NASDAQ: CAPR Corporate & Investor Presentation - Capricor Therapeutics, Inc.
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Corporate & Investor Presentation June 2021 NASDAQ: CAPR Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside
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, 2020 as filed with the Securities and Exchange Commission on March 15, 2021 and in our
Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 as filed with the Securities and
Exchange Commission on May 14, 2021. 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
Corporate Summary
• Cell and exosome-based platform therapeutics company
NASDAQ
• Two products with novel approaches to neuromuscular, CAPR
infectious, inflammatory and cardiovascular diseases
Cash (3/31/21) 1
Cell Therapy Exosomes Platform $41.9 million
Cardiosphere-derived cells (CAP-1002) Engineered Exosomes & CDC-Exosomes
Common shares1
22.9 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 US Army
both 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 reported in Form 10Q filed on May 14, 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 3
Capricor’s Product Pipeline
Target Development Phase
Candidate Indications Discovery Preclinical Phase I Phase II Phase III Status
Duchenne
CAP-1002
Muscular Positive Phase II reported
(allogeneic CDCs)
Dystrophy
CAP-1002
COVID-19 Actively recruiting
(allogeneic CDCs)
Exosome mRNA
Vaccine SARS-CoV-2 Planning IND filing
(Tripartite mRNA design)
Engineered Exosomes
Evaluating Platform
(RNA delivery)
Duchenne
CDC-Exosomes
Muscular IND submitted
(allogeneic CDC-XOs)
Dystrophy
ASTEX-Exosomes
(engineered fibroblast- Evaluating Platform
derived XOs)
Exosome VLP
Evaluating Platform
(Display Technology)
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
Exosome Platform Overview
RNA Therapeutics are 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 6
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
Extracellular vesicles - term for cell-derived vesicles, including exosomes Kidney International (2010) 78, 838–848
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 7
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 8
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 ‒ can deliver contents to the cell
relevance without integration
‒ Therapeutic development slow ‒ can be targeted (tropism)
3. Synthetic nanoparticles are
‒ can be lyophilized for ease of
untargeted delivery vehicles
handling
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 9
Exosomes Platform Goals
Building a New Class of Therapeutics
Monogenic Diseases
RNA therapeutics
Oncology
Vaccines & targeted delivery
therapeutics
Inflammatory & Vascular
Biologics
Exosomes Nucleic Acids Infectious Diseases
Vaccines
Cell
Drive research Expand and exploit
Scale
Goals and partner
through platform and IP
collaborations through partnerships
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 10Exosome Platform
Potential Vaccines and Therapeutics
01 02 03 04
Therapeutics Therapeutics Therapeutics
Vaccines
RNA Delivery Biologics Small Molecule
Modalities Targeting
Exosomes:
Platform for
RNA & Drug
Infectious Monogenic Inflammatory Evaluating
Delivery Diseases Diseases & & Vascular
(SARS-CoV-2) Oncology Disorders
Targets
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 11Exosomes 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 12Coronavirus Structure
Unique Protein Visualization
‒ Other mRNA vaccines in
development are targeting
the spike “S” protein solely
‒ Capricor’s exosome
platform vaccine addresses
multiple proteins
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 13Exosome mRNA Vaccine
4. Inject
mRNA/exosome
formulation (IM)
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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 14Exosome-mRNA Vaccine
Preclinical Results
Key findings:
• Development of safe, non-toxic exosome
formulation capable of delivering functional
mRNA in vitro and in vivo
• 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
Exosome-mRNA vaccine
demonstrates
long lasting cellular
immunity
Results from bioRxiv, Gould 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 15Exosome-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 16Exosome Platform
Potential Vaccines and Therapeutics
01 02 03 04
Therapeutics Therapeutics Therapeutics
Vaccines
RNA Delivery Biologics Small Molecule
Modalities Targeting
Exosomes:
Platform for
RNA & Drug
Infectious Monogenic Inflammatory Evaluating
Delivery Diseases Diseases & & Vascular
(SARS-CoV-2) Oncology Disorders
Targets
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 17Exosomes
Less Toxic than Lipid Nanoparticles
Results from bioRxiv, Gould 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 18Exosomes
More Efficient mRNA Delivery
A real-time view of mRNA delivery,
protein expression and enzymatic activity
Control Exo-mRNA Antares2
Results from bioRxiv, Gould 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 19CAP-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 21Mechanism 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 22Immunomodulatory 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, 2. Myocarditis
4. Immunomodulatory CCL5 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 23CAP-1002
Duchenne
Muscular
Dystrophy
Program
24DMD: 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 Muscle-Fiber
and 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 25Trajectory of CDCs in DMD (Preclinical Data)
• Hypothesis: CDCs to treat cardiomyopathy
• Left ventricular ejection fraction markedly improved vs. control
– PCapricor’s Addressable DMD Population
CAPRICOR’s
Targeted
Patient
Population
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 27Competitive Landscape for DMD
Options Challenges CAP-1002 Benefits
Exon Skipping – treats a small
portion of the DMD Immunomodulatory
Exon Skipping population
Anti-fibrotic
Gene therapy Gene therapy – potential
safety risks Pro-regenerative
NF-kB
NF-kB inhibition may not be Cellular Energy
Steroids enough
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 28Primary 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 29Capricor’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 30HOPE-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 31HOPE-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 32HOPE-2
Phase II Clinical
Study
33HOPE-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 34Clinically Meaningful Changes in Mid-Level PUL 1.2
Similar changes shown in HOPE-Duchenne
Topline Data
PUL 1.2 (mid) PUL 1.2 (mid)
MONTH 12/ET MONTH 12/ET
Mean Change from Baseline +/- SEM
Mean Change from Baseline +/- SEM
CAP-1002 5
0
PLACEBO
0
-2
improvement -5
-4
-10
-6 p=0.0974
(t test; two-tailed)
-15
p=0.0818
-8
02
O
EB
10
C
P-
A
Δ2.8 point difference in CAP-1002 vs. placebo at 12-months
A
PL
C
-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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 35Clinically Meaningful Changes Observed in PUL 2.0
(Shoulder + Mid + Distal)
Topline Data
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
-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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 36Cardiac Improvements Observed
Ejection Fraction %, CK-MB, LV-ESV and LV-EDV
LV EF (%) LV ES Volume, Indexed, ml/m2
Topline Data
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
Topline data: Comparisons treated vs. placebo using mixed model repeated measures
Visit 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 37HOPE-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 38Conclusions 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
improvements in non-ambulant continue to discuss next steps and
DMD patients pathway to approval
‒ Directionally consistent ‒ Engaged Lonza (global CMO) for
improvements in strength, scale-up of manufacturing of CAP-
respiratory and cardiac endpoints 1002
‒ First ever study in DMD that ‒ HOPE-2 Open label extension trial
correlates cardiac functional underway
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 39World-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 40CAP-1002
Cell Therapy
Overview for
COVID-19
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to BedsideCAP-1002 Targets Severe Cases of COVID-19 Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 42
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 43CAP-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
‒ Trial actively recruiting subjects – topline data expected by Q3 2021
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 44Targeted Milestones in 2021
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
Capricor Therapeutics, Inc. Developing Transformative Therapies from Bench to Bedside 45World-Class Capricor’s Research, Development and Manufacturing facilities are
Facilities and located in the Cedars-Sinai Medical Center in Los Angeles,
California
Infrastructure Capricor has access to core research facilities
46Senior Leadership Team
Stephen Gould, Ph.D.
Linda Marbán, Ph.D. Executive Consultant
Chief Executive Officer, Co-founder and Director Dr. Gould is a Professor of Biological Chemistry at Johns Hopkins
Dr. Marbán has over 25 years of experience in the University and an internationally recognized exosome expert who
biotechnology industry brings an unparalleled understanding of exosome engineering to
Been with Capricor since 2005 and CEO since 2010. 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. Gould’s team was the first to reveal the mechanistic link
Dr. Marbán began her career in academic science at the
between exosome biogenesis and virus budding, the first to
Cleveland Clinic Foundation working on the biophysical
identify mechanisms of exosome engineering and the first to
properties of cardiac muscle and continued to a postdoctoral develop an exosome-based cancer therapeutic.
fellowship at Johns Hopkins University. Dr. Gould has published numerous research articles and several
Dr. Marbán earned a Ph.D. from Case Western Reserve book chapters, received numerous public and private research
University in cardiac physiology. grants and served on an array of NIH and other grant review
panels.
Karen Krasney, J.D.
Executive Vice President & General Counsel Sudhir Borgonha, M.D., M.B.A.
Ms. Krasney’s has over 40 years of experience in domestic Vice President of Clinical Development
and international corporate and business law, as well as Dr. Borgonha previously served as Director of Translational
litigation. Medicine at the Fanconi Anemia Research Fund, where he led
Ms. Krasney served as legal counsel of Biosensors efforts to propel a spectrum of approaches including
International Group Ltd., a multinational medical device gene therapy to treat cancer in rare diseases. Prior to that, he
company. was the Medical Director of Strand Genomics, where he oversaw
Ms. Krasney received her Bachelor of Arts degree from the development of new technologies such as the liquid biopsy and
University of California, Los Angeles and her Juris Doctorate customized cancer panels, while he oversaw clinical reporting for
from the University of Southern California. over 5,000 patients a year.
Dr. Borgonha was a co-founder of Angstrom Medica (Acquired by
Pioneer Surgical), a biomaterials science company. He is also
a co-founder of ten3T, a remote cardiac monitoring company.
AJ Bergmann, M.B.A. Dr. Borgonha is a graduate of St. John’s Medical
Chief Financial Officer College, Bangalore and the Sloan School of Management
Mr. Bergmann has worked in the finance industry for over a at MIT.
decade.
Mr. Bergmann joined Capricor in 2011 and coordinated the
Company’s reverse merger, uplisting to NASDAQ and public
financings yielding over $85 million to date.
Mr. Bergmann graduated from Providence College and has a
M.B.A. from the University of Southern California’s Marshall
School of Business.
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