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Transformative Therapies from Bench to Bedside Corporate Presentation June 2014 Non-confidential Forward Looking Statements This presentation contains forward-looking statements and information that are based on the beliefs of the management

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Transformative Therapies from Bench to Bedside

Corporate Presentation June 2014

Non-confidential

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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. 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

  • f 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, 2013, as filed with the Securities and Exchange Commission on March 31, 2014, in our Quarterly Report on Form 10-Q for the period ended March 31, 2014, as filed with the Securities and Exchange Commission on May 15, 2014, and in our Amendment No. 1 to Registration Statement on Form S-1, as filed with the Securities and Exchange Commission

  • n May 23, 2014. 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.

Non-confidential

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Significant U.S. Target Market

  • 1.3M Americans will have a new or recurrent myocardial

infarction (MI) or heart attack

  • 15% will die (1 death every 39 seconds)
  • 36% will develop heart failure (HF)
  • Survival correlated with infarct size
  • 8.5M people in the US have had a heart attack
  • 6.6M Americans are living with HF
  • 50% 5-year mortality rate

American Heart Association. 2010. Wu et al. Heart. 2007.

Cardiovascular Disease Remains America’s #1 Killer

Non-confidential

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Progression of Ischemic Injury Post-MI

Heart Attack

Konstam et al., JACC, 2011

Heart Failure

Adverse Heart Remodeling

(hours~days)

Infarct expansion

(weeks~months)

Chronic LV dilation

(days~weeks)

Thinning

Non-confidential

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Slide 5

Capricor Investment Highlights

CAPR is a publicly-traded, biotech company focused on developing novel therapeutics to prevent and treat heart disease

  • Strategically positioned to treat heart failure
  • 2005: Capricor, Inc. was founded to develop autologous cardiac derived cell therapy
  • November 2013: Capricor Therapeutics was created through merger with Nile Therapeutics
  • Well capitalized through private and public funding
  • Equity investments of $12M
  • Grants of $7M
  • CIRM Loan of $19.8M
  • Janssen collaboration income of $12.5M
  • Total of $39.5M of non-dilutive capital
  • Developmental pipeline and ongoing clinical trials
  • Cardiosphere-derived cells (CDCs): CAP-1001 and CAP-1002
  • Cenderitide and CU-NP
  • Exosomes
  • Exclusive licenses with top academic and research institutions

Non-confidential

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Source of Cash Amount Committed Timing of Funding Use of Cash NIH SBIR Grant $945K Yes Complete R&D NIH Grant $3M Yes Complete Phase I Portion of ALLSTAR CIRM Loan Award $19.8M Yes Ongoing Phase II Portion of ALLSTAR NIH SBIR Grant $3M Yes 2014 Planned DYNAMIC Trial Janssen $12.5M Yes Obtained New Product Development and Manufacturing

TOTAL: $39.5M of Non-Dilutive Capital

Capricor: Capital Efficient and Operationally Leveraged

Non-confidential

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Progress to Date

  • Janssen: signed; $12.5M payment delivered
  • Janssen: CMC research collaboration underway
  • ALLSTAR Ph. I: complete
  • ALLSTAR Ph. II: enrolling
  • DYNAMIC: plan to commence in 2014
  • CENDERITIDE: under review
  • EXOSOMES: new licensed platform product

Non-confidential

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Main Product Candidates

  • CAP-1002
  • Capricor’s lead product candidate
  • Allogeneic, off-the-shelf CDC product
  • ALLSTAR Phase I/II Trial
  • Entered into collaboration and exclusive license option with Janssen Biotech, Inc.
  • Planned DYNAMIC Phase I trial
  • Exosomes
  • Cell free product
  • Delivers micro-RNA directly to the site of injury
  • Nano-particle with flexible delivery options
  • Cenderitide
  • Belongs to class of drugs called natriuretic peptides
  • Designed by Mayo Clinic scientists
  • Potential to be superior HF treatment solution
  • Outpatient therapy for patients with ADHF, the "post-acute" period

Non-confidential

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Janssen Deal Highlights

In December 2013, Capricor entered into Collaboration Agreement and Exclusive License Option with Janssen Biotech (J&J)

  • Capricor received an upfront payment of $12.5M
  • Capricor and Janssen to collaborate on elements of cell manufacturing
  • Janssen has option to enter into exclusive license agreement for CAP-

1002 up to 60 days after receipt of six month ALLSTAR Phase II data

  • If exercises option, Capricor to receive an upfront license fee and additional

milestone payments which may total up to $325M

  • Double-digit royalty to be paid on commercial sales of licensed products
  • Important validation of lead product, CAP-1002, and underlying science
  • Supports the continued development of CAP-1002

Non-confidential

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Slide 10

Cardiosphere-Derived Cells

  • Produced from cardiac tissue
  • Key functions, largely paracrine:
  • Prevent cardiomyocyte apoptosis
  • Promote cardiomyocyte proliferation

and angiogenesis

  • Attract endogenous stem cells
  • Anti-fibrotic

Makkar et al., The Lancet, 2012

Non-confidential

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CADUCEUS - Positive First-in-Man Data

  • Autologous CDCs
  • Patients with reduced EF% following MI
  • Cedars-Sinai and JHU sponsored
  • Delivered via standard interventional catheters to infarct-

related artery

  • 25 patients
  • 17 CDCs
  • 8 SOC
  • Results published in the Lancet (Makkar, 2012)

Non-confidential

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CDC Therapy Reduced Scar Size and Increased Healthy Heart Muscle in CADUCEUS

p<0.001 p:0.008

p:0.002 p<0.001

Δ scar mass Δ viable mass

6 mos 12 mos 6 mos 12 mos

Makkar et al, Lancet, 2012.

CDC Patients had a Significant Reduction in Infarct Size. We hypothesize improvement in clinical outcomes

Non-confidential

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The CADUCEUS Study: CDCs and Infarct Size

  • Both groups (CDC and CTRL)

started with IS of 24%

  • Patients treated with CDCs saw

a reduction of IS to 12.5%

  • This moved patients from a high

risk group to a low risk group.

  • CTRL patients saw no change in

IS, which puts them at great risk for adverse events CDCs

Wu, E. et al. Heart 94, 730-736 (2008).

Non-confidential

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Limitations of Autologous Therapy

Restrictions of effective autologous stem cells transplantation, in which cells are removed, stored, and returned to same patient

  • Timing constraints
  • 3-5 weeks to obtain and then develop CDCs
  • High Expense
  • Significant Risk
  • Harvesting procedure
  • Manufacturing failure
  • Suboptimal QA/QC
  • Inter-patient variability in cell potency

Non-confidential

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Advantages of Allogeneic CDCs

Donor Heart

Modified from Smith et al., Circulation 2007

Several advantages to allogeneic stem cell transplantation, in which patient receives cells from a genetically similar, but not identical, donor

  • Donors pre-screened by organ procurement organizations
  • Semi-automated explant creation
  • Large master cell banks created from each donor
  • Batch production of cryopreserved doses
  • FDA approved for Phase II clinical trial use

Explant Culture Cardiosphere Formation CDC Expansion Patient Dose

CAP-1002

Non-confidential

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ALLSTAR Phase I/II Update

  • ALLSTAR Phase I
  • 14 patients/ open label dose escalation
  • Following patients for 1-year
  • Data will be presented at TCT and/or AHA
  • Funded in large part with grant from NIH
  • ALLSTAR Phase II
  • Est.300 patients / randomized-placebo controlled
  • Currently enrolling patients
  • 15 sites active
  • Targeting approx. 35 sites
  • 6 month secondary endpoint anticipated in 2016
  • Funded in large part with loan award from CIRM

Non-confidential

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Phase I Primary Endpoint

  • ALLSTAR Phase I met the primary

safety endpoint

  • 1° Safety Endpoint: 1 month post-infusion
  • NO Acute Myocarditis attributable to CAP-1002
  • NO Death due to VT/VF
  • NO Sudden Death
  • NO Major Adverse Cardiac Events (MACE)
  • NIH DSMB approved advancement

to Phase II

Non-confidential

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DYNAMIC: Phase I a/b Trial of CDCs

Study Overview

  • IND Sponsor: Cedars-Sinai Medical Center (E. Marbán)
  • Patient criteria
  • Ischemic or non-ischemic DCM with LVEF ≤ 35%
  • NYHA Class III or ambulatory Class IV heart failure
  • Hospitalization for heart failure within 12 months
  • Delivery: Non-occlusive intracoronary infusion in 2-3 vessels
  • Trial Design
  • Phase Ia: open-label (N=14, single center): safety
  • Phase Ib: randomized, double-blinded placebo-controlled (n=28, 4

centers): safety and exploratory efficacy

Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally- regenerative Cells

Non-confidential

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Well Positioned for Success in Cell Therapy

CDCs’ Unique Mechanism of Action CDCs demonstrated a reduction in infarct size and cardiac regeneration in the POC CADUCEUS trial Ease of Use Allogeneic cells allow for an “off-the-shelf” product Stable Supply Chain Donor hearts provide a sustainable source of tissue from which CDCs can be derived Scalable Manufacturing One heart can provide thousands of doses, with a targeted gross margin of over 90% Novel Technology IP portfolio Experienced Leadership World renown scientific and management teams

Non-confidential

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What are exosomes?

  • Nanometer-sized lipid-bilayer vesicles
  • Produced via endosome fusion
  • Transfer proteins and RNAs
  • Rich in miRNAs
  • Present in virtually all body fluids
  • Released by nearly all cell types

Non-confidential

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Why investigate CDC Exosomes?

  • Possible mediators of paracrine effects of CDCs
  • Non-cellular means of attaining a cell effect
  • Readily extracted from cell conditioned media
  • Nanoparticles with flexible delivery options
  • Size ~ not limiting for safety
  • Dose ~ limited by efficacy
  • Stability ~ flexibility in storage

Non-confidential

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CDC Exosomes: Cardioprotection

CTRL NHDF- Exosomes CDC-Exosomes

25 30 35 40 45 50 1 15 30 EF (%) Days (after MI) Control CDC-XO NHDF-XO

** *

5 10 15 20 25 30 35 40 45 50

CTRL NHDF-XO CDC-XO

Viable Mass (mg)

2 4 6 8 10 12 14 16 18

CTRL NHDF-XO CDC-XO

Scar Mass (mg)

** ** ** **

Ibrahim et al., Stem Cell Reports, 2014

Non-confidential

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Cardiac Exosomes: POC Models

20 25 30 35 40 45 50 1 15 30

EF (%)

Days (after MI)

CDC-DMSO CDC-GW4869

CDC-GW4869

*

5 10 15 20 CDC-DMSO CDC-GW4869

Scar Mass (mg)

10 20 30 40 50 60 CDC-DMSO CDC-GW4869

Viable Mass (mg)

* *

CDC-DMSO

  • A. Ibrahim, Marbán Lab

Non-confidential

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Summary and Conclusions

  • CDCs secrete exosomes which mediate the

therapeutic effects of CDCs in vivo

  • CDC exosomes contain a unique repertoire of

microRNAs (miRs) compared to an inert cell control

  • CDC exosomes represent a potential new

therapeutic

Non-confidential

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Mechanism of Action

  • CDCs secrete exosomes which

mediate the therapeutic effects

  • f CDCs in vivo
  • CDC exosomes contain a unique

repertoire of microRNAs (miRs) compared to an inert cell control

  • CDC exosomes represent a

potential new therapeutic

Non-confidential

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Cenderitide: Potential Treatment for HF

Heart attack

(hours~days)

Infarct expansion

(days~weeks)

Thinning

(weeks~months)

Chronic LV dilation

Heart failure

Non-confidential

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Heart Failure Readmission Rates

30-day readmission rates for congestive heart failure exceed 25% on average for ages (18-65+)

Non-confidential

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Post-Acute HF Competitive Landscape

Acute Post-Acute Chronic Duration

3-7 days 1st 90 days after discharge 1-5 years

Location

In-Hospital Home Home

Mortality*

2-4% 8-12% ~20% (1yr) / ~50% 5 yrs

Treatment Goals

  • Relieve dyspnea
  • Off-load excess fluid
  • Survive
  • Stay out of hospital
  • Survive
  • Stay out of hospital

Treatment Options

  • Lasix
  • IV Nitroglycerine
  • Inotropes
  • Natrecor
  • No treatment specifically

targets stabilization and reducing the early re- hospitalization rate

  • Beta blockers
  • ACE inhibitors
  • ARB inhibitors
  • Aldactone
  • Cardio-Rhythm Device

* Includes all mortality up to that point (post-acute mortality = acute mortality + post-discharge mortality)

There are no drugs on the market targeting the post-acute period

Non-confidential

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Cenderitide for Post-Acute HF

Target Indication

Prevention of re-hospitalization in heart failure patients in the post-acute hospitalization period

Treatment Duration

90 days of out-patient treatment after hospital discharge for acute decompensated heart failure

Drug Delivery FDA Status

Fast Track designation granted, March 2011 Subcutaneous chronic infusion pump (validated SQ technology)

Non-confidential

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Slide 30

  • Three legged stool approach to product development
  • Pipeline is complimentary
  • Proof of Concept Established
  • CADUCEUS: CDCs demonstrated a reduction in Infarct Size and

regeneration of heart tissue

  • Active Clinical Developments
  • Phase II ALLSTAR trial using allogeneic CDCs is currently enrolling
  • Cenderitide showed promising prior human IV and SQ use
  • Exosomes as a new platform technology is under

development using the “Capricor Method”

Looking Ahead and Moving Forward

Non-confidential

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Slide 31

Senior Management & Board of Directors

Senior Management

  • Chief Executive Officer

Linda Marbán, Ph.D.

  • EVP & General Counsel

Karen Krasney, J.D.

  • VP of Medical Affairs

Andrew Hamer, M.D.

  • VP of Research & Development

Rachel Smith, Ph.D.

  • VP of Finance

AJ Bergmann, M.B.A.

Board of Directors

  • Executive Chairman

Frank Litvack, M.D.

  • Linda Marbán, Ph.D.
  • Dave Musket
  • Earl M. (Duke) Collier, Jr.
  • George W. Dunbar, Jr.
  • Louis Manzo
  • Louis J. Grasmick
  • Joshua Kazam
  • Gregory Schafer

Non-confidential