Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (June 2016)
32 slides from Capricor Therapeutics's investor presentation, June 2016
Slide 1: Investor Presentation
A Translational Medicine Company NASDAQ: CAPR Investor Presentation www.capricor.com June 2016
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: Capricor – Portfolio at a Glance
Capricor – Portfolio at a Glance 3 PROGRAM Duchenne Phase I / II TBD Muscular Dystrophy Cardiosphere- Derived Cells Adult Janssen Cardiac Conditions Phase II Option Diseases of Inflammation Internal CDC Exosomes Preclinical and Fibrosis & External Natriuretic Outpatient Management Potential Peptides
Slide 4: Cardiosphere-Derived Cells (CDCs)
4 Cardiosphere-Derived Cells (CDCs) – Derived from donor human hearts (allogeneic) – Manufactured by Capricor via proprietary process – Record of immunological safety per cumulative clinical experience – Packaged in Cryostor™, three-year frozen shelf life – IP licensed from Johns Hopkins, U. of Rome
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 – Secr
Slide 6: CDCs: Heart Cells for Heart Disease
6 CDCs: Heart Cells for Heart Disease – 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 h
Slide 7: Clinical Proof-of-Concept was Provided by CADUCEUS
7 Clinical Proof-of-Concept was Provided by CADUCEUS Lancet, 2012, 21(6): 1121-1135. – 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 M
Slide 8: Capricor – Detail by Program
Capricor – Detail by Program 8 PROGRAM Duchenne Phase I / II TBD Muscular Dystrophy Cardiosphere- Derived Cells CDC Exosomes Natriuretic Peptides
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 dilate
Slide 10: Heart Failure is the End Result of Progressive Scarring
10 Heart Failure is the End Result of Progressive Scarring – In DMD, myocardial fibrosis (scar): – Independently predicts adverse cardiac remodeling, ventricular arrhythmia, death – Increases linearly with age and strongly correlates with LV ejection fraction Menon et al, Pediatr Cardiol 2014;35:127
Slide 11: CAP-1002 Improves Cardiac Function and Exercise Capacity in DMD Mouse Model
11 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 12: CAP-1002 in Clinical Development for DMD-Associated Cardiomyopathy
12 CAP-1002 in Clinical Development for DMD-Associated Cardiomyopathy – 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 treatme
Slide 13: Randomized Phase I / II HOPE-Duchenne Trial Ongoing
13 Randomized Phase I / II HOPE-Duchenne Trial Ongoing CAP-1002 Infusion 30 d Screen Randomize W2 W6 M3 M6 M12 Usual Care – Boys with cardiomyopathy secondary to DMD Passed interim DSMB safety review – N=24, parallel-group study (3-4 sites) 1:1 randomization to one-time CAP-1002 (75 million cells)
Slide 14: Capricor – Detail by Program
Capricor – Detail by Program 14 PROGRAM Cardiosphere- Derived Cells Adult Janssen Cardiac Conditions Phase II Option CDC Exosomes Natriuretic Peptides
Slide 15: CAP-1002's Opportunity in Advanced Heart Failure
15 CAP-1002’s Opportunity in Advanced Heart Failure – >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
Slide 16: DYNAMIC Results Support CAP-1002 For Advanced HF
16 DYNAMIC Results Support CAP-1002 For Advanced HF Six-Month Data 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 m
Slide 17: DYNAMIC Results Support CAP-1002 For Advanced HF
17 DYNAMIC Results Support CAP-1002 For Advanced HF Six-Month Data Presented at the American Heart Association Annual Meeting, November 2015* less is better Quality of Life 6 Minute Walk Test & VO2 Max * N=14; two subjects lost to follow-up; AHA Annual Meeting, November 2015.
Slide 18: DYNAMIC Results Support CAP-1002 For Advanced HF
18 DYNAMIC Results Support CAP-1002 For Advanced HF Six-Month Data Presented at the American Heart Association Annual Meeting, November 2015* Left Ventricular Dynamics & Dimensions less is better – Efficacy signal continued to be observed at 12 months LVEF +17.5% (median) from baseline (p=0.02) * N=
Slide 19: Agreement with Janssen Biotech, Inc. on CAP-1002
19 Agreement with Janssen Biotech, Inc. on CAP-1002 – 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
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 CDCs or
Slide 21: ALLSTAR Phase I Results: Showed Improvement in Scar Size
21 ALLSTAR Phase I Results: Showed Improvement in Scar Size p < 0.05 * p < 0.05 # Infarct Size Infarct Size n=8 n=8 n=7 * 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-
Slide 22: Capricor – Detail by Program
Capricor – Detail by Program 22 PROGRAM Cardiosphere- Derived Cells Diseases of Inflammation Internal CDC Exosomes Preclinical and Fibrosis & External Natriuretic Peptides
Slide 23: Capricor's CDC Exosomes – CAP-2003
Capricor’s CDC Exosomes – CAP-2003 23 – 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 cells, CDC
Slide 24: CDC Exosomes Significantly Improve Cardiac Structure and Function in Preclinical Studies
24 CDC Exosomes Significantly Improve Cardiac Structure and Function in Preclinical Studies 50 Control CDC-XO Effects not observed with 45 NHDF-XO ** exosomes from other cell types MSC-XO * EF (%) 40 35 30 25 1 15 30 **p < 0.01 Control NHDF-Exosomes Days post MI *p < 0.05 20 ** ** Scar Mass (mg) 15
Slide 25: CDC Exosomes Provide Rapid and Dose-Dependent Improvement in Ocular Inflammation Model
25 CDC Exosomes Provide Rapid and Dose-Dependent Improvement in Ocular Inflammation Model – 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
Slide 26: CAP-2003 Moving into the Clinic
26 CAP-2003 Moving into the Clinic – 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
Slide 27: Capricor – Detail by Program
Capricor – Detail by Program 27 PROGRAM Cardiosphere- Derived Cells CDC Exosomes Natriuretic Outpatient Management Potential Peptides Phase II of Heart Failure Outlicense
Slide 28: Cenderitide (CD-NP) – For Potential Outlicense
Cenderitide (CD-NP) – For Potential Outlicense 28 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-concept for chr
Slide 29: Cash Runway Through Early 2017 Data Readouts
29 Cash Runway Through Early 2017 Data Readouts Cash, cash equivalents, and marketable securities $14.3 million reported at March 31, 2016 Net cash used in operations in 2015 $10.8 million Shares outstanding (May 12, 2016) 18.0 million
Slide 30: Upcoming Milestones
30 Upcoming Milestones 2016 2017 Anticipated Event Mid Announce first indication for CAP-2003 Complete enrollment in HOPE-Duchenne trial of CAP-1002 Q3 Complete enrollment in ALLSTAR trial of CAP-1002 H2 Report Phase II data (higher dose range) on Cenderitide Report six-month data from HOPE-Duchenne
Slide 31: Senior Management
31 Senior Management Linda Marbán, PhD AJ Bergmann, MBA Chief Executive Officer VP of Finance Leland Gershell, MD PhD Rachel Smith, PhD Chief Financial Officer VP of Research & Development Deborah Ascheim, MD Houman Hemmati, MD PhD Chief Medical Officer VP of New Therapy Development Karen Krasney, J
Slide 32: 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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