NASDAQ: CAPR

Transformative Therapies from Bench to Bedside

www.capricor.com

Investor Presentation

May 2016

2

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

Capricor's business are set forth in Capricor's Annual Report on Form 10-K for the year ended

December 31, 2015, as filed with the Securities and Exchange Commission on March 30, 2016,

and in its Registration Statement on Form S-3, as filed with the Securities and Exchange

Commission on September 28, 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.

Forward-Looking Statements

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Capricor – Investment Highlights

– Innovative platform technologies in regenerative medicine

Therapeutic assets include cell therapy, exosomes, natriuretic peptides

– Clinical-stage programs in heart failure, Duchenne muscular dystrophy

Proof-of-concept demonstrated by several clinical studies

Orphan drug designation for DMD

– Data expected from key clinical trials within 12 months

– Exclusive license option with Janssen Biotech

– ~$30 million in non-dilutive financial support to date

– Cash runway through early 2017 data readouts

4

Capricor’s Platforms & Therapeutic Pipeline

Cardiosphere-Derived Cells

Exosomes Platform

Natriuretic Peptide

CAP-1002, CAP-2003, and Cenderitide are investigational drug products and are not approved for any indications.

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Cardiosphere-Derived Cells (CDCs)

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CDCs – a Regenerative Medicine Platform

Cardiosphere-derived

cells (CDCs)Cardiospheres

(CSps)

Explant-derived

cells (EDCs)ExplantsCardiac Tissue

– Therapeutic potential of CDCs to address cardiovascular conditions supported by

a large body of preclinical data and results from several clinical trials

– CDCs function as a local drug delivery system:

Prevent cardiomyocyte programmed cell death

Promote cell growth and new blood vessel formation

Exert anti-scarring activity

Attract endogenous stem cells (paracrine mode of action)

– Proprietary manufacturing process:

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CADUCEUS Study Provides Clinical Proof-of-Concept

– Patients with reduced ejection fraction (EF) following myocardial infarction (MI)

– N=25 (17 active, 8 control)

– One-time intracoronary delivery of CAP-1001 = autologous CDCs (25 million cells)

– Sponsored by Cedars-Sinai Medical Center, with Johns Hopkins University

Lancet, 2012, 21(6): 1121-1135.

In CADUCEUS, treatment with CDCs following a heart attack:

• reduced the amount of scar in the heart

• increased the amount of healthy heart muscle

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CDC Therapy with CAP-1001 Reduced Scar Size

and Increased Healthy Heart Muscle

Scar size shown to be predictive of clinical outcome after heart attack

Burns et al, JACC 2002;39:30-6; Klem et al, JACC 2012;60:408-20; Wu et al, Heart 2008;94:730-6.

Makkar et al, Lancet 2012;379:895-904.

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Infarct (Scar) Size Significantly Impacts Survival

Wu et al, Heart 2008;94:730-6.

Kaplan-Meier curve for MACE

death

MI

heart failure admission

following acute ST-segment elevation MI,

divided by an 18.5% infarct size

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– Therapeutic regeneration

– Reversibility of “irreversible” injury

– Curative approach vs. stabilization and palliation

CADUCEUS Provides In-Human Evidence for Several

Groundbreaking Therapeutic Concepts

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ALLSTAR

DYNAMIC

HOPE-Duchenne

Population

Allogeneic cells from donor hearts – would provide for “off-the-shelf” product

Post-MI Cardiac

Dysfunction

Advanced

Heart Failure

DMD-Associated

Cardiomyopathy

Status

Expect to report Phase I / II data

in Q1 2017

Phase I – data reported

Phase II – six-month data

expected in Q1 2017

Six-month data reported

Expect to report 12-month data

in Q3 2016

CAP-1002 is Next-Generation CDCs

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– Collaborating with Janssen on CAP-1002 manufacturing development

– $12.5 million upfront payment received in early 2014

– Janssen has exclusive option to enter into exclusive license agreement

for worldwide rights to CAP-1002 for certain cardiovascular indications

– Option is exercisable until 60 days following delivery of the six-month

data from the ALLSTAR trial

– If exercised, Janssen to fund all future development costs and

Capricor eligible to receive:

License fee and additional milestone payments totaling up to $325 million

Double-digit royalties on product sales

Agreement with Janssen Biotech, Inc. on CAP-1002

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ALLSTAR

Janssen collaboration

CIRM loan award

Phase I / II

Six-month Phase II data

expected in Q1 2017

Evaluate safety & efficacy

in post-MI setting

CAP-1002 Clinical Development Program

DYNAMIC

NIH-SBIR grant – $3 M

Phase I / II

Six-month data: 2015 AHA

Expect to report 12-month

data in Q3 2016

Evaluate safety & POC

in advanced HF

HOPE-Duchenne

Orphan Designation

CIRM grant – $3.4 M

Phase I / II

Expect to report top-line

data in Q1 2017

Evaluate safety & POC

in DMD-cardiomyopathy

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– No immunological safety events

– Advancement to Phase II approved by DSMB

Pre-Specified Safety Endpoint

Observed (N=14)

Month 1 Month 12

Acute myocarditis possibly attributable to CAP-1002 0 (0%) 0 (0%)

Death due to ventricular tachycardia / fibrillation 0 (0%) 0 (0%)

Sudden death 0 (0%) 0 (0%)

Major Adverse Cardiac Event (MACE) 0 (0%) 0 (0%)

ALLSTAR Phase I Safety Results

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ALLSTAR Phase I Results:

Showed Improvement in Scar Size

* by groups t-test # by paired t-test

– Results shown are from the “Phase II Equivalent” populationReceived high dose CAP-1002

Prospectively matched (lacked DSAs at screening)

DSA = donor-specific antibody

p < 0.05 #p < 0.05 *

n=8 n=8 n=7

Infa

rct

Siz

e

Infa

rct

Siz

e

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ALLSTAR

Janssen collaboration

CIRM loan award

Phase I / II

Six-month Phase II data

expected in Q1 2017

Evaluate safety & efficacy

in post-MI setting

CAP-1002 Clinical Development Program

DYNAMIC

NIH-SBIR grant – $3 M

Phase I / II

Six-month data: 2015 AHA

Expect to report 12-month

data in Q3 2016

Evaluate safety & POC

in advanced HF

HOPE-Duchenne

Orphan Designation

CIRM grant – $3.4 M

Phase I / II

Expect to report top-line

data in Q1 2017

Evaluate safety & POC

in DMD-cardiomyopathy

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– Multi-vessel intracoronary infusion of allogeneic CDCs

(up to 75 million cells) appeared to be safe and feasible

in the DYNAMIC study

– Advancement of trial was approved by DSMB

Pre-Specified Safety Endpoint Observed (N=14)

TIMI Flow Score 0-2 0 (0%)

Acute myocarditis within one month 0 (0%)

Ventricular tachycardia / fibrillation within 72 hours 0 (0%)

Sudden unexpected death within 72 hours 0 (0%)

Major Adverse Cardiac Event (MACE) within one month 0 (0%)

DYNAMIC Six-Month Safety Results

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Presented at the American Heart Association

Annual Meeting, November 2015

NYHA Class Physical Activity

I No limitation

II Slight limitation

III Marked limitation

Of the 12 evaluable Class III patients at six months,

11 (92%) improved by at least one NYHA class

Overall Cardiac Status Improved in DYNAMIC

N=14; two subjects lost to follow-up.

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6 Minute Walk Test

& VO2 Max

Quality of Life

less is better

Other Clinical Improvements Observed in DYNAMIC

N=14; two subjects lost to follow-up; AHA Annual Meeting, November 2015.

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Left Ventricular Dynamics & Dimensions

less is better

DYNAMIC demonstrated an efficacy signal with concordant improvements

in functional status, quality-of-life, and left ventricular function and size

Standard Heart Parameters Improved in DYNAMIC

N=14; two subjects lost to follow-up; measurements assessed by

echocardiography; AHA Annual Meeting, November 2015.

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ALLSTAR

Janssen collaboration

CIRM loan award

Phase I / II

Six-month Phase II data

expected in Q1 2017

Evaluate safety & efficacy

in post-MI setting

CAP-1002 Clinical Development Program

DYNAMIC

NIH-SBIR grant – $3 M

Phase I / II

Six-month data: 2015 AHA

Expect to report 12-month

data in Q3 2016

Evaluate safety & POC

in advanced HF

HOPE-Duchenne

Orphan Designation

CIRM grant – $3.4 M

Phase I / II

Expect to report top-line

data in Q1 2017

Evaluate safety & POC

in DMD-cardiomyopathy

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– Duchenne muscular dystrophy is an X-linked genetic disorder of muscle

Affects one in ~3,500 newborn boys around the world

Estimated 30,000 boys afflicted with DMD in U.S. and EU1

Progressive weakness in early years, eventually become wheelchair-bound

– Cardiomyopathy is associated with a poor prognosis

Accounts for 20-40% of all deaths2

Heart Disease is a Leading Cause of Death in DMD

1 JMP Securities, Research Advances in Duchenne Muscular Dystrophy, December 2015.2 Schram et al, JACC 2013;61:948-54.

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Menon et al, Pediatr Cardiol 2014;35:1279-85.

In cardiac MRI studies in the DMD population, myocardial fibrosis (scar):

is an independent predictor of adverse cardiac

remodeling, ventricular arrhythmias, and death

increases linearly with age

and predicts LVEF

Background on DMD-Associated Cardiomyopathy

Tandon et al, J Am Heart Assoc 2015;4:e001338.

and

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– FDA Orphan Drug Designation for the treatment of DMD

– Distinct from the dystrophin-correcting therapies

Directed toward the heart muscle

Apparent lack of mutation dependence supports use in any genotype

CAP-1002 in Clinical Development for

DMD-Associated Cardiomyopathy

25

30

40

50

60

70

80

Mdx + CAP-1002

Mdx + Vehicle

*** p < 0.001 *p < 0.05

100

200

300

400

500

600

700

800

3 4 5 6

CTL

Mdx + CAP-1002

Mdx + vehicle

Week

***

1st injection

EF

(%

)

Repeat Dosing

Me

ters

2nd injection

Mdx mice + CDC, n=12 Mdx mice + vehicle, n=12 CTL (wild-type), n=5

CAP-1002 Improves Cardiac Function and

Exercise Capacity in DMD Mouse Model

ISEV, April 2015; AHA, November 2014

**

**

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– Enrolling boys with cardiomyopathy secondary to DMD

– Parallel-group, multi-center study (3-4 sites)

1:1 randomization to CAP-1002 infusion or ‘usual care’

– Passed DSMB safety review of first patient cohort

– Expect to report six-month top-line data in Q1 2017

Structural (cardiac MRI) and functional endpoints

Quality of Life endpoints

Randomize

CAP-1002

Infusion

Usual Care

M12W2 W6 M3 M630 d

Randomized Phase I / II HOPE-Duchenne Trial Ongoing

Screen

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Exosomes:

Next-Generation Regenerative Medicine Platform

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– Cell-free technology

– Nanometer-sized vesicles

– Secreted by nearly all cell types

– Rich in RNAs and proteins

– Readily cross cell membranes

– Cell signaling modality

– Broad potential to treat

inflammatory disorders

– Capricor has exclusive WW

license to CDC exosomes

technology from Cedars-Sinai

Medical Center

Camussi et al, Kidney Int 2010;78:838–48.

Exosomes are an Emerging Class of Therapeutics

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Ibrahim et al, Stem Cell Reports 2014;2:606-19.

Effects not observed with

exosomes from other cell types

CDC Exosomes Significantly Improve

Cardiac Structure and Function

0

5

10

15

20

CTRL NHDF-XO MSC-XO CDC-XO

Sc

ar

Ma

ss

(m

g)

Day 3

0

*****

25

30

35

40

45

50

1 15 30

EF

(%

)

Days post MI

ControlCDC-XONHDF-XOMSC-XO *

**

Control NHDF-Exosomes

MSC-Exosomes CDC-Exosomes

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– CDC exosomes appear to have regenerative and cell-modulating capabilities

– Broad treatment potential in inflammatory conditions

Supported by growing body of preclinical data

– Capricor plans to announce the first indication for CAP-2003 in Q2 2016

Eye and skin disorders are under consideration

CAP-2003 (CDC Exosomes) Moving into the Clinic

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-S-S-

K

L

L

DR I G

S

M

S

G

LGGF

C C

K

G

S

L

G

pGC-B agonist

Anti-fibrotic

Anti-inflammatory

Endothelial regenerating P

S

L

R

D

PR

PN

A P ST

SA

pGC-A agonist

Enhances renal function

Suppresses aldosterone

Promotes cell survival

Resistant to NEP degradation

CNP DNP (C-terminus)

Goal of combined peptide is to yield favorable

hemodynamics with blood pressure maintenance

Cenderitide (CD-NP):

A Dual Natriuretic Peptide Receptor Activator

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Cenderitide is Continuously Administered via Patch Pump

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Target Indication

Prevention of re-hospitalization in patients with a recent acute heart failure admission

Cenderitide in Development for Outpatient

and Ambulatory Heart Failure

– Phase IIa PK/PD TrialPatients with stable chronic heart failure

To assess the safety and tolerability, pharmacokinetics profiles, and

pharmacodynamic response to increasing dose levels of Cenderitide

– First arm of 14 patients completedNo significant issues with safety or Insulet pump delivery

Early results suggest tolerability and physiologic effect

– Data from second arm expected in Q2 2016

Designed to assess higher doses

– To announce further plans following results

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Cash, cash equivalents, and marketable securities

reported at December 31, 2015$13.6 million

Net cash used in operations in 2015 $10.8 million

Shares outstanding (March 29, 2016) 18.0 million

$4.1 millionraised in equity financing

$3.4 millionawarded by CIRM to support

HOPE-Duchenne clinical trial

March 2016:

Financial Summary

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AJ Bergmann, MBAVP of Finance

Leland Gershell, MD PhDChief Financial Officer

Deborah Ascheim, MDChief Medical Officer

Karen Krasney, JDEVP & General Counsel

Rachel Smith, PhDVP of Research & Development

Houman Hemmati, MD PhDVP of New Therapy Development

Luis Rodriguez-Borlado, PhDVP of Regenerative Therapies

Linda Marbán, PhDChief Executive Officer

Senior Management

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2016 2017 Anticipated Milestone

Q2Complete Phase IIa trial of Cenderitide

Announce indication for CAP-2003

Q3

Report 12-month results from DYNAMIC trial

Complete enrollment in HOPE-Duchenne trial

Complete enrollment in ALLSTAR trial

Q4 Submit IND for clinical development of CAP-2003

Q1

Report top-line data from HOPE-Duchenne trial

Six-month data from ALLSTAR trial

to be available to Capricor*

* Janssen has the right to exercise its option to CAP-1002 at any time until 60 days after

the delivery by Capricor of the six-month follow-up results from the ALLSTAR clinical trial.

Upcoming Milestones

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Capricor – Investment Highlights

– Innovative platform technologies in regenerative medicine

Therapeutic assets include cell therapy, exosomes, natriuretic peptides

– Clinical-stage programs in heart failure, Duchenne muscular dystrophy

Proof-of-concept demonstrated by several clinical studies

Orphan drug designation for DMD

– Data expected from key clinical trials within 12 months

– Exclusive license option with Janssen Biotech

– ~$30 million in non-dilutive financial support to date

– Cash runway through early 2017 data readouts

NASDAQ: CAPR

Transformative Therapies from Bench to Bedside

www.capricor.com

8840 Wilshire Boulevard – 2nd floor

Beverly Hills, CA 90211

(310) 358-3200