CAPR Capricor Therapeutics Inc

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced that an abstract featuring new preclinical data highlighting the therapeutic potential of its StealthX™ exosome platform technology has been selected for an oral presentation at the ASGCT 27th Annual Meeting taking place in Baltimore, Maryland from May 7–11, 2024. The findings highlight a potential exosome-based approach for the treatment of arginase-1 deficiency (ARG1-D), a rare genetic metabolic disease characterized by complete or partial lack of the enzyme arginase in the liver and red blood cells.

“The findings from this preclinical study have further characterized our StealthX™ exosome platform and provide a novel approach for exosome-based enzyme-replacement therapies which aim to replace a deficient or absent enzyme,” said Linda Marbán, Ph.D., Capricor’s chief executive officer. “We believe our StealthX™ platform provides an opportunity with potentially broad applications and our data continues to support the concept of exosomes as a suitable delivery vehicle for a variety of payloads. We continue to focus our exosome platform in two main areas, the development of therapeutics and vaccines, and our goal is to leverage partnership opportunities to further expand and advance these opportunities.”

Study Overview and Findings:

• In this study, exosomes were engineered to express human Arg1 enzyme inside of the exosomes and were evaluated for their in vitro functionality as an approach for the potential treatment of ARG1-D.
• Results showed the Arg1-exosomes were enzymatically active and able to convert arginine into urea in vitro. In addition, the Arg1-exosomes were capable of delivering the Arg1 protein into 293F and HepG2 cells in a time and dose-dependent manner, contrary to human recombinant Arg1 protein alone at the same or higher dose.
• The data suggests that the Arg1-exosomes were able to not only catalyze arginine efficiently at a lower delivered dose to cells in vitro but also protect the Arg1 protein to be delivered into target cells and retain its enzymatic activity in cells.
• Taken together, Capricor’s StealthX™ platform of engineered Arg1-exosomes as tested in vitro have the potential to serve as an enzyme replacement therapy to deliver Arg1 to hepatocytes and possibly have potential clinical benefits for the treatment of ARG1-D.

Presentation details:
Title:		Exosome-mediated intracellular delivery of arginase-1: a potential application for the treatment of arginase-1 deficiency
Oral session:		Exosomes, Virus like Particles and LNPs
Presenting author:		Li-En Hsieh, Ph.D., Principal Scientist
Date:		May 9, 2024 at 4:45 p.m. ET
Abstract number:		223

The abstract will be made available on the publications section of the Capricor website following the conclusion of the meeting.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company dedicated to advancing transformative cell and exosome-based therapeutics to redefine the treatment landscape for rare diseases. At the forefront of our innovation is our lead product candidate, CAP-1002 — an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown CAP-1002 to demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. CAP-1002 is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy (DMD). Capricor is also harnessing the power of our exosome technology, using our proprietary StealthX™ platform in preclinical development focused on the areas of vaccinology, targeted delivery of oligonucleotides, proteins and small molecule therapeutics to potentially treat and prevent a diverse array of diseases. At Capricor, we stand committed to pushing the boundaries of possibility and forging a path toward transformative treatments for those in need. For more information, visit capricor.com, and follow Capricor 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; manufacturing capabilities; dates for regulatory meetings; statements about our financial outlook; 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 revenue streams and 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, 2023, as filed with the Securities and Exchange Commission on March 11, 2024. 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.

Capricor has entered into a partnership for the exclusive commercialization and distribution of CAP-1002 for DMD in the United States and Japan with Nippon Shinyaku Co., Ltd. (U.S. subsidiary: NS Pharma, Inc.), subject to regulatory approval. 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 Company Contact:AJ Bergmann, Chief Financial Officerabergmann@capricor.com 858.727.1755