1. health and fitness
2. disease

Corporate Presentation
May 2015
NASDAQ: CAPR
Transformative Therapies
from Bench to Bedside
Forward Looking Statements
This presentation contains forward-looking statements and information that are based on the beliefs of the
management of Capricor Therapeutics, Inc. (Capricor) as well as assumptions made by and information currently
available to Capricor. All statements other than statements of historical fact included in this presentation are
forward-looking statements, including but not limited to statements identified by the words “anticipates,”
“believes,” “estimates,” and “expects” and similar expressions. Such forward-looking statements also include any
expectation of or dates for commencement of clinical trials, IND filings, similar plans or projections and other
matters that do not relate strictly to historical facts. These statements reflect Capricor’s current views with respect
to future events, based on what we believe are reasonable assumptions; however, the statements are subject to a
number of risks, uncertainties and assumptions. 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 our business are set forth in our Annual Report on Form 10-K for the
year ended December 31, 2014, as filed with the Securities and Exchange Commission on March 16, 2015, in
our Registration Statement on Form S-1, as filed with the Securities and Exchange Commission on March 6, 2015
and in our Form 10-Q for the quarter ended March 31, 2015, as filed with the Securities and Exchange
Commission on May 13, 2015. Should one or more of these risks or uncertainties materialize, or should underlying
assumptions prove incorrect, actual results may vary materially from those in the forward-looking statements.
Further, Capricor’s management does not intend to update these forward-looking statements and information
after the date of this presentation.
2
Capricor Therapeutics Overview
Clinical-stage biotechnology company with a diversified
pipeline focusing on cardiovascular diseases including orphan
indications
Cardiacderived stem
cells (CDCs)
Peptide
therapy for
heart failure
(Cenderitide)
Micro-RNA
Platform
(Exosomes)
3
Capricor Therapeutics Pipeline
Products
Indication
Preclinical
Phase I/II
Phase II
Phase III
Cell Therapy
Post Myocardial Infarction
CDCs (CAP-1002) Advanced Heart Failure (Class III and ambulatory Class IV)
Duchenne Muscular Dystrophy
Natriuretic Peptides
Cenderitide
Advanced Heart Failure
CU-NP
Cardiovascular
(Class III and ambulatory Class IV)
Micro-RNA Technology
Exosomes
Cardiovascular and Non-Cardio
4
Capricor: Key Metrics
Select Data (approximate)
Cash
As of 3.31.15
$24.5M
(Includes restricted cash and marketable securities)
Publicly traded: NASDAQ
CAPR
52 week range
$3.05-$10.68
Shares outstanding
16.2M
Fully diluted shares outstanding
23M
Cash through
~Q3 2016
Non-dilutive capital funding to date
$39.5M
Exclusive Licenses
Johns Hopkins University, Cedars-Sinai Medical Center, Mayo
Foundation for Medical Education and Research and The
University of Rome
Headquarters
Los Angeles, CA
Employees
32
5
Development of Ischemic Heart Failure
Heart
Failure
(hours~days)
(days~weeks)
(weeks~months)
Infarct expansion
Thinning
Chronic LV dilation
Konstam et al., JACC, 2011
6
CELL THERAPY TECHNOLOGY
Lead Product: CDCs (CAP-1002)
Cardiac Tissue
Features
Explants
Explant-derived
cells (EDCs)
Cardiospheres
(CSps)
Cardiosphere-derived
cells (CDCs)
Cardiosphere-Derived Cells (CDCs)
Cell Type
Human cardiac derived stem cell
Characteristics
Panel of cellular markers and secreted factors
Clinical Trials
CADUCEUS – completed autologous Phase I (n=25), showed regeneration in CDC-treated postMI patients (sponsored by CSMC)
ALLSTAR – completed Phase I (n=14), now in Phase II with allogeneic CDCs in post-MI patients
DYNAMIC – ongoing study of allogeneic CDCs in heart failure patients
MOA
Largely paracrine:
 Prevent cardiomyocyte apoptosis (programmed cell death)
 Promote cardiomyocyte proliferation and angiogenesis (cell growth and blood vessel
formation)
 Attract endogenous stem cells
 Anti-fibrotic (anti-scarring)
8
CADUCEUS - Positive First-in-Man Data

Published in Lancet, 2012

Autologous CDCs - 25M cells

Patients with reduced ejection fraction following MI

Sponsored by Cedars-Sinai with Johns Hopkins

Intracoronary delivery

25 patients

17 CDCs

8 Controls
9
CDC Therapy Reduced Scar Size & Increased
Healthy Heart Muscle in the CADUCEUS study
Makkar et al, Lancet, 2012.
CDC patients had a significant reduction in infarct size.
We hypothesize improvement in clinical outcomes.
10
Effect of Infarct (Scar) Size on Events
11
Wu, E. et al. Heart 94, 730-736 (2008).
CDCs: Clinical Development
1 year after MI)
NYHA Class III or
ambulatory Class IV heart
failure
Duchenne muscular
dystrophy-related
cardiomyopathy
14 patient trial
IND
Post myocardial
infarction (30 days –
Phase II
Collaboration with
Janssen Biotech (J&J),
funded in part by CIRM
Funded in part by the NIH
Orphan designation
granted
Enrolling
Data anticipated:
~Q4 2016-Q1 2017
Enrollment complete
Data anticipated:
~Q4 2015
Targeting trial initiation:
Status
Indication
HOPE Clinical Trial
Clinical
DYNAMIC Clinical Trial
Development
ALLSTAR Clinical Trial
submitted
2H 2015
12
ALLSTAR Phase I – 12 month MRI Analysis


ALLSTAR Phase I
 Met safety endpoint (1 month)
 No control group
Preliminary 12 month MRI analysis on Phase II equivalent
population (defined by tissue type compatibility)
 Ejection fraction improved by 5.2%
 Relative reduction in scar size of 20.7%
 Measurements of viable mass and regional function
also showed quantifiable improvements
13
DYNAMIC Clinical Trial
Dilated cardiomYopathy iNtervention with Allogeneic MyocardIallyregenerative Cells
Study Overview
 Patient criteria
 Ischemic or non-ischemic DCM with LVEF ≤ 35%
 NYHA Class III or ambulatory Class IV heart failure
 Delivery: Non-occlusive intracoronary infusion in 3 vessels
 Trial Design
 Feasibility Study - open-label (N=14, single center)
 Enrollment complete
 Top-line data expected Q4 2015
14
Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
 Prevalence: 1 in 3,500 male births worldwide
 ~20,000 male children affected in the US (~275,000 worldwide)
 DMD is usually fatal; a majority of deaths occur due to
cardiomyopathy
 Compelling preclinical data presented at AHA in November 2014
 IND submitted 1H 2015
 HOPE clinical trial planned for 2H 2015
 Intent to complement other dystrophin-correcting therapies for skeletal
muscle
 Orphan disease – presents potential billion dollar market opportunity
Reference: McNeil et. al, Muscle & Nerve, 2010
16
Rationale for CDCs Use in DMD
CDCs





Anti-oxidative
Anti-inflammatory
Anti-apoptotic
Anti-remodeling
Regenerative
DMD pathophysiology




Oxidative/Nitrosative stress
Inflammation
Apoptosis
Remodeling
CDC administration may be beneficial in DMD
cardiomyopathy
Tested in mdx mouse model
17
mdx Mouse Model of Duchenne
Muscular Dystrophy
 Mimics the target indication, DMD
 Aged to the point at which cardiac dysfunction becomes
evident: ≥10 months old
18
Reference: Cedars-Sinai Heart Institute
Global Cardiac Function is Improved in mdx Mice
Single Dose
Repeat Dosing
Mdx+CDC
Mdx+Vehicle
80
CTL(WT)
70
70
65
60
60
***
*
55
*
50
Baseline Wk3
M2
M3
EF(%)
EF(%)
75
Mdx+CDC
1st injection
***
2nd
*
injection
Mdx+Vehicle
*
50
*
*
Wk3
M2
40
30
Baseline Wk3
M2
M3
M3
***
*
p<0.001
p<0.05
n=12 Mdx+CDC, Mdx+vehicle
n=5 CTL(WT)
Presented at AHA - November 2014, Chicago, IL
Reference: Cedars-Sinai Heart Institute
Presented at ISEV - April 2015, Washington DC
19
Distance(m)
CDCs Increased Maximal Exercise Capacity
820
760
700
640
580
520
460
400
340
280
220
160
100
CTL
Mdx+CDC
Mdx+Vehicle
n = 6-11
**
**
**
**
wk3
wk4
wk5
wk6
Presented at AHA - November 2014, Chicago, IL
Reference: Cedars-Sinai Heart Institute
20
Duchenne Cardiomyopathy and CDCs

Injection of CDCs into mdx hearts
 Improves global function
 Decreases fibrosis
 Improves exercise capacity
 Exerts potent anti-oxidant effects
 Reverses abnormalities in mitochondrial
abundance, structure and function
 Increases cardiomyocyte proliferation and
activation/recruitment of endogenous repair
Presented at AHA - November 2014, Chicago, IL
Reference: Cedars-Sinai Heart Institute
21
Capricor’s CDCs Are Unique
Capricor’s CDCs
are the only
allogeneic,
intracoronary
delivered,
cardiac-derived
stem cells
CDCs
22
Natriuretic Peptide Technology
Cenderitide: A Unique Protein Drug
 Developed by scientists at the Mayo
Clinic and derived from the venom
of the green mamba snake
D
G
S
M
L
K
F
C
G
K
S
G
NH2
G
L
G
L
S
CD-NP
L
 Cenderitide:
 Cardiac unloading
 Renal function preserved
 Aldosterone suppressing
 Anti-fibrotic, apoptotic, and
hypertrophic
I
R
-S-S-
C
G
P
S
L
R
D
P
R
P
N
 270 patients with acute
decompensated heart failure have
been treated
A
P
S
T
S
24
A
Continuous Subcutaneous Infusion Using
the Insulet Omnipod Technology
®
 Current pump delivery systems are robust, simple, and well
tolerated
 In use by more than 300,000 patients worldwide
 Pump use is simple for all types of patients, not just diabetics
 Continuous delivery is ideal for worry-free dosing throughout the
day and night
Fill the Pod
Apply the Pod
Press Start
Sample shown: Omnipod®
25
Cenderitide’s Development
The Opportunity
R I
D
G
S
M
L
S
K
G
L
L
G
F
C
G
K
S
L
G
C
G
P
S
L
R
D
P
R
P
N
A
P
S
+
T
S
A
1st Phase II clinical
trial complete
26
Cenderitide for Outpatient
and Ambulatory Heart Failure
Target
Indication
Prevention of re-hospitalization in patients with a
recent acute heart failure admission as well as
other potential indications
Phase II Trial enrollment complete
 14 patients treated
 Patients with stable chronic heart failure
 Trial assessing the safety and tolerability, pharmacokinetics
profiles, and pharmacodynamic response to increasing dose
levels of Cenderitide
 Early results suggest tolerability and physiologic effect
 Will announce further plans in late 2015, early 2016
27
Exosomes: Micro-RNA Platform
Technology
Exosomes: Micro-RNA Platform Technology

Nanometer-sized lipid-bilayer
vesicles

Rich in miRNAs

Present in virtually all body
fluids

Released by nearly all cell
types

Potential for broad therapeutic
applicability
29
CDCs Release Bioactive Exosomes that Recapitulate
their Therapeutic Effects in Cardiac Injury
50
Control
45
CDC-XO
*
**
EF (%)
Skin-XO
40
35
CDC-Exosomes
30
Skin-Exosomes
CTRL
25
1
15
30
Days post MI
Ibrahim et al., Stem Cell Reports, 2014
30
CDC Exosomes: Isolation Methods
and Phenotype
CDCs (lines 155 or 220) or inert
NHDFs (dermal fibroblasts)
Exosome Isolation
Collect Conditioned
Media

BATCH ANALYS
BATCH ANALYSIS REPORT
220
Nanoparticle Tracking Analysis (NTA) Version 2.3 Build 0034
Nanoparticle Tracking Analysis (NTA) Version 2.3 Build 0034 Nanoparticle Tracking Analysis (NTA) Version 2.3 Build 0034
200
300
400
500
600
700
0
800
100
900
200
1000
300
4000
5.84
inert
500100
600200
700300
Size (nm)
Size (nm)
CD105 (~80kDa)
3.48
100
200
300
400
500
100
Averaged Size / Concentration
Red error bars indicate +/- 1 standard error of the mean
3.48
Concentration (E6 particles/ml)
3.77
220
155
Concentration (E6 particles/ml)
0
100
200
300
400
500
HSP70 (~70kDa)
Particle # (108)/mL
CD63 (~53kDa)
CD9 (~28kDa)
CD81 (~26kDa)
4.99
Concentration (E6 particles/ml)
4.99
3.06
Concentration (E6 particles/ml)
Nanosight
3.56
CDC Exososomal
Marker Markers
Precipitate by ExoQuick (in
vivo studies)
Isolate by ultracentrifugation
(in vitro studies in iPS cells)
Concentration (E6 particles/ml)
155

____
____
____
____
____
800400
0
900500
100
1000
200
600
Size (nm)
Averaged Size / Concentration Size / Concentration
Red error bars indicate +/- 1 standard error of the mean
155_10x Media_07231401
155_10x Media_07231402
155_10x Media_07231403
155_10x Media_07231404
155_10x Media_07231405
100
200
300
400
500
Culture in SF Media
(15 days)
300
700
0400
800
100
500
900
200
600
1000
300
700
400
800
500
900
600
100
Size (nm)
Size (nm)
Averaged Size / Concentration Size / Concentration
Red error bars indicate +/- 1 standard error of the mean
Particle Size (nm)
Operator:
Operator:
GEA
Sample:
CDCs CD63 20ulSample:
Included files:
155_10x Media_07231401
Included files:
GEA RESULTS: Operator:
BATCH AVERAGE
CDCs CD63 20ul Sample:
220_10x Media_07231401
Size Distribution:
Mean:
165 +/9.7 nm
Included
files:
155_10x Media_07231402
220_10x Media_07231402
Mode: 121 +/- 3.4 nm
155_10x Media_07231403
220_10x Media_07231403
SD: 75 +/- 15.8 nm
155_10x Media_07231404
Date/Time of Report
155_10x Media_07231405
Date/Time of Report
23/07/2014 12:07:08
Dispersant/Diluent:
PBS
Dispersant/Diluent:
Concentration:
1:100 PBS
Concentration:
BATCH AVERAGE
RESULTS:
GEA
CDCs CD63 20ul
Size Distribution: HT_10x d15Mean:
168 +/- 5.1 nm
Med_07241401
31
134 +/- 4.4 nm
HT_10x d15Mode:
Med_07241402
69 +/- 3.7 nm
HT_10x d15SD:
Med_07241403
Presented at ISEV - April 2015, Washington DC
220_10x Media_07231404
D10: 90 +/- 2.9 nm
220_10x Media_07231405
D50: 146 +/- 2.2 nm
23/07/2014 12:23:39
D90:
265 +/- 31.9
nm
Date/Time
of Report
96 +/- 2.2 nm
HT_10x d15D10:
Med_07241404
153 +/- 4.0 nm
HT_10x d15D50:
Med_07241405
255 +/- 9.8 nm
24/07/2014 D90:
09:45:49
Total Concentration:
26.61 +/- 0.95 particles/fram
PBS
Reference: Cedars-Sinai Heart Institute
PBS
Total Concentration:
1:100 PBS
19.27
+/- 0.41 particles/frame
Dispersant/Diluent:
4.07Concentration:
+/- 0.08 E8 particles/ml
Total Completed Tracks:
3851
1:100 PBS 5.72 +/- 0.21 E8 particles/m
Total Completed Tracks:
5272
Average Drift Velocity:
1030 nm/s
Advantages for Exosomes for
Tissue Repair
 Novel delivery vehicle reproducing the beneficial
effects of living cells
 Natural capacity to protect their bioactive cargo
 Potential for simple manufacturing protocols
 Reduced immunogenicity
32
EL Andaloussi et. Al. Nat Rev Drug Discov. 2013 May;12(5):347–57.
Exosomes as a Potential New Therapeutic
Product
Description
Released by nearly all cell types and body fluids,
these are nanometer sized lipid-bilayer vesicles
rich in miRNAs
Product
Function
Promotes similar paracrine effects of CDCs;
prevent apoptosis, proliferation, angiogenesis,
anti-fibrotic
Product
Development
Ongoing pre-clinical work, developing
manufacturing and pre-commercial scale up
Clinical
Status
Targeting IND in 2016
33
2015 Targeted Milestones


1H 2015
 Complete DYNAMIC trial enrollment – completed
 Complete Cenderitide Phase II enrollment – completed
 Receive Orphan Designation for DMD – completed
 Submit IND for DMD-associated cardiomyopathy – completed
2H 2015
 Initiate HOPE-DUCHENNE trial
 Report initial DYNAMIC results
 Report initial Cenderitide results
 Announce development program for natriuretic peptides
 Announce 1st indication for exosomes
34
Senior Management & Board of Directors
Senior Management






Chief Executive Officer
Linda Marbán, Ph.D. (Founder)
Scientific Advisory Board Chairman
Dr. Eduardo Marbán (Founder, JHU,
Cedars-Sinai)
VP of Research and Development
Rachel Smith, Ph.D. (Johns Hopkins)
EVP & General Counsel
Karen Krasney, J.D. (Biosensors)
VP of Medical Affairs
Andrew Hamer, M.D. (Chairman –
New Zealand Cardiac Network)
VP of Clinical Operations
Shane Smith (Nektar, Genentech)
Board of Directors









Executive Chairman
Frank Litvack, M.D. (ConorMed)
Linda Marbán, Ph.D.
Dave Musket (ProMed Partners)
Earl M. (Duke) Collier, Jr.
(Genzyme)
George W. Dunbar, Jr.
(Aastrom)
Joshua Kazam (Kite, Two-River)
Gregory Schafer (Aduro,
Onyx)
Louis Manzo (Investor)
Louis J. Grasmick (Investor)
35