Capricor Therapeutics Reports Topline Results from Phase 2 Exploratory INSPIRE Trial in Severe Hospitalized COVID-19 Patients

-Primary Objective of Study Met: CAP-1002 was Safe and Well Tolerated-

SAN DIEGO, March 28, 2022 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics, announced today that its Phase 2, INSPIRE study evaluating a single-dose intravenous infusion of CAP-1002 as a potential treatment option for hospitalized patients with advanced symptoms of COVID-19 met its primary objective of safety. All efficacy endpoints were exploratory as the study was not powered to detect treatment differences. In the study of 63 randomized patients, 31% were admitted to the ICU prior to initiation of treatment. The WHO ordinal scale indicated severe disease in 82% of patients (range: 0-8, median 5).

Study Findings

• Safety: The results from the INSPIRE trial suggest that CAP-1002 was safe, well tolerated and consistent with the historically observed safety profile of this therapy.
• Exploratory Efficacy: Overall mortality in the study was 20%, with 6 deaths in the placebo group and 5 deaths in the CAP-1002 group. (n=11; p=NS)

“We were pleased that CAP-1002 was shown to be safe and well-tolerated in this group of patients that were profoundly ill with symptoms of COVID-19 which bodes well for the general safety and tolerability of infused CAP-1002 even in this heterogeneous and severely sick population of patients,” said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. “In light of the effectiveness and widespread use of available vaccines, the reduction in mortality and the decline in COVID-19 hospitalization rates in the United States, we will continue to evaluate the next steps for this program. Further, as we continue to analyze this dataset in more detail, we will remain open to discussing potential partnering opportunities should they become available.”

About INSPIRE

The INSPIRE trial is a Phase 2, randomized, double-blind, placebo-controlled study that enrolled patients with a clinical diagnosis of COVID-19 confirmed by laboratory testing and are in severe to critical condition requiring supplemental oxygen. The study was conducted at multiple sites in the United States and randomized 63 patients. The primary objective of the study was to evaluate the safety of a single-dose of intravenously infused CAP-1002 as well as evaluate multiple exploratory efficacy outcomes. Eligible patients were randomized to either the CAP-1002 or placebo group (1:1 ratio) and underwent baseline safety and efficacy assessments out to 90 days following infusion.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 patients across several clinical trials.

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 capable of delivering nucleic acids, including mRNA as well as proteins, to treat or prevent a variety of diseases. For more information, visit www.capricor.com, and follow the Company on Facebook, Instagram and Twitter.

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; the ability to achieve product milestones and to receive milestone payments from commercial partners; 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, 2021, as filed with the Securities and Exchange Commission on March 11, 2022. 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:

Capricor Media Contact:
Raquel Cona
KCSA Strategic Communications
rcona@kcsa.com
212.896.1204

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

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

Released March 28, 2022