Capricor Collaborates with Lonza for the Development of CAP-1002, its Cell Therapy Candidate for the Treatment of Duchenne Muscular Dystrophy and Other Indications

—Capricor to leverage Lonza’s expertise in technology transfer and development of cellular therapies to take CAP-1002 through potential product launch and commercial supply—

—The collaboration aims to expand Capricor’s manufacturing capacity for potential late-stage clinical trials and commercialization—

—Process development to take place in Lonza’s Houston (TX) center of excellence—

LOS ANGELES and BASEL, Switzerland, Jan. 12, 2021 (GLOBE NEWSWIRE) -- Capricor Therapeutics, (NASDAQ: ), a clinical-stage biotechnology company focused on the development of the first-in-class cell and exosome-based therapeutics for the treatment and prevention of serious diseases, and Lonza today announced that the companies have entered into an agreement for the development of CAP-1002, its leading clinical asset using allogeneic cardiosphere-derived cells (CDC) technology for the treatment of Duchenne Muscular Dystrophy (DMD) and complications arising from COVID-19.

“As we continue to expand our manufacturing efforts for CAP-1002, our lead cell therapy product candidate, this collaboration with Lonza provides us with a partner which has world-class expertise in technology transfer and an established track record of commercializing biologics,” said Linda Marbán, Chief Executive Officer of Capricor. “We are excited because this is an important step in our ability to potentially bring CAP-1002 closer to commercialization and allows us to bring this important therapy to patients with Duchenne muscular dystrophy as quickly as possible, if approved.”

CAP-1002 completed the positive HOPE-2 phase 2 clinical trial and has been granted orphan drug designation by the FDA for the treatment of DMD. CAP-1002 also received FDA acceptance of its IND application for a phase 2 clinical trial of CAP-1002 in patients with COVID-19 in August 2020, as .

“Capricor’s lead candidate CAP-1002 is demonstrating efficacy in late-stage clinical studies to significantly benefit patients,” said Alberto Santagostino, SVP, Head of Cell and Gene Technologies, Lonza. “We will leverage our process development expertise and industrial manufacturing capabilities to enable Capricor to scale this therapy and make it available to patients globally, once approved for commercialization.”

The agreement aims to expand Capricor’s manufacturing capacity for potential late-stage clinical trials and commercialization. Operations will begin with a tech-transfer to Lonza’s Houston (TX) center of excellence, where Lonza will perform process development activities for late-clinical and commercial-scale GMP manufacturing of CAP-1002.

About Lonza

At Lonza, we combine technological innovation with world class manufacturing and process excellence. Together, these enable our customers to deliver their discoveries in the healthcare, preservation, and protection sectors.

We are a preferred global partner to the pharmaceutical, biotech and specialty ingredients markets. We work to prevent illness and promote a healthier world by enabling our customers to deliver innovative medicines that help treat or even cure a wide range of diseases. We also offer a broad range of microbial control solutions, which help to create and maintain a healthy environment.

Founded in 1897 in the Swiss Alps, Lonza today operates in 120 sites and offices in more than 35 countries. With approximately 15,500 full-time employees, we are built from high-performing teams and of individual employees who make a meaningful difference to our own business, as well as the communities in which we operate. The company generated sales of CHF 5.9 billion in 2019 with a CORE EBITDA of CHF 1.6 billion. Find out more at and follow us on Twitter @LonzaGroup or Facebook @LonzaGroupAG.

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class cell and exosome-based therapeutics for the treatment and prevention of diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy and COVID-19. Capricor is also investigating the field of extracellular vesicles and exploring the potential of exosome-based candidates to treat or prevent a variety of disorders. Capricor is now developing two potential vaccines for COVID-19 as part of its exosome platform. For more information, visit and follow the Company on Facebook, Instagram and Twitter.

About CAP-1002

CAP-1002 consists of allogeneic cardiosphere-derived cells, or CDCs, a type of cardiac cell therapy that has been shown in pre-clinical and clinical studies to exert potent immunomodulatory activity. It is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The cells function by releasing exosomes that are taken up largely by macrophages and T-cells and begin a cycle of repair. CDCs have been the subject of over 100 peer-reviewed scientific publications and administered to over 200 human subjects across several clinical trials.

Lonza Contact Details

Dr. Sanna Fowler

Head of External Communications

Lonza Group Ltd

Tel

 

Dirk Oehlers

Investor Relations

Lonza Group Ltd

Tel

 

Capricor Contact Details

Media:

Caitlin Kasunich

KCSA Strategic Communications

  

Tel 212.896.1241

Investor:

Joyce Allaire

LifeSci Advisors, LLC

  

Tel 617.435.6602

Company:

AJ Bergmann, Chief Financial Officer

  

310.358.3201

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited (“SGX-ST”). Lonza Group Ltd is not subject to the SGX-ST’s continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

Cautionary Note Regarding Forward-Looking Statements for Capricor

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 process development and manufacturing; 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, 2019 as filed with the Securities and Exchange Commission on March 27, 2020 and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 as filed with the Securities and Exchange Commission on November 13, 2020. 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.