Capricor Therapeutics to Present Final Data from HOPE-2 Trial with CAP-1002 in Duchenne Muscular Dystrophy at World Muscle Society 2021

-Principal Investigator Dr. Craig McDonald Will Deliver Late-Breaking Oral Presentation, One of Only Three Selected by WMS-

LOS ANGELES, Sept. 02, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR) (“Capricor” or “the Company”), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, announced today that the Company will present final data from its HOPE-2 clinical trial with asset CAP-1002 at this year’s World Muscle Society Virtual Congress (WMS). HOPE-2 is Capricor’s randomized, double-blind, placebo-controlled, Phase II clinical trial in 20 patients with late-stage Duchenne muscular dystrophy (DMD), conducted at nine sites in the U.S. The late-breaking results will be presented in an oral session on Sept. 24, 2021, from 4:15 to 5 p.m. BST (11:15 a.m. to 12 p.m. EST).

We are pleased to be presenting the final data from our HOPE-2 clinical trial at this year’s World Muscle Society Meeting,” said Dr. Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “While we were encouraged by our topline data presented last year, we believe that the final data is even more supportive of CAP-1002’s effect on DMD’s progression in non-ambulant patients. While gene therapies show promise for improving DMD’s outcomes, this treatment paradigm, especially in older patients, who suffer from more advanced disease, will likely require a multi-pronged approach. This is where CAP-1002 may fit in with its immunomodulatory, anti-fibrotic mechanism of action that promotes skeletal and cardiac muscle regeneration.”

Eighty percent of patients in Capricor’s HOPE-2 trial were non-ambulant, and the average age of participants was approximately 14 years. The primary efficacy endpoint was measured by the Performance of the Upper Limb (PUL) 1.2 mid-level, and all CAP-1002 patients were on standard of care medications, which includes corticosteroids. Patients with DMD typically do not see function restoration or preservation beyond what is mediated by steroids.

Dr. Craig McDonald, Capricor’s Principal Investigator for the HOPE-2 study, will be available to discuss the data following his oral presentation in a live Q&A session.

Dr. McDonald is a professor of pediatrics, professor and chair of the Department of Physical Medicine and Rehabilitation, director of Rehabilitation Services and director of the Neuromuscular Disease Clinics at UC Davis Health. He has served as a principal investigator for more than 30 industry-sponsored trials for DMD and is the study chair for the Duchenne Natural History Study of the Cooperative International Neuromuscular Research Group, a consortium of medical and scientific investigators from academic and research centers who share the common goal of aiming to positively impact the lives of neuromuscular disease patients and their families by conducting well-controlled clinical studies. 

Capricor’s presentation will be made available to conference attendees. The World Muscle Society Virtual Congress will take place virtually September 20-24, 2021.

About Capricor Therapeutics
Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on developing transformative cell and exosome-based therapeutics and vaccines for treating and preventing a broad spectrum of diseases. Capricor's lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in clinical development for treating Duchenne muscular dystrophy and the cytokine storm associated with COVID-19. Capricor is also developing its exosome technology as a next-generation therapeutic platform. The Company’s current focus is on developing exosomes loaded with nucleic acids, including mRNA, to treat or prevent a variety of diseases. For more information, visit www.capricor.com, and follow the Company on Facebook, Instagram and Twitter.

About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles. Patients suffering from DMD typically lose their ability to walk in their teenage years and generally die of cardiac or respiratory complications by age 30. It occurs in one in every 3,600 live male births across all races, cultures and countries. DMD afflicts approximately 200,000 boys and young men around the world. Treatment options are limited, and there is no cure.

Cautionary Note Regarding Forward-Looking Statements
Statements in this press release regarding the efficacy, safety, and intended utilization of Capricor's product candidates; the initiation, conduct, size, timing and results of discovery efforts and clinical trials; the pace of enrollment of clinical trials; plans regarding regulatory filings, future research and clinical trials; regulatory developments involving products, including the ability to obtain regulatory approvals or otherwise bring products to market; plans regarding current and future collaborative activities and the ownership of commercial rights; scope, duration, validity and enforceability of intellectual property rights; future royalty streams, revenue projections; expectations with respect to the expected use of proceeds from the recently completed offerings and the anticipated effects of the offerings; and any other statements about Capricor's management team's future expectations, beliefs, goals, plans or prospects constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not statements of historical fact (including statements containing the words "believes," "plans," "could," "anticipates," "expects," "estimates," "should," "target," "will," "would" and similar expressions) should also be considered to be forward-looking statements. 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 is set forth in Capricor's Annual Report on Form 10-K for the year ended December 31, 2020 as filed with the Securities and Exchange Commission on March 15, 2021 and in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2021 as filed with the Securities and Exchange Commission on August 13, 2021. All forward-looking statements in this press release are based on information available to Capricor as of the date hereof, and Capricor assumes no obligation to update these forward-looking statements.

CAP-1002 is an Investigational New Drug and is not approved for any indications. None of Capricor’s exosome-based candidates have been approved for clinical investigation.

For more information, please contact:

Media Contact:
Caitlin Kasunich / Raquel Cona
KCSA Strategic Communications
ckasunich@kcsa.com / rcona@kcsa.com
212.896.1241 / 212.896.1204

Investor Contact:
Joyce Allaire
LifeSci Advisors, LLC
jallaire@lifesciadvisors.com
617.435.6602

Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
310.358.3200

Released September 2, 2021