CORPORATE & INVESTOR PRESENTATION - NASDAQ: CAPR - Capricor Therapeutics
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CORPORATE
& INVESTOR
PRESENTATION
NASDAQ: CAPR
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; the ability to achieve product milestones and to receive milestone payments from commercial partners; 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, 2021 as filed with the Securities and Exchange Commission on March 11, 2022 and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as filed with the Securities and Exchange Commission on May 11, 2022. 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
Capricor Therapeutics
Company Highlights NASDAQ: CAPR
Cell and Exosome-Based Platform Therapeutics Company
Commencing Phase 3 in Duchenne Muscular Dystrophy
• Strategic partnership: $705 million in potential milestones
• Market potential over $1 billion annual sales
• Positive phase 2 data published in The Lancet
Exosomes Platform Expansion
• Natural intracellular delivery technology for RNA and proteins
Broad Scientific and Intellectual Property Profile Headquarters: San Diego, California
• Over 100 publications from multiple institutions worldwide NASDAQ: CAPR
Founded: 2006, public in 2013
• 100 patents and patent applications Outstanding Shares: 24.3M
Strong External Collaborations Employees: ~50
• US Army, US Department of Defense, Stephen Gould, Ph.D. Laboratory at Johns Hopkins
University, Cedars-Sinai Medical Center and Nippon Shinyaku, Co.
Cash Runway for over 2 Years
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3
Capricor’s Recent Achievments
Cell Therapy Program in Duchenne Muscular Dystrophy
• Entered exclusive partnership with Nippon Shinyaku, Co. for U.S. distribution rights
• Received $30 million, additional $705 million in potential milestones plus revenue share
• Phase 2 data published in The Lancet
• Phase 3 pivotal study cleared by FDA to proceed
• Orphan Drug, RMAT and Rare Pediatric Disease Designations
Engineered Exosomes Platform Expansion
• Signed exclusive, worldwide licensing agreement with Johns Hopkins University for vaccines
and therapeutics
• Published new advances in exosome mRNA delivery
Relocation and Expansion of Senior Leadership Team
• Relocated headquarters to San Diego, California
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 4
Capricor’s Product Pipeline
Target Development Phase
Candidate Indications Discovery Preclinical Phase I Phase II Phase III Status
CAP-1002 Duchenne Muscular
Phase III initiating Q2 2022
(allogeneic CDCs) Dystrophy
Exosome-Based Vaccine
Infectious diseases Preclinical
(Multivalent design)
Engineered Exosomes
Undisclosed Discovery
(RNA and protein delivery)
CDC-Exosomes Duchenne Muscular
IND submitted
(allogeneic CDC-XOs) Dystrophy
Cell Therapy Exosome Platform
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 5
Capricor’s CAP-1002 Cell Therapy Technology Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 6
Capricor’s Cell Therapy Technology
CAP-1002
CAP-1002: biologic consisting Manufactured
of allogeneic cardiosphere- from donated heart muscle
derived cells (CDCs)
Does not act by «stemness»
Has been investigated in the cells do not engraft into host tissue
multiple independent clinical
trials and approximately Mechanism: cells secrete exosomes:
200 human subjects to date • 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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 7
CAP-1002 Mechanism of Action: Defined
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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Aminzadeh, et al. Stem Cell Reports. 2018. 8
CAP-1002
Supporting the Mechanism of Action
Purity: miR-#1 Identity: miR-#2 Potency miR-#3
Non-Potent Potent Non-Potent Potent
MSC-EXO CDC-EXO MSC-XO CDC-EXO Non-Potent Potent
CAP-1002’s purity, identity and potency can be defined by specific miRNAs
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 9
Duchenne Muscular Dystrophy CAP-1002 Cell Therapy Technology Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 10
Duchenne Muscular Dystrophy
Market Opportunity
Duchenne Muscular Dystrophy: the most severe form of muscular dystrophy
• X-linked genetic disorder
• Unable to produce dystrophin: essential protein for muscle formation and
growth
• Deficiency leads to loss of ambulation; severe cardiac and respiratory symptoms
• Approx. 20,000 cases in United States, globally 1 in 3,500 male births
Unmet Medical Need
• No known cure; disease is fatal
• Present standard of care involves corticosteroids
• Very few options in development for non-ambulant patients
CAP-1002 aims to attenuate skeletal and cardiac muscle damage
• Clinical data demonstrates improvement in upper limb and cardiac function in
two clinical trials
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 11Duchenne Muscular Dystrophy
Lack of Dystrophin Predisposes Muscle to Damage
Dystrophin is a structural protein
located within the muscle fiber membrane
Acts both as a cushion and glue
Without dystrophin, muscles are unable to
Dystrophin
function properly, suffer progressive
damage and eventually die
Whole
Muscle
Tissue Muscle-
Fiber
Much of the muscle injury that occurs
Membrane in dystrophin-deficiency is attributable to
secondary damage caused by inflammation
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 12Capricor’s Addressable DMD Population
Capricor’s Targeted
Patient Population
Estimated that over 50%
of DMD patients in U.S.
are non-ambulant
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 13Trajectory of CAP-1002 in DMD
Preclinical Data
HYPOTHESES
CDCs to treat CDCs to improve CDCs to improve CDCs to treat
cardiomyopathy exercise capacity skeletal muscle diaphragm
Left ventricular ejection Exercise performance function muscle
fraction markedly approximately doubled Twitch force, tetanic force, and Fibrosis in the diaphragm
improved vs. control vs. control fibrosis in soleus markedly declined
(slow-twitch) and vs. control
extensor digitorum
longus (fast-twitch) muscles
significantly improved vs.
control
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Aminzadeh, et al. Stem Cell Reports. 2018. 14Competitive Landscape for DMD
01 02 03
Exon Gene Steroids
Options Skipping therapy
01 02 03 CAP-1002
Benefits
Exon Skipping Gene therapy Steroids
Challenges treats a small potential have adverse
portion of the safety risks side-effects
DMD population • Immunomodulatory
• Anti-fibrotic
CAP-1002 • Pro-regenerative
has significant data supporting • Cellular Energy
use as a monotherapy and can
We believe be used synergistically with
other therapies to treat DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 15Performance of Upper Limb (PUL Test)
To Assess Skeletal Muscle Function
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*
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside *Mayhew et al, 2019; Pane et al, 2018. 16Capricor’s Regulatory Designations
Duchenne Muscular Dystrophy
Products may also be eligible for
accelerated approval
• RMAT provides benefits that include more frequent 01
meetings with FDA to discuss the development plan
for the product candidate
RMAT
GOAL OF FDA’S • Eligibility for rolling review and priority review
Designation
RMAT
02
Rare Pediatric
DESIGNATION Similar to breakthrough
therapy designation:
Disease
Designation
To facilitate efficient • On the basis of a surrogate or intermediate endpoint 03
development and expedite reasonably likely to predict long-term clinical benefit
review of a drug Orphan
• Reliance upon data obtained from a meaningful
number of sites Drug
Designation
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 17HOPE-Duchenne
Phase I/II Overview
• Phase I/II study: 25 patients, randomized and open-label
• One-time, multi-vessel, intracoronary delivery of 75M 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
Study funded with the support of CIRM
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in Neurology, 2019. 18HOPE-Duchenne Results
Reduced Cardiac Scar and Improved PUL
Scar Size Mid+distal PUL
Scar
R.G. Victor et al., AHA LBCT 2017; M. Taylor et al., submitted
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in Neurology, 2019. 19HOPE-2 Phase II Design
• 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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 20HOPE-2 Phase 2 Results Published in The Lancet, March 2022 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 21
HOPE-2 Upper Limb Improvements
Primary Efficacy Endpoint: PUL 1.2 Mid Level
Mid-level PUL 1.2
p=0.01
improvement
71% slowing of disease (PUL) Δ2.6 points in CAP-1002
vs. placebo at 12-months
Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as
dependent variable and percentile ranked baseline score, treatment, visit, treatment-by-visit interaction, PUL entry-item
score at randomization, and site as model effects. Adjusted model outcomes are report as least-squares means (LS-Mean).
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 22HOPE-2 Upper Limb Improvements
PUL 2.0 Full Full PUL 2.0
p=0.04
improvement
Δ1.8 points in CAP-1002
vs. placebo at 12-months
Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as
dependent variable and percentile ranked baseline score, treatment, visit, treatment-by-visit interaction, PUL entry-item
score at randomization, and site as model effects. Adjusted model outcomes are report as least-squares means (LS-Mean).
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 23HOPE-2 Cardiac Improvements
Ejection Fraction and CKMB
LV Ejection Fraction Creatine Kinase MB/ Total Creatine Kinase (%)
improvement
p=0.002 p=0.02
Δ=4% in LVEF by cardiac MRI Biomarker to assess cardiac muscle damage
Mixed Model for Repeated Measures (MMRM) analysis was performed using percentile ranked change from baseline as
dependent variable and percentile ranked baseline score, treatment, visit, treatment-by-visit interaction, PUL entry-item
score at randomization, and site as model effects. Adjusted model outcomes are report as least-squares means (LS-Mean).
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 24HOPE-2 Safety Results
69total infusions
Generally safe
and well tolerated throughout the study
With the exception of three
were performed in HOPE-2 hypersensitivity reactions;
no safety signals were identified
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 25CAP-1002 Proteomic Biomarkers in DMD
A B Treated vs.
C
Placebo (mons)
Blood Protein Detection Function and Canonical pathways 3 6
component conc. method Binding of mononuclear leukocytes
Cell movement of natural killer cells
Mobilization of blood cells
Resident
Mitra blood device LC-MS
Dual Plasma LC-MS
Attraction of T-lymphocytes
blood
Migration of macrophages
proteins
Adhesion of mononuclear leukocytes Reduced
SAA
Recruitment of mononuclear leukocytes Reduced glucose trafficking of
Systemic VWF metabolism leukocytes
response CRP Recruitment of T-lymphocytes
dysfunction
Cell death of lympatic system cells Inhibition of
factors/ neutrophils
ICAM/ LXR/RXR activation
Tissue Reduced damageto
Trem1 signaling in neutrophils + monocytes
Cytokine ELISA
VCAM
leakage cartilage&bone Infection
TGFB1 HMGB1 signaling from monocytes +
macrophages Reduced
proinflammatory Synthesis of
TH2 pathway Decrease
IL-6 prostaglandin E2
Cytokines IL23 pathway response
functionof Reduced Activation of
Senescence pathways leukocytes recruitmentof dendriticcells
IL8 pathway neutrophils
Acute phase response
NF-kB signaling
Panel A: Complimentary approaches required to analyze blood-based markers depending on
their concentration range
Panel B: CAP-1002 treatment alters inflammatory and immune pathways in DMD
Panel C: Dynamic proteomic and cytokine changes induced by CAP-1002 treatment are linked to
a series of downstream end-effects in DMD
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results published in The Lancet, March 2022. 26Phase III Pivotal Trial
Initiating in Q2 2022
• HOPE-3: Phase III, randomized, double-blind, placebo-controlled trial
to be conducted in the United States
• Target Enrollment: 70 patients
• Number of estimated sites: 20-30
• Patient Population: Non-ambulant DMD patients
• Primary Endpoint: PUL 2.0 at 12 months
• Secondary Endpoints: Cardiac, QOL, clinical status
• Initiation and Patient Screening: underway
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 27Partnership with Nippon Shinyaku Exclusive Distribution and Commercialization Commercial Rights: CAP-1002 for the treatment of DMD • US rights only • Nippon to appoint NS Pharma, its US subsidiary, as exclusive sales and distribution partner Deal Valued at up to $735M • $30M upfront • Up to $705M in additional development and sales milestones Meaningful double-digit revenue share of product revenue to Capricor Capricor responsible for the conduct of the Phase 3 trial and manufacturing of CAP-1002 Nippon Shinyaku recently launched Viltepso, an exon skipping agent for the treatment of DMD and has a fully assembled U.S. team to support a broad commercialization effort Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 28
World-Class Duchenne Advisory Board
Craig McDonald,
Pat Furlong Kan Hor, M.D. Thomas Voit, M.D.
M.D. (National PI)
Parent Project Muscular Nationwide Children's Hospital University College
University of California Dystrophy (USA) (USA) London (UK)
at Davis (USA)
Michelle Eagle, Oscar Henry Mayer, Eugenio Mercuri, Francesco Muntoni,
Ph.D., M.Sc., MCSP M.D. M.D., Ph.D. M.D.
Atom International Children's Hospital Catholic University of University College
Ltd (UK) of Philadelphia (USA) the Sacred Heart (Italy) London (UK)
Michael Taylor,
Richard Finkel, M.D. John Jefferies, M.D. Lee Sweeney, Ph.D.
M.D., Ph.D.
Nemours Children’s Cincinnati Children's Hospital University of Florida
Hospital (USA) Medical Center (USA) (USA) Cincinnati Children's Hospital
Medical Center (USA)
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 29Capricor’s Exosome Platform Therapeutics and Vaccines Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 30
RNA Therapeutics
A New Class of Medicines
Broad Market Opportunities
Capital Efficiency for Development
Rapid Innovation Possible
Proof of Concept Established in Vaccines
Barrier to Success:
Effective Delivery Systems of Nucleic Acids
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 31Exosomes are Nature’s Delivery Tool
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, proteins and
other drugs
Kidney International (2010) 78, 838–848
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Extracellular vesicles - term for cell-derived vesicles, including exosomes 32Exosomes Platform Development Tools
Production Loading Targeting Delivery
• Scalable production • Evaluate loading • Unmodified for local • Exosome targeting,
processes in requirements and size injection cargo delivery, dosing,
development constraints • Cell-specific targeting safety, distribution
• Extensive • mRNA, siRNA, small for systemic injection • In vitro and in vivo
characterization of cell molecule and protein • Potential to target efficacy
line cargos muscle, brain, etc.
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 33Exosomes Potential Solutions
to Drug Delivery Challenges
Problems Possible Solutions
• Gene therapy using viral delivery (AAV) Exosomes:
• Immune response • low immunogenicity
• Delivery of RNAs
• can deliver contents to the cell
• Uptake to render biologic relevance
without integration
• Therapeutic development slow
• Synthetic nanoparticles are untargeted • can be targeted (tropism)
delivery vehicles • can be lyophilized for ease of
handling
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 34Targeted Exosome Platform
Allows for Broad Applicability
SURFACE-MODIFIED EXOSOMES
Specific Tropism
• Modify exosome membrane
• Targeted delivery
CDC-EXOSOMES
Regenerative Medicine 2.0
• Immunomodulation
• Tissue regeneration
ENGINEERED EXOSOMES
Loaded Exosomes
• Exosomes loaded with therapeutic
cargo
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 35Exosomes as a Delivery Vehicle Vaccine: Proof of Concept Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 36
Novel Exosome-Based Vaccine Platform
Display and mRNA Technologies
Exosome-based approach
• Natural intracellular delivery particle
• Allows for specificity in cellular targeting
• Innovative technology, yet is well characterized
Unique multi-antigen approach
• Potentially confers greater immunity by targeting at least S and N proteins
Exosomes potentially superior to lipid nanoparticles
• Low immunogenicity
• Deliver payload directly to cytoplasm
• Target different immune cell populations to modulate their activity for
therapeutic purposes
Two differentiated vaccine approaches under development
• Display and mRNA approaches
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 37Coronavirus Structure
Unique Protein Visualization
Spike Protein
generates antibody response* Other
vaccines
Spike in development are targeting
the spike “S” protein solely
(S)
Capricor’s exosome
vaccine
Addresses both “S” and “N”
proteins
Nucleocapsid
(N)
Unique vaccine
approach utilizing
exosomes as delivery vehcicle
Nucleocapsid Protein
RNA generates T-cell response*
viral genome
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside *Results from Journal of Biological Chemistry, Gould, 2021. 38Exosome: Display Vaccine Approach
Broad Potential Use in Infectious and Other Diseases
01 02 03 04 05
Create vectors Transfect Grow the cells, Purify the exosomes: Immunize
that express into 293 cells which produce which carry the S and N with purified VLPs,
the SARS-CoV-2 SARS-CoV-2 VLPs proteins in their native, including protective
structural proteins such authentic, biochemical/cell immunity to SARS-
as S and N biological context Cov-2
and conformation
Capricor’s
exosome display
vaccine addresses
N
multiple proteins
of SARS-CoV-2
S
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 39Exosome mRNA Vaccine
Preclinical Results: Cellular and Humoral Response
Study key findings:
• Drives cell and antibody immunity to nucleocapsid protein (SARS-CoV-2)
• Drives cell and antibody immunity to spike protein (SARS-CoV-2)
• Confirms multiplexed mRNA approach
• Unique antigen design
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside Results from Journal of Biological Chemistry, Gould, 2021. 40Exosomes Platform Goals
Building a New Class of Medicines
Monogenic Diseases
RNA and protein therapeutics
Oncology
Vaccines and targeted delivery
therapeutics
Inflammatory & Vascular
Biologics
Exosomes Drug Payload Infectious Diseases
Vaccines
Cell
Drive research Expand and exploit
Scale
Goals and partner
through platform and IP through
collaborations partnerships
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 41Recent and Targeted Milestones
Cash Runway
CAP-1002 for Duchenne Muscular Dystrophy
Reported 1-year positive results from HOPE-2 at World Muscle Society
Announced FDA acceptance of Phase 3 trial protocol and design
Announced partnership with Nippon Shinyaku for US distribution rights
Published Phase 2 results in The Lancet
Phase III initiation of enrollment Q2 2022
Pursue additional partnerships outside U.S. 2022-2023
Exosomes Platform
Entered exclusive worldwide licensing agreement with Johns Hopkins University for
engineered exosome platform of vaccines and therapeutics
Published positive preclinical data using multivalent exosome vaccine
Published preclinical data in conjunction with US Army for trauma and shock
Publish new preclinical data on platform expansion 2022
Announce further pipeline expansion and indications 2022-2023
Cash Position1
$58.3 million as of 3/31/22, cash runway for at least 2 years
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 1As reported in Form 10Q filed on 5/11/22 42Senior Leadership Team
Linda Marbán, Ph.D. AJ Bergmann, M.B.A. Karen Krasney, J.D. Daniel Paulson, M.D. Kristi Elliott, Ph.D. Yasmine Shad Stephen Gould, Ph.D.
Chief Executive Officer, Chief Financial Officer Executive Vice President Vice President of Vice President of R&D Vice President of Quality Executive Consultant
Co-founder & Director • & General Counsel Clinical Development • Dr. Gould is a Professor of
• Dr. Elliott oversees
• • • Capricor’s exosome platform • Ms. Shad oversees all facets of Biological Chemistry at Johns
and pipeline development. Quality Assurance and Quality Hopkins University
Control for Capricor’s product and an internationally
• She has approximately 15 pipeline. recognized exosome expert
• •
years of experience in who brings an unparalleled
biotechnology • She has approximately 20 years understanding of
• • of experience in the quality exosome engineering
• • As an innovator in the assurance field of
exosome field, Dr. Elliott has biotechnology • Dr. Gould is co-Founder and
been involved in the acting President of the
conception and • Ms. Shad previously held the American Society
implementation of exosome position of Executive Director, for Exosomes and Microvesicles
•
• purification, scale-up, Clinical Quality, Site Head for
• Kite Pharma, a Gilead Company, • Dr. Gould’s team was the first
loading and targeting
• processes for multiple where she led clinical trial stage to reveal the mechanistic link
exosome-based product quality functions including but between exosome biogenesis
candidates. not limited to quality systems, and virus budding, the first to
quality operations, quality identify mechanisms of
• Dr. Elliott received her B.A. engineering, LIMS, QC exosome engineering and the
•
in biology and M.S. in analytical, QC microbiology, and first to develop an exosome-
• molecular biology from sample management. based cancer therapeutic
Rutgers University. She
• earned her Ph.D. in human • Ms. Shad received her B.S. and • Dr. Gould has published
genetics and molecular M.S. in Biochemistry from numerous research articles and
biology from The Johns Laurentian University and the several book chapters, received
Hopkins School of Medicine. University of Guelph in Ontario, numerous public and private
Canada, and later went on to research grants
obtain an additional M.S. in
Regulatory Affairs from San
Diego State University.
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