28 annual roth conference nasdaq: caprcapricor.com/wp-content/uploads/2014/05/capr_roth_031516.pdf20...

NASDAQ: CAPR

Transformative Therapies from Bench to Bedside

www.capricor.com

28th Annual ROTH Conference

March 15, 2016

2

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 Capricor's business are set forth in Capricor's 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 its Registration Statement on Form S-3, as filed with the Securities and

Exchange Commission on September 28, 2015, and in its Quarterly Report on Form 10-Q for the

quarter ended September 30, 2015, as filed with the Securities and Exchange Commission on

November 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.

3

Changing the Paradigm

– Focus on cardiovascular indications

– Clinical programs in heart failure, Duchenne’s muscular dystrophy

– Innovative platforms: Regenerative Medicine, Exosomes

– Technology licensed from world-renowned academic institutions

4

Cardiosphere-Derived Cells

Exosomes Platform

Natriuretic Peptide

Capricor’s Platforms & Therapeutic Pipeline

5

Cardiosphere-Derived Cells (CDCs)

6

CDCs – Regenerative Medicine Platform

Cardiosphere-derived

cells (CDCs)

Cardiospheres

(CSps)

Explant-derived

cells (EDCs) Explants Cardiac Tissue

CDCs Function as a Local Drug Delivery System:

– Prevent cardiomyocyte apoptosis (programmed cell death)

– Promote cardiomyocyte proliferation (cell growth) and

angiogenesis (new blood vessel formation)

– Anti-fibrotic (anti-scarring)

– Attract endogenous stem cells (paracrine)

7

CADUCEUS: Proof-of-Concept Clinical Data

– One-time intracoronary delivery of CAP-1001 = autologous CDCs

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

– 25 patients (17 active, 8 control)

– Sponsored by Cedars-Sinai Medical Center

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

Results showed that treatment with CDCs following a heart attack:

• reduced the amount of scar in the heart

• increased the amount of healthy heart muscle

8

Scar size and myocardial reserve are

predictors of clinical outcome after heart attack

CDC Therapy with CAP-1001 Reduces

Scar Size & Increased Healthy Heart Muscle

Burns et al, JACC 2002;39:30-6.

Klem et al, JACC 2012;60:408-20.

Wu et al, Heart 2008;94:730-736.

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

9

CADUCEUS Provides Evidence for Several Groundbreaking Therapeutic Concepts

– Therapeutic regeneration

– Reversibility of “irreversible” injury

– Curative approach vs. stabilization & palliation

10

ALLSTAR

CAP-1002 is Next-Generation CDCs

DYNAMIC

HOPE-Duchenne

Population

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

Heart failure

(post-MI)

Heart failure

(advanced)

DMD-associated

cardiomyopathy

Status

Ph. II – to report Q1 2017

Ph. I – reported

Ph. II – to report Q1 2017

6-month data reported

12-month data pending

11

ALLSTAR

Janssen Biotech (J&J)

collaboration

CIRM loan award

Phase II

Phase II enrolling

Data expected Q1 2017

Evaluate safety & POC

in HF post-MI

with ALLO cells

CAP-1002 Clinical Development

12

ALLSTAR Phase I results: Showed Improvement in Scar Size

P<0.05*

n=4

14

16

18

20

22

24

26

28

IS (

% L

V)

Infarct Size

Baseline

12 Months

14

16

18

20

22

24

26

28

Baseline 12 Months

IS (

% L

V)

Infarct Size

P<0.05#

“Phase II Equivalent” Population = High Dose, w/o DSAs

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

13

ALLSTAR Phase I results: Showed Improvement in Cardiac Function

14

ALLSTAR


collaboration

CIRM loan award

Phase II

Phase II enrolling



in HF post-MI

with ALLO cells


Phase I / II


in advanced HF

with ALLO cells

6-month data at AHA 2015


DYNAMIC

NIH grant – $3M

15

Overall Cardiac Status Improved in DYNAMIC

AHA, November 2015

6 month data for 2 subjects pending

NYHA Class Physical Activity

I No limitation

II Slight limitation

III Marked limitation

Of the 12 Class III patients who started the trial and were evaluable at

six months for this analysis, 11 (92%) improved by at least one NYHA class.

16

Other Clinical Improvements Observed in DYNAMIC

AHA, November 2015

6 Minute Walk Test

& VO2 Max

Quality of Life


less is better

17

Key Heart Measures Improved in DYNAMIC

Left Ventricular Dynamics & Dimensions

AHA, November 2015

Measurements assessed by echocardiography


less is better

DYNAMIC demonstrated an efficacy signal with concordant improvements

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

18

ALLSTAR


collaboration

CIRM loan award

Phase II

Phase II enrolling



in HF post-MI

with ALLO cells


Phase I / II


in advanced HF

with ALLO cells

6-month data at AHA 2015


DYNAMIC

NIH grant – $3M

Phase I / II


in DMD-cardiomyopathy

with ALLO cells

FDA Orphan designation

CIRM grant* – $3.4M

Enrolling


HOPE-Duchenne

*pending final approval

19

CAP-1002 in Development for Heart Disease

Associated with DMD

– CAP-1002 is orthogonal to dystrophin-correcting therapies

in development, which target skeletal muscle

Expected to be complementary to other DMD therapies

No evidence for other DMD therapies to provide cardiac benefit

FDA Orphan status

Currently being evaluated in a Phase I / II clinical trial

20

30

40

50

60

70

80

Baseline Wk3 M2 M3 Wk3 M2 M3

Mdx+CDC

Mdx+Vehicle

ISEV, April 2015

AHA, November 2014

CAP-1002 Improves Cardiac Function and

Exercise Capacity in DMD Model

*** p<0.001 * p<0.05

100160220280340400460520580640700760820

3 4 5 6

CTL

Mdx + CDC

Mdx + vehicle

Week

* *

*** * *

1st injection

EF

(%

)

Repeat Dosing

Me

ters

2nd injection

n=12 Mdx + CDC

n=12 Mdx + vehicle

n=5 CTL (wild-type)

21

HOPE-Duchenne Trial Underway

– Phase I / II clinical trial

– Randomized, open label multi-center study (3-4 sites)

12 boys randomized to CAP-1002 infusion

12 boys randomized to ‘usual care’

– Delivered by triple vessel intra-coronary infusion

– Actively enrolling patients

– Preliminary efficacy to be assessed at 6 and 12 months

Halt cardiomyOPathy progrEssion in Duchenne

22

Exosomes:

Next-Generation Regenerative Medicine Platform

23

Exosomes are an Emerging Class of Therapeutics

with Broad Potential

– Cell-free technology

– Nanometer-sized vesicles

– Secreted by nearly all cell types

– Rich in RNAs and proteins

– Readily cross cell membranes

– Cell signaling modality

– Capricor has exclusive WW

license to exosomes technology

originating from CDCs from

Cedars-Sinai

Camussi et al, Kidney Int 2010;78:838–848.

24

CDC Exosomes Significantly Improve Cardiac

Structure and Function

0

2

4

6

8

10

12

14

16

18

CTRL NHDF-XO MSC-XO CDC-XO

Scar

Mas

s (m

g)

** **

*

Control NHDF-Exosomes

MSC-Exosomes CDC-Exosomes

25

30

35

40

45

50

1 15 30

EF (%

)

Days post MI

Control

CDC-XO

NHDF-XO

MSC-XO

** *

Ibrahim et al, Stem Cell Reports 2014;2:606-619.

Effects not observed with exosomes from other cell types

25

Capricor’s CAP-2003 moving into the clinic

– Proof-of-principle has been demonstrated

– CDC exosomes appear to possess regenerative capabilities

– Capricor plans to announce the first indication for its CDC exosome

technology in 1H 2016 (eyes, skin, cancer are in consideration)

– Capricor expects to meet with FDA to discuss clinical development

in 2Q 2016, and to file an IND by YE 2016

26

Cenderitide (CD-NP): A Dual Natriuretic Peptide Receptor Activator

-S-S-

K

L

L

D R I G

S

M

S

G

L G G F

C C

K

G

S

L

G

pGC-B agonist

Anti-fibrotic

Anti-inflammatory

Endothelial regenerating P

S

L

R

D

P

R

P

N

A

P

S

T

S

A

pGC-A agonist

Enhances renal function

Suppresses aldosterone

Promotes cell survival

Resistant to NEP degradation

CNP DNP (C-terminus)

27

Cenderitide is administered via

continuous SC pump

28

Cenderitide for Outpatient

and Ambulatory Heart Failure

Target Indication

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

– Phase IIa PK/PD Trial

1st arm - 14 patients treated, enrollment complete

Patients with stable chronic heart failure

Trial assessed the safety and tolerability, pharmacokinetics profiles, and pharmacodynamic response to increasing dose levels of Cenderitide

No significant issues with safety or Insulet pump delivery

Early results suggest tolerability and physiologic effect

– Initiated a second study to further assess higher doses

– First portion has completed

– To announce further plans following results

29

Financial Summary

Cash, cash equivalents, and marketable securities

reported at September 30, 2015 $17.2 million

Cash used in operations

in the nine months ended September 30, 2015 $7.2 million

Shares outstanding (November 12, 2015) 16.3 million

30

Senior Management

AJ Bergmann, MBA VP of Finance

Leland Gershell, MD PhD Chief Financial Officer

Deborah Ascheim, MD Chief Medical Officer

Karen Krasney, JD EVP & General Counsel

Rachel Smith, PhD VP of Finance

Houman Hemmati, MD PhD VP of New Therapy Development

Luis Rodriguez-Borlado, PhD VP of Regenerative Therapies

Linda Marbán, PhD Chief Executive Officer

31

Board of Directors

Louis Grasmick Founding Investor Linda Marbán, PhD – PRESIDENT

Excigen

George Dunbar, Jr. Aastrom Biosciences

Arboretum Ventures

Earl M. (Duke) Collier, Jr. Genzyme

Joshua Kazam Two River

Kite Pharmaceuticals

Louis Manzo Founding Investor

Gregory Schafer Aduro Pharmaceuticals

Onyx Pharmaceuticals

Frank Litvack, MD – EXECUTIVE CHAIRMAN Conor Medsystems, Savacor

David Musket ProMed Partners

32

Upcoming Milestones

2016 2017

Meet with FDA on exosomes clinical development

Q2

Announce first indication in exosomes program

Complete enrollment in HOPE-Duchenne trial Q3

Report data from Phase I / II HOPE-Duchenne trial

Q1

Report data from Phase II ALLSTAR trial

33

Transformative Therapies from Bench to Bedside

28 annual roth conference nasdaq: caprcapricor.com/wp-content/uploads/2014/05/capr_roth_031516.pdf20...

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