Safety of Allogeneic Cardiosphere-Derived Cells for Dilated Cardiomyopathy

DESCRIPTION provided by applicant Project Abstract Capricor Inc has an allogeneic cell therapy product CAP presently in clinical development CAP consists of cardiosphere derived cells CDCs grown from a donor unrelated to the recipient using a proprietary process exclusively licensed to Capricor Autologous CDCs have demonstrated their utility as a regenerative agent able to reduce infarct size a k a scar size add viable tissue mass and attenuate adverse left ventricular LV remodeling when administered to patients several months after a myocardial infarction MI The magnitude of benefit seen in CDC treated patients could foreshadow a reduction in future adverse clinical events Allogeneic CDCs are currently being tested in a similar post MI population in a randomized double blinded placebo controlled multi center safety and efficacy study The current proposal aims to test the safety of CAP administered in the acute MI setting adjunctive to the gold standard treatment i e PCI percutaneous coronary intervention rapidly after restoration of blood flow or reperfusion as a cardioprotective agent Capricor has an approved IND Investigational New Drug application for the use of CAP in the treatment and or prevention of LV dysfunction following MI and the proposed clinical trial falls under the umbrella of this indication Preclinial data support the safe use of CAP in acute MI and reveal the productandapos s ability to limit infarct size and reduce acute myocardial damage CAP is manufactured in an existing GMP Good Manufacturing Practice compliant facility and will be stored at the clinical site for immediate availability CAP is formulated such that it can be prepared for use within minutes and administered within minutes Patients will be treated open label with CAP within hours of successful reperfusion Safety endpoints will include assessments of blood flow in the coronary arteries and microvasculature resolution of myocardial damage immune reaction arrhythmias and MACE major adverse cardiac events Efficacy assessments will examine infarct size LV volumes and ejection fraction by MRI magnetic resonance imaging Funding for a follow on trial that would demonstrate efficacy will be pursued concurrently Capricor is well positioned to secure financing for the next study particularly with an ongoing multi center study utilizing the same product in a complementary patient population The company has a sound regulatory strategy and advisors in place to guide interactions with the FDA Food and Drug Administration as CAP is advanced toward approval The current manufacturing scale and facility will suffice through early commercialization and plans to scale up are underway Capricor has identified several potential avenues by which to enter the public market and maintains active business development efforts The company anticipates being revenue generating within a few years of project completion The ongoing and proposed clinical trial series encompass nearly the full continuum of disease states following MI CAP developed successfully for all MI patients would offer both a cardioprotective and regenerative therapy to be administered as needed PUBLIC HEALTH RELEVANCE Project Narrative Cardiovascular disease is the No cause of death in the United States and myocardial infarction MI affects more than million Americans every year Cardiosphere derived cells CDCs will be tested as a safe and effective therapy when administered immediately after MI in the hopes of limiting the amount of damage caused by the event lowering the number of adverse clinical events experienced in the future and reducing the population of Americans living with and dying from heart failure after MI