Capricor Therapeutics, Inc. · NASDAQ:CAPR
Capricor Therapeutics — Investor Presentation (March 2020)
32 slides from Capricor Therapeutics's investor presentation, Mar 11, 2020
Slide 1: Corporate & Investor Presentation March 2020
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside NASDAQ : CAPR Corporate & Investor Presentation March 2020
Slide 2: Forward-Looking Statements
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Forward - Looking Statements 2 Statements in this presentation regarding the efficacy, safety, and intended utilization of Capricor's product candidates ; the initiation, conduct, size, timing and results of discov
Slide 3: Investment Highlights
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3 Investment Highlights Synergistic Asset Portfolio ▪ Phase II – cardiac cell therapy for DMD ▪ Preclinical – exosome platform technology ▪ Core IP licensed from the Johns Hopkins University, Cedars - Sinai Medical
Slide 4: Capricor's Product Pipeline
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s Product Pipeline 4 Candidate Target Indications Development Phase Status Discovery Preclinica l Phase I Phase II Phase III CAP - 1002 (allogeneic CDCs) Duchenne Muscular Dystrophy Phase II final data exp
Slide 5: Capricor's CAP-1002 Technology
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s CAP - 1002 Technology CAP - 1002 is a biologic consisting of allogeneic cardiosphere - derived cells (CDCs) • Manufactured from donated heart muscle • Does not act by “stemness” - the cells do not engraf
Slide 6: CAP-1002 Duchenne Muscular Dystrophy Program
CAP - 1002 Duchenne Muscular Dystrophy Program 6
Slide 7: Lack of Dystrophin Predisposes Muscle to Damage
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Lack of Dystrophin Predisposes Muscle to Damage • Dystrophin is a structural protein located within the muscle fiber membrane • Acts both as a cushion and a kind of glue • Without dystrophin, muscles are unable to
Slide 8: Mechanism of Action: Defined in Stem Cell Reports
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Mechanism of Action: Defined in “Stem Cell Reports” Oxidative Stress Fibrosis Inflammation Cellular Energy Muscle Cell Generation phospho - Akt Nrf2 (cytoplasmic) Nrf2 (nuclear) HO - 1 catalase SOD - 2
Slide 9: Trajectory of CDCs in DMD (Preclinical Data)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Trajectory of CDCs in DMD (Preclinical Data) • Hypothesis: CDCs to treat cardiomyopathy • Left ventricular ejection fraction markedly improved vs. control – P <0.05 at all timepoints through 12 weeks of follow - up
Slide 10: Capricor's Addressable Market
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s Addressable Market CAPRICOR’s Targeted Patient Population 10
Slide 11: Competitive Landscape for DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Competitive Landscape for DMD We believe CAP - 1002 may be used synergistically with other therapeutics aimed to treat DMD Exon Skipping Gene therapy NF - kB Steroids Options Challenges CAP - 1002 Benefits Exon Ski
Slide 12: Primary Efficacy Endpoint PUL v.2.0
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Primary Efficacy Endpoint PUL v.2.0: • 3 - point response scale - more robust and reproducible than v1.2 • Compensatory strategies allowed to achieve tasks (not allowed in v1.2) • v2.0: better able to detect cha
Slide 13: Capricor's Regulatory Designations - DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s Regulatory Designations - DMD Similar to breakthrough therapy designation: • RMAT provides benefits that include more frequent meetings with FDA to discuss the development plan for the product candidate
Slide 14: HOPE-Duchenne Focused on Older DMD Patients
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - Duchenne Focused on Older DMD Patients • Phase I/II study: 25 patients, randomized and open - label • One - time, multi - vessel, intracoronary delivery of cells • HOPE population were all on stable corticos
Slide 15: HOPE-Duchenne: Reduced Cardiac Scar and Improved PUL
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - Duchenne: Reduced Cardiac Scar and Improved PUL R.G. Victor et al., AHA LBCT 2017; M. Taylor et al., submitted *p - values are based on absolute change from baseline INFERIOR WALL IMPROVEMENT Scar 15
Slide 16: HOPE-2 Phase II Clinical Study
HOPE - 2 Phase II Clinical Study 16
Slide 17: HOPE-2 Clinical Trial
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside HOPE - 2 Clinical Trial • Design: Phase II, randomized, double - blind, placebo - controlled trial in participants with DMD and reduced skeletal muscle function • Objective: Evaluate safety and efficacy of CAP - 10
Slide 18: Improvements in PUL 2.0
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Improvements in PUL 2.0 • As shown by these data, preservation of function is maintained for at least 6 months. • CAP - 1002 may offer a treatment for a primarily non - ambulant DMD population for whom no other opt
Slide 19: Improvements in Pulmonary Function Observed
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside -30 -25 -20 -15 -10 -5 0 5 10 15 20 Month 3 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoints PEF Percentage Predicted PLACEBO CAP-1002 -0.3 -0.2 -0.1 0.0 0.1 0.2 0.3 0.4 Month 3 Month 6 M e a n
Slide 20: Cardiac Function as Measured by MRI
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside -12 -7 -2 3 8 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoint Systolic Wall Thickening -Anterior PLACEBO CAP-1002 -12 -7 -2 3 8 Month 6 M e a n C h a n g e f r o m B a s e l i n e ± S D Timepoin
Slide 21: Conclusions and Future Directions
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Conclusions and Future Directions CAP - 1002 for DMD • Positive outcomes from two clinical trials showing improvements in skeletal and cardiac muscle function • First placebo - controlled trial showing upper limb f
Slide 22: World-Class DMD Advisory Board
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside World - Class DMD Advisory Board Craig McDonald, M.D. (National PI) University of California at Davis (USA) Michelle Eagle, Ph.D., M.Sc., MCSP Atom International Ltd (UK) Richard Finkel, M.D. Nemours Children's Hos
Slide 23: Exosomes Technology
Exosomes Technology 23
Slide 24: From Discovery to Platform Development
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside From Discovery to Platform Development 24 Publications covering our technology have been published by us or our collaborators in multiple peer - reviewed journals.
Slide 25: Extracellular Vesicles (Exosomes) Cell Free Regenerative Medicine
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Extracellular Vesicles (Exosomes) Cell Free Regenerative Medicine ▪ Extracellular vesicles - term for cell - derived vesicles, including exosomes ▪ Nanometer - sized lipid - bilayer vesicles ▪ Rich in RNAs and prot
Slide 26: The Field of Exosomes: Increasing Market Opportunity
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside The Field of Exosomes: Increasing Market Opportunity 26 Problems Possible Solutions Large Addressable Market The global market for exosome diagnostic and therapeutics is expected to reach revenue of $580 million by
Slide 27: Capricor's Potential Solutions to Complex Problems
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Capricor’s Potential Solutions to Complex Problems 1. Gene therapy using viral delivery (AAV) ‒ Immune response 2. Delivery of RNAs ‒ Uptake to render biologic relevance ‒ Therapeutic development very slow 3. Synth
Slide 28: Why Engineer Exosomes?
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Why Engineer Exosomes? Platform Technology 28 ▪ increase potency ▪ reduce variability ▪ help product development ▪ Cargo; nucleic acids (mRNA and miRNAs) ▪ Membrane Modifications; ▪ Tropism Change, Coating with imm
Slide 29: CDC-Exosomes: POC Established in Multiple Indications
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside CDC - Exosomes: POC Established in Multiple Indications LUPUS NEPHRITIS SEPSIS F4/80 + CD11b + Macrophage: 46.5% Macrophage: 14.5% Renal function Spleen size XOs reduce lymphadenopathy and improve renal function in
Slide 30: Targeted Exosome Platform Allows for Broad Applicability
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Targeted Exosome Platform Allows for Broad Applicability Regenerative Medicine 2.0 • Immunomodulation • Tissue regeneration CDC - XO (Exosomes) Specific Tropism • Modify XO membrane • Targeted delivery Surface - Mo
Slide 31: Recent and Targeted Catalysts in 2020
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Recent and Targeted Catalysts in 2020 31 CAP - 1002 in Duchenne Muscular Dystrophy x Feb. 2019: HOPE - Duchenne (Phase I/II) study published in Journal of Neurology x Oct. 2019 : Reported positive six - month resul
Slide 32: Senior Leadership Team
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Senior Leadership Team Linda Marbán, Ph.D. Chief Executive Officer, Co - founder and Director ▪ Dr. Marbán has over 25 years of experience in the biotechnology industry ▪ Been with Capricor since 2005 and CEO since
Capricor Therapeutics — Investor Presentation (March 2020) on 6ix