: capr capricor therapeutics, inc. developing ...1.gene therapy using viral delivery (aav) ‒...

Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to BedsideNASDAQ: CAPR

Corporate & Investor Presentation December 2020

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 clinicaltrials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research andclinical trials; regulatory developments involving products, including the ability to obtain regulatoryapprovals or otherwise bring products to market; plans regarding current and future collaborativeactivities and the ownership of commercial rights; scope, duration, validity and enforceability ofintellectual property rights; future royalty streams, revenue projections; expectations with respect tothe expected use of proceeds from the recently completed offerings and the anticipated effects of theofferings, 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 PrivateSecurities 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 beforward-looking statements. There are a number of important factors that could cause actual resultsor events to differ materially from those indicated by such forward-looking statements. Moreinformation about these and other risks that may impact Capricor's business is set forth in Capricor'sAnnual Report on Form 10-K for the year ended December 31, 2019 as filed with the Securities andExchange Commission on March 27, 2020 and in our Quarterly Report on Form 10-Q for the quarterended 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 toCapricor as of the date hereof, and Capricor assumes no obligation to update these forward-lookingstatements.CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’sexosome-based candidates have been approved for clinical investigation.


Corporate Summary

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NASDAQTicker: CAPR

Cash (9/30/20) 1$35.3 million

Common shares120.4 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 of 11/13/20 as reported in Form 10Q

Cell TherapyCardiosphere-derived cells (CAP-1002)

Exosomes PlatformEngineered Exosomes & CDC-Exosomes


Capricor’s Product Pipeline

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CandidateTarget

IndicationsDevelopment Phase

StatusDiscovery Preclinical Phase I Phase II Phase IIICAP-1002

(allogeneic CDCs)Duchenne Muscular

Dystrophy Positive Phase II reported

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

Exosome mRNA VaccineTripartite mRNA design SARS-CoV-2 In development

Exosome VLP Display Vaccine

4-part antigen designSARS-CoV-2 In development

CDC-Exosomes(allogeneic CDC-XOs)

Duchenne Muscular Dystrophy IND submitted

Engineered Exosomes(RNA delivery) Evaluating Platform

ASTEX-Exosomes(engineered fibroblast-

derived XOs)Evaluating Platform

Capricor's exosomes technology has not yet been approved for clinical investigation.

Cell Therapy Exosome Platform


Exosome Platform Overview


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 technologyoriginating from cardiosphere-derived cells (CDCs)

Kidney International (2010) 78, 838–848

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From Discovery to Platform Development

7

Publications covering our

technology have been published by

us or our collaborators in multiple peer-

reviewed journals.


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


Exosomes: Building a New Class of Therapeutics

9

Infectious Diseases

mRNA and VLP vaccine

approaches

OncologyVaccines &

targeted delivery

therapeutics

Monogenic Diseases

mRNA therapeutics

Neuromuscular & Inflammatory

Protein or mRNA

therapeutics

Ongoing Collaborations• Stephen Gould, Ph.D.• US Army Institute for Surgical Research• US Department of Defense• Cedars-Sinai Medical Center

Goals• Drive innovation and research through

collaborations• Scale and partner• Expand and exploit platform and IP

through partnerships

Engineered Exosome Platform

mRNA and VLP Exosome-based Vaccines

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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


‒ 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

Coronavirus Structure Unique Protein Visualization

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4 antigens likely to yield increased protective response

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


Capricor’s Exosome-Based Vaccines Approach is Distinctive

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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


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

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Exosome-mRNA Vaccine Preclinical Results

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Key findings:• Development of safe, non-toxic exosome

formulation capable of delivering functionalmRNA in vitro and in vivo

• Creation of a multiplexed exosome-RNA vaccinethat expresses viral antigens engineered toinduce cellular immunity and antibody responsesto multiple proteins of SARS-CoV-2

• Validation that an exosome mRNA vaccine caninduce:• 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 Sproteins

• Following a low-dose immunization protocol, noevidence of vaccine-induced adverse events

Results from bioRxiv, Gould Nov. 2020

Exosome-mRNA vaccine demonstrates

long lasting cellular immunity

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 Nov. 2020


Exosome VLP: Display Vaccine Approach*

*Makes use of highly optimized expression system developed by the Gould lab

17

Capricor’s exosome platform vaccine addresses

all 4 proteins: N,S,M,E

CAP-1002Cell Therapy Overview


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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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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CAP-1002Duchenne MuscularDystrophy Program

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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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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’sTargeted Patient

Population

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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 populationGene therapy – potential safety

risksNF-kB inhibition may not be

enoughSteroids have adverse side-

effects

Immunomodulatory

Anti-fibrotic

Pro-regenerative

Cellular Energy

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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’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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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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https://n.neurology.org/content/92/8/e866


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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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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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-1

002

PLAC

EBO

-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)

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

32

improvement

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

Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 33

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

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


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


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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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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CAP-1002Cell Therapy Overview for COVID-19

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CAP-1002 Targets Severe Cases of COVID-19

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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

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


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

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


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 (severe to critical) ‒ Patient participation will be a maximum of 13 weeks from screening‒ Trial actively recruiting subjects


Targeted Milestones into 2021

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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 programEngineered 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

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

43


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 Reserve Universityin 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 as litigation. Ms. Krasney served as legal counsel of Biosensors International

Group Ltd., a multinational medical device company. Ms. Krasney received her Bachelor of Arts degree from the

University of California, Los Angeles and her Juris Doctorate fromthe 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 expertwho brings an unparalleled understanding of exosomeengineering to Capricor.

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.

44

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 heled efforts to propel a spectrum of approaches includinggene therapy to treat cancer in rare diseases. Prior to that,he was the Medical Director of Strand Genomics, where heoversaw development of new technologies such as theliquid biopsy and customized cancer panels, while heoversaw clinical reporting for over 5,000 patients a year.

Dr. Borgonha was a co-founder of Angstrom Medica(Acquired by Pioneer Surgical), a biomaterials sciencecompany. He is also a co-founder of ten3T, aremote cardiac monitoring company.

Dr. Borgonha is a graduate of St. John’s MedicalCollege, Bangalore and the Sloan School of Managementat MIT.

Slide Number 1Forward-Looking StatementsCorporate Summary Capricor’s Product PipelineSlide Number 5Exosomes Platform �Potential New Class of Therapeutics From Discovery to Platform Development Capricor’s Potential Solutions to Complex ProblemsExosomes: Building a New Class �of Therapeutics Engineered Exosome Platform ��mRNA and VLP �Exosome-based Vaccines � Exosomes Target Cargo Directly to the Cell�Slide Number 12Capricor’s Exosome-Based Vaccines �Approach is DistinctiveExosome mRNA VaccineExosome-mRNA Vaccine Preclinical Results Slide Number 16Exosome VLP: Display Vaccine Approach* �Slide Number 18Capricor’s CAP-1002 TechnologyMechanism of Action:�Defined in “Stem Cell Reports”Slide Number 21DMD: Lack of Dystrophin Predisposes Muscle to DamageTrajectory of CDCs in DMD (Preclinical Data) Capricor’s Addressable DMD Population Competitive Landscape for DMD Primary Efficacy EndpointCapricor’s Regulatory Designations - DMDHOPE-Duchenne Focused on Older DMD PatientsHOPE-Duchenne: Phase I/II Results �Reduced Cardiac Scar and Improved PULSlide Number 30HOPE-2 Clinical TrialSlide Number 32Slide Number 33HOPE-2 Safety Results Conclusions and Future DirectionsWorld-Class DMD Advisory BoardSlide Number 37CAP-1002 Targets Severe Cases of COVID-19�Immunomodulatory Effects of CAP-1002�CAP-1002: COVID-19 Program �Compassionate Use Data CAP-1002: COVID-19 Program �Phase II Trial Underway Targeted Milestones into 2021World-Class Facilities and Infrastructure Slide Number 44

: capr capricor therapeutics, inc. developing ...1.gene therapy using viral delivery (aav) ‒...

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