Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (January 2016)
29 slides from Capricor Therapeutics's investor presentation, January 2016
Slide 1: Transformative Therapies from Bench to Bedside
Transformative Therapies from Bench to Bedside January 2016 NASDAQ: CAPR
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 Financial Highlights
3 Capricor Financial Highlights – Capricor, Inc. founded in 2005 (Baltimore, MD; JHU spinoff) – Completed reverse merger with Nile Therapeutics in November 2013 – Uplisted to NASDAQ in March 2015 – Total Cash as of Sept. 30, 2015: $17.2M (current cash out: ῀Q3 2016) – Non-dilutive capital funding to
Slide 4: Capricor's Platform & Therapeutic Pipeline
4 Capricor’s Platform & Therapeutic Pipeline Cardiosphere-Derived Cells CAP-1002 Natriuretic Peptide therapy Micro-RNA Platform
Slide 5: Cardiosphere-Derived Cells (CDCs)
5 Cardiosphere-Derived Cells (CDCs):
Slide 6: Cell Therapy Platform: CDCs (CAP-1002)
6 Cell Therapy Platform: CDCs (CAP-1002) Explant-derived Cardiospheres Cardiosphere-derived Cardiac Tissue Explants cells (EDCs) (CSps) cells (CDCs) Features Cardiosphere-Derived Cells (CDCs) Cell Type Human cardiac derived cell Characteristics Unique panel of cellular markers and secreted factors M
Slide 7: CADUCEUS: Proof of Concept First-in-Man Data
7 CADUCEUS: Proof of Concept First-in-Man Data Lancet, 2012, 21(6): 1121-1135. – Intracoronary delivery of autologous CDCs - 25M cells – Patients with reduced ejection fraction following MI – 25 patients (17 CDCs, 8 Controls) – Sponsored by Cedars-Sinai Medical Center – Results: CDCs reduced the amo
Slide 8: CADUCEUS: CDC Therapy Reduced Scar Size & Increased Healthy Heart Muscle
8 CADUCEUS: CDC Therapy Reduced Scar Size & Increased Healthy Heart Muscle Makkar et al, Lancet, 2012. CDC patients had a significant reduction in infarct (scar) size and an increase in healthy heart muscle mass. We hypothesize improvement in clinical outcomes.
Slide 9: CADUCEUS: Larger Implications for Medicine
9 CADUCEUS: Larger Implications for Medicine – Therapeutic regeneration may be possible – “Irreversible” injury may, in some cases, be reversible – Hope for curative approach rather than stabilization and palliation – Autologous manufacturing is not a viable business model in this indication
Slide 10: CDCs: Clinical Development
10 CDCs: Clinical Development ALLSTAR Clinical Trial Validate CADUCEUS Indication data POC with ALLO cells Phase I/ II Clinical Development Collaboration with Janssen Biotech (J&J) ῀$20M loan award from CIRM Phase II Enrolling Status Data anticipated: Q1 2017
Slide 11: Efficacy in Phase I Patients
11 Efficacy in Phase I Patients Phase II Equivalent Population = High Dose, wo DSAs Infarct Size Infarct Size 28 28 P<0.05* P<0.05# 26 26 24 24 Baseline IS (% LV) 22 IS (% LV) 22 12 Months 20 20 18 18 16 16 14 14 n=4 Baseline 12 Months *by groups t-test # by paired t-test
Slide 12: Post-MI LV Dysfunction
12 Post-MI LV Dysfunction – Advantages: – Proof of concept of therapeutic regeneration – Appreciable number will go on to develop overt heart failure – Disadvantages: – Rarefied population – Patients often asymptomatic – Low clinical event rates
Slide 13: CDCs: Clinical Development
13 CDCs: Clinical Development ALLSTAR Clinical Trial DYNAMIC Clinical Trial Validate CADUCEUS Adult Heart Failure Market Indication data (5M HF patients US POC with ALLO cells $32B/annual cost) Phase I/ II Phase I/II Clinical Development Collaboration with Janssen $3M funded by NIH Biotech (J&J) ῀$2
Slide 14: DYNAMIC: Concordance of Data Suggest Improvement
14 DYNAMIC: Concordance of Data Suggest Improvement LV Function & Dimensions (echo) Less is better NYHA class AHA: November 2015 6 month data for 2 subjects pending
Slide 15: DYNAMIC: Concordance of Data Suggest Improvement
15 DYNAMIC: Concordance of Data Suggest Improvement Less is better Quality of Life 6MWT & VO2 Max AHA: November 2015 6 month data for 2 subjects pending
Slide 16: CDCs: Clinical Development
16 CDCs: Clinical Development ALLSTAR Clinical Trial DYNAMIC Clinical Trial HOPE Clinical Trial Validate CADUCEUS Adult Heart Failure Market Orphan Disease Indication data (5M HF patients US Small market/ Big Upside POC with ALLO cells $32B/annual cost) Phase I/ II Phase I/II Phase I/II Clinical Dev
Slide 17: Orphan Drug Designation Granted to CAPR
17 Orphan Drug Designation Granted to CAPR – CAP-1002 can be used in CONJUNCTION with ANY other dystrophin- correcting therapies targeting skeletal muscle These therapies do not appear effective in cardiac muscle Very few clinical trials to treat DMD cardiomyopathy – Presents potential billion d
Slide 18: Global Cardiac Function and Exercise Capacity is improved in mdx Mice
18 Global Cardiac Function and Exercise Capacity is improved in mdx Mice Mdx+CDC 80 Repeat Dosing 820 CTL Mdx+Vehicle 760 Mdx+CDC 70 1st injection 2nd injection 700 Mdx+Vehicle 640 580 60 * *** 520 EF(%) * 460 50 * * 400 340 40 280 220 160 30 100 wk3 wk4 wk5 wk6 *** p<0.001 * p<0.05 n=12 Mdx + CDC,
Slide 19: Halt cardiomyOPathy progrEssion in Duchenne (HOPE)
19 Halt cardiomyOPathy progrEssion in Duchenne (HOPE) – FDA approved Phase I/II clinical trial (orphan designation) – Randomized, open label multi-center study (῀3-4 sites) 12 boys randomized to CDC (CAP-1002) infusion 12 boys randomized to ‘usual care’ – Triple vessel intra-coronary infusion –
Slide 20: Exosomes: Next Generation Regenerative Medicine Therapeutic Platform
20 Exosomes: Next Generation Regenerative Medicine Therapeutic Platform
Slide 21: Exosomes: Cell Free Regenerative Medicine
21 Exosomes: Cell Free Regenerative Medicine – Nanometer-sized lipid-bilayer vesicles – Rich in RNAs and proteins – Secreted by nearly all cell types – Cell signaling modality – Potential for broad therapeutic applicability – IP: Exclusive world-wide license agreement with Cedars-Sinai Medical Cente
Slide 22: CDC Exosomes Improve Cardiac Function and Preserve Muscle Mass
22 CDC Exosomes Improve Cardiac Function and Preserve Muscle Mass 50 Control CDC-XO ** 45 NHDF-XO * EF (%) 40 MSC-XO 35 CTRL NHDF-Exosomes 30 25 1 15 30 Days post MI 18 ** 16 ** 14 * MSC-Exosomes CDC-Exosomes Scar Mass (mg) 12 10 8 6 4 2 0 CTRL NHDF-XO MSC-XO CDC-XO Ibrahim et al, Stem Cell Reports,
Slide 23: Exosomes: Targeted Milestones
23 Exosomes: Targeted Milestones – Targeting announcing First-in-Man clinical indication: Q1 2016 – Indications under consideration: Eyes Skin Cancer – Meet with FDA: Q1 2016 – Targeting IND submission: 2016
Slide 24: Natriuretic Peptide Technology
24 NATRIURETIC PEPTIDE TECHNOLOGY
Slide 25: Mayo Designed CD-NP/Cenderitide: Only Dual NP Receptor Activator
25 Mayo Designed CD-NP/Cenderitide: Only Dual NP Receptor Activator R I G S D M C-Terminus of DNP L K CD-NP S pGC-A agonist CNP L Cenderitide G Enhances renal function pGC-B agonist G L Suppresses aldosterone F G Anti-fibrotic C -S-S- C Promotes cell survival Endothelial regenerating G P Highly resi
Slide 26: The Opportunity
26 Cenderitide The Opportunity R I G S D M L S K G L G L F G C C G -S-S- P K S S L L R G D P N R H2 P 1st arm of N A P S Phase II + T S A complete
Slide 27: Cenderitide for Outpatient and Ambulatory Heart Failure
27 Cenderitide for Outpatient and Ambulatory Heart Failure Target Prevention of re-hospitalization in patients with a recent acute heart failure admission as well as other potential Indication indications – Phase IIa PK/PD Trial 1st arm - 14 patients treated, enrollment complete Patients with st
Slide 28: Senior Management & Board of Directors
28 Senior Management & Board of Directors Senior Management Board of Directors – Chief Executive Officer – Executive Chairman Linda Marbán, Ph.D. (Founder, JHU, Cleveland Frank Litvack, M.D. (ConorMed) Clinic) – Linda Marbán, Ph.D. – Chief Medical Officer – Dave Musket (ProMed Partners) Deborah Asch
Slide 29: Transformative Therapies from Bench to Bedside
Transformative Therapies from Bench to Bedside www.capricor.com January 2016 NASDAQ: CAPR
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