Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to BedsideNASDAQ: CAPR
Corporate & Investor Presentation June 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Forward-Looking Statements
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Statements in this presentation regarding the efficacy, safety, and intended utilization of Capricor'sproduct 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 clinicaltrials; regulatory developments involving products, including the ability to obtain regulatory approvals orotherwise bring products to market; plans regarding current and future collaborative activities and theownership 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 proceedsfrom the recently completed offerings and the anticipated effects of the offerings, and any otherstatements about Capricor's management team's future expectations, beliefs, goals, plans or prospectsconstitute forward-looking statements within the meaning of the Private Securities Litigation Reform Actof 1995. Any statements that are not statements of historical fact (including statements containing thewords "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would"and similar expressions) should also be considered to be forward-looking statements. There are anumber of important factors that could cause actual results or events to differ materially from thoseindicated by such forward-looking statements. More information about these and other risks that mayimpact Capricor's business is set forth in Capricor's Annual Report on Form 10-K for the year endedDecember 31, 2020 as filed with the Securities and Exchange Commission on March 15, 2021 and in ourQuarterly Report on Form 10-Q for the quarter ended March 31, 2021 as filed with the Securities andExchange Commission on May 14, 2021. All forward-looking statements in this press release are basedon information available to Capricor as of the date hereof, and Capricor assumes no obligation to updatethese 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
Corporate Summary
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NASDAQCAPR
Cash (3/31/21) 1
$41.9 million
Common shares1
22.9 million
Non-Dilutive Capital$45 million
External Collaborators US Army
US Department of DefenseStephen Gould, Ph.D.
Cedars-Sinai Medical Center
• Cell and exosome-based platform therapeutics company
• Two products with novel approaches to neuromuscular, infectious, inflammatory and cardiovascular diseases
• Late-stage clinical development in DMD and rapidly progressing program in COVID-19
• Expanding engineered exosome platform delivering RNA
• Over 100 publications from multiple institutions worldwide on both platforms with extensive in-vivo and clinical data
• Efficient use of capital including non-dilutive sources
• Experienced management team
1As reported in Form 10Q filed on May 14, 2021
Cell TherapyCardiosphere-derived cells (CAP-1002)
Exosomes PlatformEngineered Exosomes & CDC-Exosomes
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Capricor’s Product Pipeline
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CandidateTarget
IndicationsDevelopment Phase
StatusDiscovery Preclinical Phase I Phase II Phase III
CAP-1002(allogeneic CDCs)
Duchenne Muscular Dystrophy
Positive Phase II reported
CAP-1002(allogeneic CDCs) COVID-19 Actively recruiting
Exosome mRNA Vaccine
(Tripartite mRNA design)SARS-CoV-2 Planning IND filing
Engineered Exosomes(RNA delivery) Evaluating Platform
CDC-Exosomes(allogeneic CDC-XOs)
Duchenne Muscular Dystrophy
IND submitted
ASTEX-Exosomes(engineered fibroblast-
derived XOs)Evaluating Platform
Exosome VLP (Display Technology) Evaluating Platform
Capricor's exosomes technology has not yet been approved for clinical investigation.
Cell Therapy Exosome Platform
Exosome Platform Overview
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
RNA Therapeutics are a New Class of Medicines
Broad Market Opportunities
Rapid Innovation Possible
Barrier to Success: Effective Delivery Systems of Nucleic Acids
Capital Efficiency for Development
Proof of Concept Established in Vaccines
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosomes Are a Natural Drug Delivery System
• ~100 nanometer vesicles
• Made by nearly all cells
• Abundant in blood and all biofluids
• Transfers signals and molecules to other cells
• Decades of transfusion and transplantation medicine demonstrates safety
• Can be used to deliver RNAs and other drugs
Kidney International (2010) 78, 838–848
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Extracellular vesicles - term for cell-derived vesicles, including exosomes
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
From Discovery to Platform Development
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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
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 slow
3. Synthetic nanoparticles are untargeted delivery vehicles
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Exosomes:
‒ nature’s delivery vehicle
‒ low immunogenicity
‒ can deliver contents to the cell without integration
‒ can be targeted (tropism)
‒ can be lyophilized for ease of handling
Problems Possible Solutions
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosomes Platform GoalsBuilding a New Class of Therapeutics
Goals
OncologyVaccines & targeted delivery therapeutics
Infectious Diseases Vaccines
Inflammatory & Vascular Biologics
Monogenic Diseases RNA therapeutics
Drive research throughcollaborations
Expand and exploit platform and IP through partnerships
Scale and partner
Cell
Exosomes Nucleic Acids
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosome Platform Potential Vaccines and Therapeutics
Modalities
01 02 03 04Vaccines Therapeutics
Biologics Therapeutics RNA Delivery
TherapeuticsSmall Molecule Targeting
Targets
Infectious Diseases
(SARS-CoV-2)
Monogenic Diseases & Oncology
Inflammatory & Vascular Disorders
Evaluating
Exosomes: Platform for RNA & Drug
Delivery
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosomes Target Cargo Directly to the Cell
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‒ Exosomes serve as a natural signalingsystem
‒ CD-9, CD-81 and CD-63 are surfacemarkers of exosomes and can serve astargeting molecules for effective deliveryto cells and confirmation of exosomeloading
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
‒ Other mRNA vaccines in development are targeting the spike “S” protein solely
‒ Capricor’s exosome platform vaccine addresses multiple proteins
Coronavirus Structure Unique Protein Visualization
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
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 multiple proteins for better immune
response ‒ Exosomes address the delivery problem by targeting cells of interest
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4. Inject mRNA/exosome formulation (IM)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 15
Key findings:• Development of safe, non-toxic exosome
formulation capable of delivering functionalmRNA in vitro and in vivo
• Drives cell and antibody immunity tonucleocapsid protein (SARS-CoV-2)
• Drives cell and antibody immunity to spikeprotein (SARS-CoV-2)
• Confirms multiplexed mRNA approach• Unique antigen design
Exosome-mRNA vaccine demonstrates
long lasting cellular immunity
Results from bioRxiv, Gould 2021
Exosome-mRNA Vaccine Preclinical Results
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 16
Exosome-mRNA Vaccine Preclinical Results
Exosome-mRNA vaccine demonstrates long lasting humoral
immunity
Results from bioRxiv, Gould 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosome Platform Potential Vaccines and Therapeutics
Modalities
01 02 03 04Vaccines Therapeutics
Biologics Therapeutics RNA Delivery
TherapeuticsSmall Molecule Targeting
Targets
Infectious Diseases
(SARS-CoV-2)
Monogenic Diseases & Oncology
Inflammatory & Vascular Disorders
Evaluating
Exosomes: Platform for RNA & Drug
Delivery
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
ExosomesLess Toxic than Lipid Nanoparticles
Results from bioRxiv, Gould 2021
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Exosomes More Efficient mRNA Delivery
Control Exo-mRNA Antares2
Results from bioRxiv, Gould 2021
A real-time view of mRNA delivery, protein expression and enzymatic activity
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CAP-1002Cell Therapy Overview
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Capricor’s CAP-1002 TechnologyCAP-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
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Mechanism of Action:Defined in “Stem Cell Reports”
OxidativeStress
Fibrosis
Inflammation
Cellular Energy
Muscle CellGeneration
phospho-Akt Nrf2 (cytoplasmic) Nrf2 (nuclear)
HO-1
catalase SOD-2
GCLC cat. sub.
phospho-IkB p65 (nuclear)MCP1
CD68+ macrophages
CD3+ T cells
collagen I
collagen III
mitochondrial DNA copy number
level of respiratory chain subunits
RESTORED mitochondrial ultrastructureNORMALIZED deficient respiratory capacity
of isolated mitochondria
Ki67+ cardiomyocytes
Aurora B cardiomyocytes
NF-kB
*CDCs have been the subject of >100 peer-reviewed papers since 2007.Aminzadeh et al. Stem Cell Reports. 2018.
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Immunomodulatory Effects of CAP-1002
CDCs: Cardiosphere-derived cells
Macrophages
Effector T-cells
Proinflammatory Cytokines
IFN-γ, TNFα, IL-1β, IL-6, IL-8, CXCL10, CCL2, CCL3, CCL5
Systemic Inflammation
Multiorgan dysfunction
1. Cardiac inflammation2. Lung inflammation3. Cardiomyocyte death4. Cardiac dysfunction5. Skeletal muscle injury6. Tissue Fibrosis
1. Cardiomyogenesis2. Cardiomyocyte survival3. Anti-inflammatory4. Immunomodulatory5. Angiogenic6. Anti-fibrotic
CDCs: Mechanism of Action
1. Enhanced cell debris2. Decreased TNFα, IL-1β,
CCL5 production3. Increased levels of IL-10 by
macrophages
CDCs: Pro-inflammatory cellular targets
1. Myocardial ischemia (CADUCEUS, Phase I/II ALLSTAR, DYNAMIC Phase IIa)
2. Myocarditis3. Muscular dystrophy (HOPE-Duchenne, HOPE-2)4. Heart failure with preserved ejection fraction (REGRESS,
Phase I)5. Senescence 6. Non-ischemic dilated cardiomyopathy 7. Pulmonary arterial hypertension (ALPHA, Phase I)
CDCs: Efficacy (Pre-clinical and Clinical)
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Published https://link.springer.com/content/pdf/10.1007/s00395-020-0795-1.pdf
CAP-1002Duchenne MuscularDystrophy Program
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
DMD: 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 function properly, suffer progressive damage and eventually die
• Much of the muscle injury that occurs in dystrophin-deficiency is attributable to secondary damage caused by inflammation
Whole Muscle Tissue
Muscle-FiberMembrane
Dystrophin
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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*
• Hypothesis: CDCs to improve exercise capacity• Exercise performance approximately doubled vs. control
– P<0.005 at all timepoints through 12 weeks of follow-up*
• Hypothesis: CDCs to improve skeletal muscle function• Twitch force, tetanic force, and fibrosis in soleus (slow-twitch)
and extensor digitorum longus (fast-twitch) muscles significantly improved vs. control
– P<0.05; muscles isolated at three weeks post-treatment*
*Aminzadeh et al. Stem Cell Reports. 2018.
• Hypothesis: CDCs to treat diaphragm muscle• Fibrosis in the diaphragm markedly declined vs. control
– P<0.0001; muscles isolated at 3- and 12 months post-treatment
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Capricor’s Addressable DMD Population
CAPRICOR’sTargeted Patient
Population
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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 Skipping – treats a small portion of the DMD
population
Gene therapy – potential safety risks
NF-kB inhibition may not be enough
Steroids have adverse side-effects
Immunomodulatory
Anti-fibrotic
Pro-regenerative
Cellular Energy
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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 change at 12 months at all levels of ability*
*Mayhew et al, 2019; Pane et al, 2018.
Performance of the Upper Limb (PUL: v1.2) to Assess Skeletal Muscle
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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
• 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
GOAL OF FDA’S RMAT DESIGNATION To facilitate efficient development and expedite review of a drug
Rare Pediatric Disease Designation
OrphanDrug Designation
RMAT Designation
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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 corticosteroids
• Very limited options for this patient population
https://n.neurology.org/content/92/8/e866. Study funded with the support of CIRMhttps://clinicaltrials.gov/ct2/show/NCT02485938.
• 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
RESULTS
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
HOPE-Duchenne: Phase I/II Results 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
Scar
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HOPE-2 Phase II Clinical Study
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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-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.
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
PUL 1.2 (mid)
MONTH 12/ET
-8
-6
-4
-2
0 CAP-1002PLACEBO
p=0.0818
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
PUL 1.2 (mid)
MONTH 12/ET
CAP-1002
PLACEBO-15
-10
-5
0
5
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
p=0.0974(t test; two-tailed)
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improvement
Clinically Meaningful Changes in Mid-Level PUL 1.2 Similar changes shown in HOPE-Duchenne
Δ2.8 point difference in CAP-1002 vs. placebo at 12-months-Clinical meaningfulness assessed as 1 point change-
Topline data: 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
Topline Data
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
PUL 2.0 (full)
MONTH 12/ET
-5
-4
-3
-2
-1
0 CAP-1002PLACEBO
p=0.0532
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
PUL 2.0 (full)
MONTH 12/ET
CAP-1002
PLACEBO-8
-6
-4
-2
0
2
4
p=0.0201(t test; two-tailed)
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
Clinically Meaningful Changes Observed in PUL 2.0(Shoulder + Mid + Distal)
Topline data: 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
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improvement
Δ2.4 points in CAP-1002 vs. placebo at 12-months-Clinical meaningfulness assessed as 1 point change-
Topline Data
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 37
LV EF (%)
MONTH 12/ET
-3
-2
-1
0
1 CAP-1002PLACEBO
p=0.0042
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
improvement
LV ES Volume, Indexed, ml/m2
MONTH 12/ET
-4
-2
0
2
4CAP-1002PLACEBO
p=0.0122
Mea
n C
hang
e fro
m B
asel
ine
+/- S
EM
Has been used as a surrogate endpoint for approval in adult heart
failure
LV ED Volume, Indexed, ml/m2
MONTH 12/ET
-15
-10
-5
0
5 CAP-1002PLACEBO
p=0.0699M
ean
Cha
nge
from
Bas
elin
e +/
- SEM
improvement
improvement
Month 12-2
-1
0
1
2
3
4
Creatine Kinase MB/Total Creatine Kinase (%)Change From Baseline
Visit
% C
K-M
B
CAP-1002Placebo
✱✱✱
p=0.006
improvement
Enzyme associated with breakdown
of cardiac muscle cells
Cardiac Improvements ObservedEjection Fraction %, CK-MB, LV-ESV and LV-EDV
Topline data: 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
Topline Data
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
HOPE-2 Safety Results
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• 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
Conclusions and Future DirectionsConclusions:‒ First placebo-controlled trial
showing upper limb functional improvements in non-ambulant DMD patients
‒ Directionally consistent improvements in strength, respiratory and cardiac endpoints
‒ First ever study in DMD that correlates cardiac functional stabilization with reduction of a biomarker of myocardial cell damage
‒ Consistent results shown pre-clinically, Phase I/II and Phase II
Moving Forward:‒ FDA continues to encourage us to
conduct a Phase III study; we continue to discuss next steps and pathway to approval
‒ Engaged Lonza (global CMO) for scale-up of manufacturing of CAP-1002
‒ HOPE-2 Open label extension trial underway
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
World-Class DMD Advisory BoardCraig 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)
John Jefferies, M.D. Cincinnati Children's Hospital Medical Center (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)
Michael Taylor, M.D., Ph.D. Cincinnati Children's Hospital Medical Center (USA)
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
CAP-1002Cell Therapy Overview for
COVID-19
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
CAP-1002 Targets Severe Cases of COVID-19
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
CAP-1002: COVID-19 Program Compassionate Use Data
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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 supportImproved 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
CAP-1002: COVID-19 Program Phase II Trial Underway
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‒ 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‒ Trial actively recruiting subjects – topline data expected by Q3 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Targeted Milestones in 2021
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Cell Therapy (CAP-1002) Technology Plan to publish HOPE-2 final results Continue to work with FDA on next steps in pathway forward for DMD Continue to pursue partnership opportunities for DMD program Plan to complete enrollment in INSPIRE study Plan to announce top-line results from INSPIRE study
Engineered Exosomes Platform Technology Plan to publish preclinical data from exosomes technology Plan to file IND for exosome-mRNA vaccine Plan to announce pipeline expansion for engineered exosome program Continue to pursue grant funding activities Continue to pursue partnership opportunities
World-Class Facilities and Infrastructure
Capricor’s Research, Development and Manufacturing facilities are located in the Cedars-Sinai Medical Center in Los Angeles, California
Capricor has access to core research facilities
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Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
Senior Leadership TeamLinda 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 2010. Previous experience includes Excigen, Inc. where she was
responsible for business development and operations. Dr. Marbán began her career in academic science at the
Cleveland Clinic Foundation working on the biophysicalproperties of cardiac muscle and continued to a postdoctoralfellowship at Johns Hopkins University.
Dr. Marbán earned a Ph.D. from Case Western ReserveUniversity in cardiac physiology.
Karen Krasney, J.D.Executive Vice President & General Counsel Ms. Krasney’s has over 40 years of experience in domestic
and international corporate and business law, as well aslitigation.
Ms. Krasney served as legal counsel of BiosensorsInternational Group Ltd., a multinational medical devicecompany.
Ms. Krasney received her Bachelor of Arts degree from theUniversity of California, Los Angeles and her Juris Doctoratefrom the University of Southern California.
Stephen Gould, Ph.D.Executive Consultant Dr. Gould is a Professor of Biological Chemistry at Johns Hopkins
University and an internationally recognized exosome expert whobrings an unparalleled understanding of exosome engineering toCapricor.
Dr. Gould is co-Founder and acting President of the AmericanSociety for Exosomes and Microvesicles (ASEMV).
Dr. Gould’s team was the first to reveal the mechanistic linkbetween exosome biogenesis and virus budding, the first toidentify mechanisms of exosome engineering and the first todevelop an exosome-based cancer therapeutic.
Dr. Gould has published numerous research articles and severalbook chapters, received numerous public and private researchgrants and served on an array of NIH and other grant reviewpanels.
AJ Bergmann, M.B.A.Chief Financial Officer Mr. Bergmann has worked in the finance industry for over a
decade. Mr. Bergmann joined Capricor in 2011 and coordinated the
Company’s reverse merger, uplisting to NASDAQ and publicfinancings yielding over $85 million to date.
Mr. Bergmann graduated from Providence College and has aM.B.A. from the University of Southern California’s MarshallSchool of Business.
Sudhir Borgonha, M.D., M.B.A.Vice President of Clinical Development Dr. Borgonha previously served as Director of Translational
Medicine at the Fanconi Anemia Research Fund, where he ledefforts to propel a spectrum of approaches includinggene therapy to treat cancer in rare diseases. Prior to that, hewas the Medical Director of Strand Genomics, where he oversawdevelopment of new technologies such as the liquid biopsy andcustomized cancer panels, while he oversaw clinical reporting forover 5,000 patients a year.
Dr. Borgonha was a co-founder of Angstrom Medica (Acquired byPioneer Surgical), a biomaterials science company. He is alsoa co-founder of ten3T, a remote cardiac monitoring company.
Dr. Borgonha is a graduate of St. John’s MedicalCollege, Bangalore and the Sloan School of Managementat MIT.
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