Transformative Therapies from Bench to Bedside Corporate - PowerPoint PPT Presentation

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 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 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, 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 on 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 Slide 2

Significant U.S. Target Market Cardiovascular Disease R emains America’s #1 Killer  1.3M Americans will have a new or recurrent myocardial infarction (MI) or heart attack o 15% will die (1 death every 39 seconds) o 36% will develop heart failure (HF) o Survival correlated with infarct size  8.5M people in the US have had a heart attack  6.6M Americans are living with HF o 50% 5-year mortality rate Wu et al. Heart . 2007. American Heart Association. 2010. Non-confidential Slide 3

Progression of Ischemic Injury Post-MI Heart Attack Adverse Heart Remodeling Heart Failure (hours~days) (days~weeks) (weeks~months) Konstam et al., JACC , 2011 Infarct expansion Thinning Chronic LV dilation Slide 4 Non-confidential

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 o 2005: Capricor, Inc. was founded to develop autologous cardiac derived cell therapy o November 2013: Capricor Therapeutics was created through merger with Nile Therapeutics  Well capitalized through private and public funding o Equity investments of $12M o Grants of $7M o CIRM Loan of $19.8M o Janssen collaboration income of $12.5M o Total of $39.5M of non-dilutive capital  Developmental pipeline and ongoing clinical trials o Cardiosphere-derived cells (CDCs): CAP-1001 and CAP-1002 o Cenderitide and CU-NP o Exosomes  Exclusive licenses with top academic and research institutions Non-confidential Slide 5

Capricor: Capital Efficient and Operationally Leveraged Timing of Source of Cash Amount Committed Funding Use of Cash NIH SBIR Grant $945K Yes Complete R&D NIH Grant $3M Yes Complete Phase I Portion of ALLSTAR CIRM Loan $19.8M Yes Ongoing Phase II Portion of Award 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 Non-confidential Slide 6

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 Slide 7

Main Product Candidates  CAP-1002 o Capricor’s lead product candidate o Allogeneic, off-the-shelf CDC product o ALLSTAR Phase I/II Trial o Entered into collaboration and exclusive license option with Janssen Biotech, Inc. o Planned DYNAMIC Phase I trial  Exosomes o Cell free product o Delivers micro-RNA directly to the site of injury o Nano-particle with flexible delivery options  Cenderitide o Belongs to class of drugs called natriuretic peptides o Designed by Mayo Clinic scientists o Potential to be superior HF treatment solution o Outpatient therapy for patients with ADHF, the "post-acute" period Non-confidential Slide 8

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 o 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 Slide 9

Cardiosphere-Derived Cells Makkar et al., The Lancet , 2012  Produced from cardiac tissue  Key functions, largely paracrine: o Prevent cardiomyocyte apoptosis o Promote cardiomyocyte proliferation and angiogenesis o Attract endogenous stem cells o Anti-fibrotic Non-confidential Slide 10

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 o 8 SOC o Results published in the Lancet (Makkar, 2012)  Non-confidential Slide 11

CDC Therapy Reduced Scar Size and Increased Healthy Heart Muscle in CADUCEUS Δ scar mass Δ viable mass 6 mos 12 mos 6 mos 12 mos p<0.001 p:0.002 p<0.001 p:0.008 Makkar et al, Lancet, 2012. CDC Patients had a Significant Reduction in Infarct Size. We hypothesize improvement in clinical outcomes Non-confidential Slide 12

The CADUCEUS Study: CDCs and Infarct Size  Both groups (CDC and CTRL) started with IS of 24%  Patients treated with CDCs saw CDCs 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 Wu, E. et al. Heart 94, 730-736 (2008). Non-confidential Slide 13

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 o High Expense  Significant Risk  Harvesting procedure o Manufacturing failure o Suboptimal QA/QC  Inter-patient variability in cell potency  Non-confidential Slide 14

Advantages of Allogeneic CDCs 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 Cardiosphere Patient Donor Heart Explant Culture CDC Expansion Formation Dose CAP-1002 Modified from Smith et al., Circulation 2007 Non-confidential Slide 15

ALLSTAR Phase I/II Update ALLSTAR Phase I  o 14 patients/ open label dose escalation o Following patients for 1-year o Data will be presented at TCT and/or AHA o Funded in large part with grant from NIH ALLSTAR Phase II  o Est.300 patients / randomized-placebo controlled o Currently enrolling patients o 15 sites active o Targeting approx. 35 sites o 6 month secondary endpoint anticipated in 2016 o Funded in large part with loan award from CIRM Non-confidential Slide 16

Phase I Primary Endpoint  ALLSTAR Phase I met the primary safety endpoint  1 ° Safety Endpoint: 1 month post-infusion o NO Acute Myocarditis attributable to CAP-1002 o NO Death due to VT/VF o NO Sudden Death o NO Major Adverse Cardiac Events (MACE)  NIH DSMB approved advancement to Phase II Non-confidential Slide 17

DYNAMIC: Phase I a/b Trial of CDCs Dilated cardiomYopathy iNtervention with Allogeneic MyocardIally- regenerative Cells Study Overview  IND Sponsor: Cedars-Sinai Medical Center (E. Marbán)  Patient criteria o Ischemic or non- ischemic DCM with LVEF ≤ 35% o NYHA Class III or ambulatory Class IV heart failure o Hospitalization for heart failure within 12 months  Delivery: Non-occlusive intracoronary infusion in 2-3 vessels  Trial Design o Phase Ia: open-label (N=14, single center): safety o Phase Ib: randomized, double-blinded placebo-controlled (n=28, 4 centers): safety and exploratory efficacy Non-confidential Slide 18