NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.

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NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Corporate & Investor Presentation
December 2020                                                                      NASDAQ: CAPR
Capricor Therapeutics, Inc.         Developing Transformative Therapies from Bench to Bedside
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Forward-Looking Statements
     Statements in this presentation regarding the efficacy, safety, and intended utilization of Capricor's
     product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical
     trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and
     clinical trials; regulatory developments involving products, including the ability to obtain regulatory
     approvals or otherwise bring products to market; plans regarding current and future collaborative
     activities and the ownership of commercial rights; scope, duration, validity and enforceability of
     intellectual property rights; future royalty streams, revenue projections; expectations with respect to
     the expected use of proceeds from the recently completed offerings and the anticipated effects of the
     offerings, and any other statements about Capricor's management team's future expectations, beliefs,
     goals, plans or prospects constitute forward-looking statements within the meaning of the Private
     Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact
     (including statements containing the words "believes," "plans," "could," "anticipates," "expects,"
     "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be
     forward-looking statements. There are a number of important factors that could cause actual results
     or events to differ materially from those indicated by such forward-looking statements. More
     information about these and other risks that may impact Capricor's business is set forth in Capricor's
     Annual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities and
     Exchange Commission on March 27, 2020 and in our Quarterly Report on Form 10-Q for the quarter
     ended September 30, 2020 as filed with the Securities and Exchange Commission on November 13,
     2020. All forward-looking statements in this press release are based on information available to
     Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking
     statements.
     CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s
     exosome-based candidates have been approved for clinical investigation.
Capricor Therapeutics, Inc.                                   Developing Transformative Therapies from Bench to Bedside   2
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Corporate Summary
  • Cell and exosome-based platform therapeutics company
                                                                                                                NASDAQ
  • Two products with novel approaches to neuromuscular, infectious,                                         Ticker: CAPR
    inflammatory and cardiovascular diseases
                                                                                                            Cash (9/30/20) 1
                   Cell Therapy                       Exosomes Platform                                      $35.3 million
       Cardiosphere-derived cells (CAP-1002)   Engineered Exosomes & CDC-Exosomes
                                                                                                            Common shares1
                                                                                                              20.4 million
  • Late-stage clinical development in DMD and rapidly progressing
    program in COVID-19                                                                                   Non-Dilutive Capital
                                                                                                              $45 million
  • Expanding engineered exosome platform delivering RNA
                                                                                                         External Collaborators
  • Over 100 publications from multiple institutions worldwide on both                                      US Army
    platforms with extensive in-vivo and clinical data                                             US Department of Defense
                                                                                                      Stephen Gould, Ph.D.
  • Efficient use of capital including non-dilutive sources                                        Cedars-Sinai Medical Center

  • Experienced management team

                                                                                                                1As   of 11/13/20 as reported in Form 10Q

Capricor Therapeutics, Inc.                                          Developing Transformative Therapies from Bench to Bedside                      3
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Capricor’s Product Pipeline
                                   Target                         Development Phase
          Candidate              Indications        Discovery   Preclinical    Phase I        Phase II          Phase III                Status
             CAP-1002           Duchenne Muscular
                                                                                                                               Positive Phase II reported
         (allogeneic CDCs)          Dystrophy

             CAP-1002
                                    COVID-19                                                                                   Actively recruiting patients
         (allogeneic CDCs)

     Exosome mRNA Vaccine
                                   SARS-CoV-2                                                                                        In development
      Tripartite mRNA design

      Exosome VLP Display
              Vaccine              SARS-CoV-2                                                                                        In development
       4-part antigen design

           CDC-Exosomes         Duchenne Muscular
                                                                                                                                      IND submitted
       (allogeneic CDC-XOs)         Dystrophy

      Engineered Exosomes
                                    Evaluating                                                                                            Platform
          (RNA delivery)

        ASTEX-Exosomes
      (engineered fibroblast-       Evaluating                                                                                            Platform
           derived XOs)

            Cell Therapy           Exosome Platform

                                                                              Capricor's exosomes technology has not yet been approved for clinical investigation.
Capricor Therapeutics, Inc.                                             Developing Transformative Therapies from Bench to Bedside                             4
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Exosome
       Platform
       Overview

Capricor Therapeutics, Inc.   Developing Transformative Therapies from Bench to Bedside
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Exosomes Platform
   Potential New Class of Therapeutics

             Extracellular vesicles - term for cell-derived
              vesicles, including exosomes

             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 Center for IP
              rights related to the exosomes technology
              originating from cardiosphere-derived cells
              (CDCs)

                                                                                                       Kidney International (2010) 78, 838–848

Capricor Therapeutics, Inc.                                    Developing Transformative Therapies from Bench to Bedside                         6
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
From Discovery to Platform Development

       Publications
      covering our
    technology have
   been published by
         us or our
     collaborators in
      multiple peer-
   reviewed journals.

Capricor Therapeutics, Inc.   Developing Transformative Therapies from Bench to Bedside   7
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Capricor’s Potential Solutions to
  Complex Problems

                              Problems                                  Possible Solutions

          1. Gene therapy using viral delivery             Exosomes:
             (AAV)                                            ‒ nature’s delivery vehicle
             ‒   Immune response
                                                              ‒ low immunogenicity
          2. Delivery of RNAs
             ‒   Uptake to render biologic relevance          ‒ can deliver contents to the cell
                                                                without integration
             ‒   Therapeutic development slow
          3. Synthetic nanoparticles are                      ‒ can be targeted (tropism)
             untargeted delivery vehicles
                                                              ‒ can be lyophilized for ease of
                                                                handling

Capricor Therapeutics, Inc.                            Developing Transformative Therapies from Bench to Bedside   8
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Exosomes: Building a New Class
   of Therapeutics

                     Infectious     Oncology           Ongoing Collaborations
                      Diseases                         • Stephen Gould, Ph.D.
                                    Vaccines &
                                                       • US Army Institute for Surgical Research
                 mRNA and VLP        targeted
                                                       • US Department of Defense
                    vaccine          delivery
                  approaches       therapeutics        • Cedars-Sinai Medical Center

                                  Neuromuscular
                    Monogenic                          Goals
                                  & Inflammatory
                     Diseases                          • Drive innovation and research through
                                    Protein or           collaborations
                      mRNA
                                      mRNA             • Scale and partner
                   therapeutics
                                   therapeutics        • Expand and exploit platform and IP
                                                         through partnerships

Capricor Therapeutics, Inc.                        Developing Transformative Therapies from Bench to Bedside   9
NASDAQ: CAPR Corporate & Investor Presentation December 2020 - Capricor Therapeutics, Inc.
Engineered Exosome
Platform

mRNA and VLP
Exosome-based Vaccines
                         10
Exosomes Target Cargo Directly to the Cell
                              ‒ Exosomes serve as a natural signaling
                                system
                              ‒ CD-9, CD-81 and CD-63 are surface
                                markers of exosomes and can serve as
                                targeting molecules for effective delivery
                                to cells and confirmation of exosome
                                loading

Capricor Therapeutics, Inc.      Developing Transformative Therapies from Bench to Bedside   11
Coronavirus Structure
   Unique Protein Visualization

                                                                ‒ Other mRNA vaccines in
                                                                  development are targeting
                                                                  the spike “S” protein solely
                                                                ‒ Capricor’s exosome
                                                                  platform vaccine addresses
                                                                  all 4 proteins: E,M,N,S

         4 antigens likely to yield increased
                protective response

     Two unique vaccine approaches utilizing exosomes:
     (1) mRNA & (2) VLP vaccines

Capricor Therapeutics, Inc.                     Developing Transformative Therapies from Bench to Bedside   12
Capricor’s Exosome-Based Vaccines
   Approach is Distinctive

    Exosome-based approach
      ‒ Allows for specificity in cellular targeting
      ‒ Innovative technology - yet is well characterized
    Unique 4-antigen approach
      ‒ Potentially confers greater immunity by targeting all 4 antigens
    Exosomes potentially superior to liposomes
      ‒ Not recognized as foreign by immune system
      ‒ Deliver payload directly to cytoplasm
      ‒ Superior targeting permits lower doses
      ‒ Target both T and B cells – allows for immediate activity and long-term memory
    Two differentiated vaccine approaches under development
      ‒ Platform permits accelerated innovation of both mRNA and VLP vaccines

Capricor Therapeutics, Inc.                    Developing Transformative Therapies from Bench to Bedside   13
Exosome mRNA Vaccine

       Exosome-based mRNA vaccine can have the following potential benefits:
         ‒ Delivering payload directly to cytoplasm
         ‒ Superior targeting may permit lower doses
         ‒ Exosome based mRNA vaccine will have all 4 proteins for better immune response
         ‒ Exosomes address the delivery problem by targeting cells of interest

Capricor Therapeutics, Inc.                          Developing Transformative Therapies from Bench to Bedside   14
Exosome-mRNA Vaccine Preclinical Results
   Key findings:
   •    Development of safe, non-toxic exosome
        formulation capable of delivering functional
        mRNA in vitro and in vivo
   •    Creation of a multiplexed exosome-RNA vaccine
        that expresses viral antigens engineered to
        induce cellular immunity and antibody responses
        to multiple proteins of SARS-CoV-2
   •    Validation that an exosome mRNA vaccine can
        induce:
       •     Persistent cellular immune responses to
             the SARS-CoV-2 N and S proteins
       •     Moderate      but     sustained   antibody
             responses to the SARS-CoV-2 N and S
             proteins
   •    Following a low-dose immunization protocol, no
        evidence of vaccine-induced adverse events
                                                                                   Exosome-mRNA vaccine
                                                                                        demonstrates
                                                                                     long lasting cellular
                                                                                          immunity

                                                                                                         Results from bioRxiv, Gould Nov. 2020

Capricor Therapeutics, Inc.                               Developing Transformative Therapies from Bench to Bedside                    15
Exosome-mRNA Vaccine Preclinical Results

         Exosome-mRNA
       vaccine demonstrates
       long lasting humoral
             immunity

                                                                           Results from bioRxiv, Gould Nov. 2020

Capricor Therapeutics, Inc.   Developing Transformative Therapies from Bench to Bedside                  16
Exosome VLP: Display Vaccine Approach*

                Capricor’s exosome
            platform vaccine addresses
               all 4 proteins: N,S,M,E
                                                           *Makes use of highly optimized expression system developed by the Gould lab

Capricor Therapeutics, Inc.              Developing Transformative Therapies from Bench to Bedside                               17
CAP-1002
Cell Therapy
Overview
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 engraft
     into host tissue

   • MOA: cells secrete exosomes:
          ‒ Contain miRNAs, non-coding RNAs and proteins
          ‒ Internalized by target cells
          ‒ Stimulate diverse and lasting changes in cellular
            behavior
          ‒ 3 known miRNAs drive CAP-1002 potency

   • CAP-1002 has been investigated in multiple
     independent clinical trials and approximately 200
     human subjects to date

Capricor Therapeutics, Inc.                              Developing Transformative Therapies from Bench to Bedside   19
Mechanism of Action:
   Defined in “Stem Cell Reports”
                                         phospho-Akt                         HO-1            GCLC cat. sub.
           Oxidative
                                         Nrf2 (cytoplasmic)
            Stress                                                            catalase        SOD-2
                                         Nrf2 (nuclear)

                                                                phospho-IkB                 CD68+ macrophages
         Inflammation                    NF-kB                 p65 (nuclear)
                                                                MCP1                        CD3+ T cells

                                         collagen I
             Fibrosis
                                         collagen III

                                         mitochondrial DNA copy number                         RESTORED mitochondrial ultrastructure
        Cellular Energy                                                                         NORMALIZED deficient respiratory capacity
                                         level of respiratory chain subunits                                of isolated mitochondria

                                         Ki67+ cardiomyocytes
          Muscle Cell
          Generation                     Aurora B cardiomyocytes

*CDCs have been the subject of >100 peer-reviewed papers since 2007.
 Aminzadeh et al. Stem Cell Reports. 2018.

Capricor Therapeutics, Inc.                                                      Developing Transformative Therapies from Bench to Bedside   20
CAP-1002
Duchenne
Muscular
Dystrophy
Program

            21
DMD: Lack of Dystrophin Predisposes
  Muscle to Damage

     • Dystrophin is a structural protein
       located within the muscle fiber membrane
                                                                                                  Whole Muscle
     • Acts both as a cushion and a kind of glue                                                    Tissue

     • Without dystrophin, muscles are unable to
       function properly, suffer progressive damage and                                              Muscle-Fiber
       eventually die                                                                                 Membrane

     • Much of the muscle injury that occurs in
       dystrophin-deficiency is attributable to
       secondary damage caused by inflammation

                                                                                      Dystrophin

Capricor Therapeutics, Inc.                        Developing Transformative Therapies from Bench to Bedside     22
Trajectory of CDCs in DMD (Preclinical Data)
                 • Hypothesis: CDCs to treat cardiomyopathy
                 • Left ventricular ejection fraction markedly improved vs. control
                      – P
Capricor’s Addressable DMD Population

                                                                 CAPRICOR’s
                                                               Targeted Patient
                                                                  Population

Capricor Therapeutics, Inc.   Developing Transformative Therapies from Bench to Bedside   24
Competitive Landscape for DMD
                    Options                Challenges                            CAP-1002 Benefits

                                    Exon Skipping – treats a small
                                   portion of the DMD population                    Immunomodulatory
                  Exon Skipping
                                   Gene therapy – potential safety                       Anti-fibrotic
                   Gene therapy                risks
                                                                                      Pro-regenerative
                       NF-kB        NF-kB inhibition may not be
                                              enough                                    Cellular Energy
                      Steroids
                                     Steroids have adverse side-
                                               effects

       We believe CAP-1002 may be used synergistically with other therapeutics aimed to
                                        treat DMD

Capricor Therapeutics, Inc.                          Developing Transformative Therapies from Bench to Bedside   25
Primary Efficacy Endpoint
   Performance of the Upper Limb (PUL: v1.2) to Assess Skeletal Muscle

               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 change at 12 months at all levels of ability*

                    *Mayhew et al, 2019; Pane et al, 2018.
Capricor Therapeutics, Inc.                                     Developing Transformative Therapies from Bench to Bedside   26
Capricor’s Regulatory Designations - DMD
   GOAL OF FDA’S RMAT DESIGNATION
   To facilitate efficient development and expedite review of a drug

   Similar to breakthrough therapy designation:
      • RMAT provides benefits that include more frequent meetings with FDA to discuss the development
        plan for the product candidate
      • Eligibility for rolling review and priority review

   Products may also be eligible for accelerated approval
      • On the basis of a surrogate or intermediate endpoint reasonably likely to predict long-term clinical
        benefit
      • Reliance upon data obtained from a meaningful number of sites
                                                                                     Rare Pediatric
                                                                                  Disease Designation

                         RMAT Designation

                                                                                        Orphan
                                                                                    Drug Designation

Capricor Therapeutics, Inc.                                  Developing Transformative Therapies from Bench to Bedside   27
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 corticosteroids
     • Very limited options for this patient population

       RESULTS
       • Reduction in cardiac scar at 6 and 12 months measured by
         MRI
       • Improvement in cardiac function (systolic wall thickening) at
         6 and 12 months
       • Improvements shown in PUL (mid + distal)
          – Best improvement shown within the first 3 months
       • Study published in February 2019 in Journal of Neurology
                                                                                                 https://n.neurology.org/content/92/8/e866.
                                                                                                 Study funded with the support of CIRM
                                                                                                 https://clinicaltrials.gov/ct2/show/NCT02485938.

Capricor Therapeutics, Inc.                                      Developing Transformative Therapies from Bench to Bedside                          28
HOPE-Duchenne: Phase I/II Results
  Reduced Cardiac Scar and Improved PUL

    Scar

                              R.G. Victor et al., AHA LBCT 2017; M. Taylor et al., submitted

                                                                                                           *p-values are based on absolute change from baseline

Capricor Therapeutics, Inc.                                                Developing Transformative Therapies from Bench to Bedside                              29
HOPE-2
Phase II Clinical Study

                          30
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-1002

    • Dosing Regimen: 150M cells delivered
      intravenously every 3 months

    • Sites: 9 sites (USA)

    • Data: ITT population - 20 subjects

    • Demographics

       ‒ Mean age: 14.3 years

       ‒ All patients were on corticosteroids

       ‒ ~ 80% of patients were non-ambulant

                                                                           https://www.clinicaltrials.gov/ct2/show/study/NCT03406780.

Capricor Therapeutics, Inc.                                 Developing Transformative Therapies from Bench to Bedside             31
Clinically Meaningful Changes Observed in PUL 2.0
   (Shoulder + Mid + Distal)
                                               PUL 2.0 (full)                                                                                              PUL 2.0 (full)

                                                                                                                                                          MONTH 12/ET
                                               MONTH 12/ET

                                                                                                     Mean Change from Baseline +/- SEM
      Mean Change from Baseline +/- SEM

                                                                                                                                         4
                                          0                                    CAP-1002
                                                                               PLACEBO                                                   2
                                          -1                                                                                             0

                                                                                                                                         -2
                                          -2
                                                                             improvement                                                 -4
                                          -3
                                                                                                                                         -6                  p=0.0201
                                                                                                                                                        (t test; two-tailed)

                                          -4                                                                                             -8

                                                                                                                                                   02

                                                                                                                                                                                          O
                                                 p=0.0532

                                                                                                                                                                                     EB
                                                                                                                                                 10
                                          -5

                                                                                                                                                                              C
                                                                                                                                               P-

                                                                                                                                                                             A
                                                                                                                                               A

                                                                                                                                                                          PL
                                                                                                                                              C
                                                Δ2.4 points in CAP-1002 vs. placebo at 12-months

                                                                                                                                                                 Comparisons treated vs. placebo using mixed model repeated measures ANOVA
                                                                                                                                                                 with covariates at baseline, 3 months, 6 months, 9 months and 12 months
                                                                                                                                                                 P-values are nominal values unadjusted for multiple testing

Capricor Therapeutics, Inc.                                                                        Developing Transformative Therapies from Bench to Bedside                                                                        32
Cardiac Improvements Observed
  Ejection Fraction %, CK-MB, LV-ESV and LV-EDV
                                                                        LV EF (%)                                      LV ES Volume, Indexed, ml/m2

                                                                       MONTH 12/ET                                                                          MONTH 12/ET

                                                                                                               Mean Change from Baseline +/- SEM
                              Mean Change from Baseline +/- SEM
                                                                                            CAP-1002                                                                          CAP-1002
                                                                  1                                                                                4
                                                                                            PLACEBO                                                                           PLACEBO
                                                                                                                                                                                                         Has been used as a
                                                                  0                                                                                2                                                   surrogate endpoint for
                                                                                                                                                                                                       approval in adult heart
                                                                                                                                                                                                               failure
                                                                  -1                      improvement                                              0

                                                                                                                                                                              improvement
                                                                  -2                                                                               -2
                                                                         p=0.0042                                                                               p=0.0122
                                                                  -3                                                                               -4

                                                                                                                                   LV ED Volume, Indexed, ml/m2
                         Creatine Kinase MB/Total Creatine Kinase (%)
                                    Change From Baseline                                                                                                     MONTH 12/ET

                                                                                                               Mean Change from Baseline +/- SEM
                                                                                                                                                                               CAP-1002
                                                     4                      ✱✱✱                                                                         5
                                                                                                CAP-1002                                                                       PLACEBO
                                                     3
                                                                                                Placebo                                                 0
                                                     2
                         % CK-MB

  Enzyme associated                                  1                                                                                                                        improvement
   with breakdown                                                                                                                                   -5
   of cardiac muscle                                 0
          cells                                                                              improvement
                                              -1                                                                                                   -10
                                                                                p=0.006
                                                                                                                                                                   p=0.0699
                                              -2
                                                                           Month 12                                                                -15
                                                                                                                                                                                Comparisons treated vs. placebo using mixed model repeated measures ANOVA
                                                                             Visit                                                                                              with covariates at baseline, 3 months, 6 months, 9 months and 12 months
                                                                                                                                                                                P-values are nominal values unadjusted for multiple testing

Capricor Therapeutics, Inc.                                                                                Developing Transformative Therapies from Bench to Bedside                                                                               33
HOPE-2 Safety Results
   •      A total of 69 infusions (CAP-1002 or placebo) were performed in
          HOPE-2
         •      Generally safe and well tolerated throughout the study
         •      With the exception of hypersensitivity reactions, no safety
                signals were identified

Capricor Therapeutics, Inc.                      Developing Transformative Therapies from Bench to Bedside   34
Conclusions and Future Directions
      Conclusions:                               Moving Forward:
         ‒ First placebo-controlled trial         ‒ FDA continues to encourage us to
           showing upper limb functional            conduct a Phase III study; we continue
           improvements in non-ambulant             to discuss next steps and pathway to
           DMD patients                             approval
         ‒ Directionally consistent               ‒ Engaged Lonza (global CMO) for scale-
           improvements in strength,                up of manufacturing of CAP-1002
           respiratory and cardiac endpoints      ‒ HOPE-2 Open label extension trial
         ‒ First ever study in DMD that             underway
           correlates cardiac functional
           stabilization with reduction of a
           biomarker of myocardial cell
           damage
         ‒ Consistent results shown pre-
           clinically, Phase I/II and Phase II

Capricor Therapeutics, Inc.                      Developing Transformative Therapies from Bench to Bedside   35
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 Hospital (USA)

         Pat Furlong                          Parent Project Muscular Dystrophy (USA)

         Kan Hor, M.D.                        Nationwide Children's Hospital (USA)

                                              Cincinnati Children's Hospital Medical Center
         John Jefferies, M.D.
                                              (USA)

         Oscar Henry Mayer, M.D.              Children's Hospital of Philadelphia (USA)

         Eugenio Mercuri, M.D., Ph.D.         Catholic University of the Sacred Heart (Italy)

         Francesco Muntoni, M.D.              University College London (UK)

         Thomas Voit, M.D.                    University College London (UK)

         Lee Sweeney, Ph.D.                   University of Florida (USA)

                                              Cincinnati Children's Hospital Medical Center
         Michael Taylor, M.D., Ph.D.
                                              (USA)

Capricor Therapeutics, Inc.                   Developing Transformative Therapies from Bench to Bedside   36
CAP-1002
       Cell Therapy Overview
       for COVID-19

Capricor Therapeutics, Inc.    Developing Transformative Therapies from Bench to Bedside   37
CAP-1002 Targets Severe Cases of COVID-19

Capricor Therapeutics, Inc.   Developing Transformative Therapies from Bench to Bedside   38
Immunomodulatory Effects of CAP-1002

                                                 CDCs: Cardiosphere-derived cells

          Macrophages                     Proinflammatory                          Systemic Inflammation                      1.     Cardiac inflammation
                                             Cytokines                                                                        2.     Lung inflammation
         Effector T-cells                                                                                                     3.     Cardiomyocyte death
                                                                               Multiorgan dysfunction                         4.     Cardiac dysfunction
                                          IFN-γ, TNFα, IL-1β,
                                          IL-6, IL-8, CXCL10,                                                                 5.     Skeletal muscle injury
                                           CCL2, CCL3, CCL5                                                                   6.     Tissue Fibrosis

              CDCs: Mechanism of Action          CDCs: Pro-inflammatory cellular                          CDCs: Efficacy (Pre-clinical and Clinical)
                                                             targets

         1.    Cardiomyogenesis                                                             1.   Myocardial ischemia (CADUCEUS, Phase I/II ALLSTAR,
         2.    Cardiomyocyte survival       1.     Enhanced cell debris                          DYNAMIC Phase IIa)
         3.    Anti-inflammatory            2.     Decreased TNFα, IL-1β, CCL5              2.   Myocarditis
         4.    Immunomodulatory                    production                               3.   Muscular dystrophy (HOPE-Duchenne, HOPE-2)
         5.    Angiogenic                   3.     Increased levels of IL-10 by             4.   Heart failure with preserved ejection fraction (REGRESS,
         6.    Anti-fibrotic                       macrophages                                   Phase I)
                                                                                            5.   Senescence
                                                                                            6.   Non-ischemic dilated cardiomyopathy
                                                                                            7.   Pulmonary arterial hypertension (ALPHA, Phase I)

                                                                                                       Published https://link.springer.com/content/pdf/10.1007/s00395-020-0795-1.pdf

Capricor Therapeutics, Inc.                                                          Developing Transformative Therapies from Bench to Bedside                                 39
CAP-1002: COVID-19 Program
   Compassionate Use Data

  Six critical COVID-19 patients with ARDS
  Published in Basic Research in Cardiology*
    ‒ Treated at Cedars-Sinai Medical Center in Los Angeles, CA
    ‒ Received intravenous infusions of 150 million cells of CAP-1002
  Results:
    ‒ Within 1-4 days following infusion
         ‒ 4 of 5 patients no longer required ventilator support
  Improved biomarkers:
    ‒ Ferritin, absolute lymphocyte count and CRP
  No adverse events related to the administration of CAP-1002 were observed

                                              *Published https://link.springer.com/content/pdf/10.1007/s00395-020-0795-1.pdf

Capricor Therapeutics, Inc.                       Developing Transformative Therapies from Bench to Bedside                40
CAP-1002: COVID-19 Program
   Phase II Trial Underway
      ‒ INSPIRE: Phase II, randomized, double-blind, placebo-controlled trial
      ‒ Aim: to treat up to 60 patients in the US
      ‒ Study enrolling patients who have a confirmed diagnosis of SARS-CoV-2 and require
        supplemental oxygen (severe to critical)
      ‒ Patient participation will be a maximum of 13 weeks from screening
      ‒ Trial actively recruiting subjects

Capricor Therapeutics, Inc.                          Developing Transformative Therapies from Bench to Bedside   41
Targeted Milestones into 2021
    Cell Therapy (CAP-1002) Technology
     1H 2021: Plan to publish HOPE-2 final results
     1H 2021: Plan to complete enrollment in INSPIRE study
     1H 2021: Plan to announce results from INSPIRE study
     2021: Continue to work with FDA on next steps in pathway forward for DMD
     2021: Continue to pursue partnership opportunities for DMD program
    Engineered Exosomes Platform Technology
     1Q 2021: Plan to meet with FDA in PRE-IND meeting for mRNA vaccine
     1H 2021: Plan to publish preclinical data from exosomes technology
     1H 2021: Plan to announce pipeline expansion for engineered exosome program
     2021: Plan to announce updates on vaccine program for COVID-19
     2021: Continue to pursue grant funding activities
     2021: Continue to pursue partnership opportunities

Capricor Therapeutics, Inc.                      Developing Transformative Therapies from Bench to Bedside   42
World-Class
Facilities and    Capricor’s Research, Development and Manufacturing facilities are
                   located in the Cedars-Sinai Medical Center in Los Angeles, California
Infrastructure    Capricor has access to core research facilities

                                                                                           43
Senior Leadership Team
                         Linda Marbán, Ph.D.                                                                           Stephen Gould, Ph.D.
                         Chief Executive Officer, Co-founder and Director                                              Executive Consultant
                                                                                                                              Dr. Gould is a Professor of Biological Chemistry at Johns Hopkins
                              Dr. Marbán has over 25 years of experience in the biotechnology
                                                                                                                               University and an internationally recognized exosome expert
                               industry
                                                                                                                               who brings an unparalleled understanding of exosome
                              Been with Capricor since 2005 and CEO since 2010.
                                                                                                                               engineering to Capricor.
                              Previous experience includes Excigen, Inc. where she was
                                                                                                                              Dr. Gould is co-Founder and acting President of the American
                               responsible for business development and operations.
                                                                                                                               Society for Exosomes and Microvesicles (ASEMV).
                              Dr. Marbán began her career in academic science at the
                                                                                                                              Dr. Gould’s team was the first to reveal the mechanistic link
                               Cleveland Clinic Foundation working on the biophysical
                                                                                                                               between exosome biogenesis and virus budding, the first to
                               properties of cardiac muscle and continued to a postdoctoral
                                                                                                                               identify mechanisms of exosome engineering and the first to
                               fellowship at Johns Hopkins University.
                                                                                                                               develop an exosome-based cancer therapeutic.
                              Dr. Marbán earned a Ph.D. from Case Western Reserve University
                                                                                                                              Dr. Gould has published numerous research articles and several
                               in cardiac physiology.
                                                                                                                               book chapters, received numerous public and private research
                                                                                                                               grants and served on an array of NIH and other grant review
                         Karen Krasney, J.D.                                                                                   panels.
                         Executive Vice President & General Counsel
                              Ms. Krasney’s has over 40 years of experience in domestic and
                               international corporate and business law, as well as litigation.
                              Ms. Krasney served as legal counsel of Biosensors International                             Sudhir Borgonha, M.D., M.B.A.
                               Group Ltd., a multinational medical device company.                                         Vice President of Clinical Development
                              Ms. Krasney received her Bachelor of Arts degree from the                                        Dr. Borgonha previously served as Director of Translational
                               University of California, Los Angeles and her Juris Doctorate from                                Medicine at the Fanconi Anemia Research Fund, where he
                               the University of Southern California.                                                            led efforts to propel a spectrum of approaches including
                                                                                                                                 gene therapy to treat cancer in rare diseases. Prior to that,
                                                                                                                                 he was the Medical Director of Strand Genomics, where he
                                                                                                                                 oversaw development of new technologies such as the
                                                                                                                                 liquid biopsy and customized cancer panels, while he
                     AJ Bergmann, M.B.A.                                                                                         oversaw clinical reporting for over 5,000 patients a year.
                     Chief Financial Officer                                                                                    Dr. Borgonha was a co-founder of Angstrom Medica
                            Mr. Bergmann has worked in the finance industry for over a                                          (Acquired by Pioneer Surgical), a biomaterials science
                             decade.                                                                                             company. He is also a co-founder of ten3T, a
                            Mr. Bergmann joined Capricor in 2011 and coordinated the                                            remote cardiac monitoring company.
                             Company’s reverse merger, uplisting to NASDAQ and public                                           Dr. Borgonha is a graduate of St. John’s Medical
                             financings yielding over $85 million to date.                                                       College, Bangalore and the Sloan School of Management
                            Mr. Bergmann graduated from Providence College and has a                                            at MIT.
                             M.B.A. from the University of Southern California’s Marshall
                             School of Business.

Capricor Therapeutics, Inc.                                                                         Developing Transformative Therapies from Bench to Bedside                                44
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