Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (November 2014)
38 slides from Capricor Therapeutics's investor presentation, Nov 6, 2014
Slide 1: Transformative Therapies from Bench to Bedside
Transformative Therapies from Bench to Bedside Corporate Presentation November 2014 Non - confidential Clinical - stage biotechnology company developing novel therapeutics for the treatment of cardiovascular diseases Ticker: CAPR
Slide 2: Forward Looking Statements
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 Overview
Capricor Overview Non - confidential
Slide 4: Capricor Investment Opportunity
Capricor Investment Opportunity ▪ Ongoing Phase II clinical trial with Cardiosphere - derived cells (CDCs) ▪ Phase II funded by $19.8M loan award from CIRM ▪ Janssen Biotech (J&J) collaboration ▪ Operational leverage permits cost effective product development and evaluation ▪ Approximately 12 - 15 m
Slide 5: Senior Management & Board of Directors
Senior Management & Board of Directors Senior Management ▪ Chief Executive Officer Linda Marbán, Ph.D. ▪ EVP & General Counsel Karen Krasney, J.D. ▪ VP of Research & Development Rachel Smith, Ph.D. ▪ VP of Finance AJ Bergmann, M.B.A . ▪ VP of Medical Affairs Andrew Hamer, M.D. ▪ VP of Clinical Opera
Slide 6: Capricor: Key Metrics
Capricor: Key Metrics Select Data (approximate) As of 6.30.14 Cash , cash equivalents and marketable securities $11.9M Publicly traded: OTCBB Ticker: CAPR 52 week range $2.15 - $17.15 Shares outstanding 11.7M Fully diluted shares outstanding 16.9M Cash through Q3 2015 – Q4 2015 Non - dilutive capita
Slide 7: Capricor's Product Pipeline
CARDIOSPHERE - DERIVED CELLS (CAP - 1002) Strategic Collaboration with Janssen Biotech (J&J) Currently enrolling Phase II – ALLSTAR trial Planned Phase I 1H 2015 Ischemic Heart Disease Heart Failure Duchenne Muscular Dystrophy Targeting IND 2015 - 2016 CELL THERAPY NATRIURETIC PEPTIDE MICRO - RNA Cl
Slide 8: Janssen Deal Highlights
Janssen Deal Highlights In December 2013, Capricor entered into Collaboration Agreement and Exclusive License Option with Janssen Biotech (J&J) ▪ Capricor received an upfront payment of $12.5M ▪ Capricor and Janssen collaborating on elements of cell manufacturing ▪ Janssen has option to enter into e
Slide 9: Significant U.S. Target Market
Significant U.S. Target Market ▪ 1.3M Americans will have a new or recurrent myocardial infarction (MI) or heart attack ▪ 15% will die (1 death every 39 seconds) ▪ 36% will develop heart failure (HF) ▪ Survival correlated with infarct size ▪ 8.5M people in the US have had a heart attack ▪ 6.6M Ameri
Slide 10: Cell Therapy Technology
Cell Therapy Technology Non - confidential
Slide 11: Discovery of Cardiosphere Derived Cells (CDCs)
Discovery of Cardiosphere Derived Cells ( CDC s) ▪ Discovered and patented at Johns Hopkins University in 2004 by Eduardo Marbán, M.D., Ph.D. ▪ CDCs are isolated from cardiac tissue and possess unique characteristics and potentially regenerative capabilities ▪ CDCs act to stimulate cardiomyocyte pro
Slide 12: Lead Product: CDCs (CAP-1002)
Lead Product: CDCs (CAP - 1002) Cardiosphere - derived cells (CDCs) Cardiospheres (CSps) Explant - derived cells (EDCs) Explants Cardiac Tissue Features Cardiosphere - Derived Cells (CDCs) Cell source Human Cardiac Tissue Cell Type Cardiac Derived Stem Cell Dose Size 25M cells Target Patient Patient
Slide 13: Development of Ischemic Heart Failure
Development of Ischemic Heart Failure Non - confidential Heart attack (hours~days) Infarct expansion (days~weeks) Thinning (weeks~months) Chronic LV dilation
Slide 14: CADUCEUS, Lancet: 2012
CADUCEUS, Lancet: 2012 Non - confidential
Slide 15: CADUCEUS - Positive First-in-Man Data
CADUCEUS - Positive First - in - Man Data ▪ Autologous CDCs - 25M cells ▪ Patients with reduced Ejection Fraction % following MI ▪ Cedars - Sinai and Johns Hopkins ▪ Intracoronary delivery ▪ 25 patients ▪ 17 CDCs ▪ 8 C ontrols Non - confidential
Slide 16: CDC Therapy Reduced Scar Size and Increased Healthy Heart Muscle
CDC Therapy Reduced Scar Size and Increased Healthy Heart Muscle in the CADUCEUS study p<0.001 p:0.008 p:0.002 p<0.001 Δ scar mass Δ viable mass 6 mos 12 mos 6 mos 12 mos Makkar et al, Lancet, 2012. CDC Patients had a Significant Reduction in Infarct Size. We hypothesize improvement in clinical outc
Slide 17: Effect of Infarct Size on Survival
Effect of Infarct Size on Survival ▪ Both groups (CDC and CTRL) started with Infarct Size of 24% ▪ Patients treated with CDCs saw a reduction of Infarct Size to 12.5% ▪ This moved patients from a high risk group to a low risk group ▪ CTRL patients saw no change in Infarct Size, which puts them at gr
Slide 18: Advantages of Allogeneic CDCs
Advantages of Allogeneic CDCs ▪ Donors pre - screened by organ procurement organizations ▪ Single donor permits manufacturing of thousands of doses ▪ Freezing permits off - the shelf product availability ▪ COGS reduced by 10X compared to autologous cells Patient Dose of CAP - 1002 Non - confidential
Slide 19: CDCs in Active Clinical Development
CDCs in Active Clinical Development Post Myocardial Infarction (30 days – 1 year after MI) Drug Delivery FDA Status Target Indication ALLSTAR Clinical Trial NYHA Class III or ambulatory Class IV heart failure DYNAMIC Clinical Trial Single vessel i ntracoronary Multi - vessel intracoronary Estimated
Slide 20: ALLSTAR: Capricor's Phase I/II Trial using CDCs
ALLSTAR: Capricor’s Phase I/II Trial using CDCs ▪ Ejection Fraction ≤ 45% and Infarct Size ≥ 15 % (MRI ) ▪ Primary Endpoint – Infarct Size by MRI at 1 year ▪ Secondary Endpoints – multiple; EF, volumes, quality of life, etc. ▪ Phase I: 14 patients – complete ▪ Phase II: Est. 300 patients – currently
Slide 21: ALLSTAR: Phase I Primary Endpoint Met
ALLSTAR: Phase I Primary Endpoint Met ▪ 1 month post - infusion ▪ No Acute Myocarditis attributable to CAP - 1002 ▪ No clinically relevant immune events ▪ No Death due to VT/VF ▪ No Sudden Death ▪ No Major Adverse Cardiac Events (MACE) ▪ NIH DSMB approved advancement to Phase II Non - confidential
Slide 22: ALLSTAR Phase I MRI Analysis
ALLSTAR Phase I – 12 month MRI Analysis ▪ ALLSTAR Phase I ▪ 14 treated patients - open label dose escalation ▪ Met safety endpoint (1 month) ▪ No control group ▪ Preliminary 12 month MRI analysis on Phase II equivalent population (defined by tissue type compatibility) ▪ Ejection fraction improved by
Slide 23: New Indication: Duchenne Muscular Dystrophy
New Indication: Duchenne Muscular Dystrophy Non - confidential
Slide 24: Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy ▪ Duchenne Muscular Dystrophy (DMD) – rare genetic disorder caused by a mutation of the dystrophin gene ▪ Affects 1 in 3,500 male births worldwide ▪ Approximately 20,000 male children affected in the US ( 275,000 worldwide) ▪ Symptoms often appear in males before age six
Slide 25: CAP-1002 as a Potential Treatment for Cardiomyopathy Associated with DMD
CAP - 1002 as a Potential Treatment for Cardiomyopathy A ssociated with DMD ▪ CAP - 1002 has been shown to be safe and has been shown to reduce scar size in damaged hearts. ▪ Pre - clinical work on - going ▪ Targeting IND mid - 2015 ▪ Phase I planned for 2015 ▪ Presentation: AHA Nov. 2014 Non - conf
Slide 26: Capricor's CDCs Are Unique in Cardiac Cell Therapy
Capricor’s CDCs Are Unique in Cardiac Cell Therapy Capricor’s CDCs are the only allogeneic, intracoronary delivered, cardiac - derived stem cells CDCs Non - confidential
Slide 27: Natriuretic Peptide Technology
Natriuretic Peptide Technology Non - confidential
Slide 28: Cenderitide's Treatment for Heart Failure
Cenderitide’s treatment for Heart Failure Non - confidential Heart attack (hours~days) Infarct expansion (days~weeks) Thinning (weeks~months) Chronic LV dilation
Slide 29: The Post-Acute Hospitalization Period
The Post - Acute Hospitalization Period (90 days): When the Rate of Re - hospitalization and Death are Highest 0% 20% 40% 60% - 60 120 180 240 300 360 Percent of Patients Days After Hospital Admission for Heart Failure % of Patients with Death or Re - hospitalization after admission for Acute HF Ref
Slide 30: Post-Acute Heart Failure: New Indication
Post - Acute Heart Failure: New Indication Acute Post - Acute Chronic Duration 3 - 7 days 1 st 90 days after discharge 1 - 5 years Location In - Hospital Home Home Mortality* 2 - 4% 8 - 12% ~20% (1yr) / ~50% 5 yrs Treatment Goals • Relieve dyspnea • Off - load excess fluid • Survive • Stay out of ho
Slide 31: Cenderitide: Dual GC-A and GC-B Agonist
Cenderitide: Dual GC - A and GC - B Agonist - S - S - K L L D R I G S M S G L G G F C C K G S L G NH 2 P S L R D P R P N A P S T S A CD - NP ▪ Dual receptor activation means anti - fibrotic as well as natriuretic effects with less side effects ▪ Developed by scientists at the Mayo Clinic and derived
Slide 32: Continuous Subcutaneous Infusion Using the Insulet Omnipod Technology
Continuous Subcutaneous Infusion Using the Insulet Omnipod ® Technology ▪ Current pump delivery systems are robust, simple, and well tolerated ▪ In use by more than 300,000 patients worldwide ▪ Pump use is simple for all types of patients, not just diabetics ▪ Continuous delivery is ideal for worry
Slide 33: Cenderitide as a Potential Treatment for Post-Acute Heart Failure
Cenderitide as a Potential T reatment for Post - Acute Heart Failure Target Indication Treatment Duration 90 days of out - patient treatment after hospital discharge for acute decompensated heart failure Drug Delivery FDA Status Subcutaneous infusion using the Insulet Omnipod ® validated technology
Slide 34: Micro-RNA Technology
Micro - RNA Technology Non - confidential
Slide 35: Exosomes: Micro-RNA Platform Technology
Exosomes: Micro - RNA P latform T echnology ▪ Nanometer - sized lipid - bilayer vesicles ▪ Produced via endosome fusion ▪ Transfer proteins and RNAs ▪ Rich in miRNAs ▪ Present in virtually all body fluids ▪ Released by nearly all cell types Non - confidential
Slide 36: CDC Exosomes: Cardioprotection
CDC Exosomes: Cardioprotection CTRL Skin - Exosomes CDC - Exosomes Ibrahim et al., Stem Cell Reports , 2014 Non - confidential 0 5 10 15 20 25 30 35 40 45 50 CTRL Skin-XO CDC-XO Viable Mass (g) ** ** 0 2 4 6 8 10 12 14 16 18 CTRL Skin-XO CDC-XO Scar Mass (mg) ** ** 25 30 35 40 45 50 1 15 30 EF (%) D
Slide 37: Exosomes as a potential new therapeutic
Exosomes as a potential new therapeutic Product Description Product Function Product Mechanism Target Indications Intercellular signaling and regulation Promotes similar paracrine effects of CDCs; prevent apoptosis, proliferation, angiogenesis, anti - fibrotic Released by nearly all cell types and b
Slide 38: Exosomes: Development Plan
Exosomes: Development Plan ▪ Ongoing pre - clinical work ▪ Developing manufacturing and pre - commercial scale - up ▪ Exclusively licensed IP portfolio from Cedars - Sinai Medical Center ▪ Targeting IND 2015 - 2016 Non - confidential
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