cash equivalents, and marketable securities reported at March 31, 2016 $14.3 million Net cash used in operations in 2015 $10.8 million Shares outstanding (May 12, 2016) 18.0 million Cash Runway Through Early 2017 Data Readouts
Slide 30: Upcoming Milestones
30 * Janssen has the right to exercise its option to CAP - 1002 at any time until 60 days after the delivery by Capricor of the six - month follow - up results from the ALLSTAR clinical trial. Upcoming Milestones 2016 2017 Anticipated Event Mid Announce first indication for CAP - 2003 Q3 Complete en
Slide 31: Senior Management
31 AJ Bergmann, MBA VP of Finance Leland Gershell, MD PhD Chief Financial Officer Deborah Ascheim, MD Chief Medical Officer Karen Krasney, JD EVP & General Counsel Rachel Smith, PhD VP of Research & Development Houman Hemmati, MD PhD VP of New Therapy Development Luis Rodriguez - Borlado, PhD VP of
Slide 32: 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