Capricor Therapeutics Presents at 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Rhea-AI Summary

Capricor Therapeutics (NASDAQ: CAPR) reported promising 18-month results from its HOPE-2 open-label extension study for CAP-1002, aimed at treating Duchenne muscular dystrophy (DMD). Presented by Dr. Craig McDonald at the Muscular Dystrophy Association Clinical & Scientific Conference, the findings show statistically significant improvements in the Performance of the Upper Limb (PUL version 2.0) scale (p=0.02) compared to the placebo group. Both groups showed reduced disease progression after starting treatment. CAP-1002 maintained a good safety profile, and the study indicates potential long-term benefits for patients. The company is advancing to the HOPE-3 Phase 3 trial.

Positive

• Significant improvement in Upper Limb function (PUL scale) with CAP-1002 (p=0.02).
• Consistent safety and tolerability profile of CAP-1002 throughout the study.
• HOPE-2 results suggest long-term benefits in preserving muscle function in DMD patients.

Negative

• None.

Late-Breaking Presentation Highlighted Positive18-Month Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy Patients

SAN DIEGO, March 23, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, announced today that the Company presented the 18-month results from its HOPE-2 open-label extension (OLE) study with lead asset, CAP-1002, for the treatment of Duchenne muscular dystrophy (DMD) at this year’s Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which took place virtually and in-person in Dallas, Texas from March 19-22, 2023. Dr. Craig McDonald, national Principal Investigator and University of California, Davis, Professor and Department of Physical Medicine and Rehabilitation Chair, presented the findings during a late-breaking session.

Key results from the study, include:

• Statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group when compared to the original placebo group from HOPE-2 (p=0.02). 
• Both groups experienced reduced disease progression once all patients began treatment in the OLE study.
• CAP-1002 treatment during the OLE portion of the study continues to yield a consistent safety profile and has been well-tolerated throughout the study.
• The HOPE-2 OLE study previously met the primary endpoint at the one-year time-point and these 18-month results suggest that patients accumulate benefit over time with preservation of skeletal muscle function, which underscore the potential long-term benefit of CAP-1002.

“There are thousands of boys and young men across the world who continue to face the devastating and immobilizing effects of DMD, and for whom few therapeutic options currently exist,” said Dr. Linda Marbán, CEO of Capricor. “Our 18-month results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the long-term benefit for patients. We were pleased to present these results alongside other industry leaders who share Capricor’s mission of developing novel treatments for patients suffering from rare neuromuscular conditions.”

Capricor is currently conducting the HOPE-3, Phase 3 trial, designed as a randomized, double-blind, placebo-controlled study with enrollment criteria similar to HOPE-2. The Phase 3 study is currently enrolling subjects (NCT05126758).

For more information on the MDA conference, please visit www.mdaconference.org.

About HOPE-2 Open Label Extension (OLE) Study

HOPE-2 was a randomized, double-blind, placebo-controlled, Phase 2 clinical study of Capricor’s lead investigational therapy, CAP-1002, in boys and young men who have DMD and are non-ambulant, the later stage of the disease process. The study was conducted at nine sites across the United States. Study patients were treated via intravenous delivery with either CAP-1002 (150 million cells per infusion) or placebo every 3 months. Data from a total of 20 patients was analyzed (12 placebo and 8 treated) at the 12-month time-point and the results were published in The Lancet.

After the completion of the HOPE-2 study, all patients stopped treatment for approximately 392 days (mean, range [239, 567]), which is referred to as the gap phase. Then all eligible patients who wished to remain on treatment re-entered the OLE study where they received CAP-1002 (150 million cells per infusion) every three months over the course of 18 months. Patients continued through the gap phase (off treatment for both groups) and the OLE Phase (on treatment for both groups).

Patients in the study were evaluated using the Performance of the Upper Limb (PUL 2.0), a validated tool specifically designed for assessing high (shoulder), mid (elbow) and distal (wrist and hand) function, with a conceptual framework reflecting the progression of weakness in upper limb function.

About Duchenne Muscular Dystrophy (DMD)

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. 

About Capricor Therapeutics

Capricor Therapeutics, Inc. (NASDAQ: CAPR) is a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases. Capricor’s lead candidate, CAP-1002, is an allogeneic cardiac-derived cell therapy that is currently in late-stage clinical development for treating Duchenne muscular dystrophy. Capricor is also developing its exosome technology as a next-generation therapeutic platform. Capricor’s 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 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; 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 and 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, 2022, as filed with the Securities and Exchange Commission on March 17, 2023. 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

FAQ

What are the latest results for CAPR's CAP-1002 in DMD treatment?

Capricor reported 18-month results showing significant improvements in DMD patients' muscle function compared to placebo, indicating potential long-term benefits.

When was the HOPE-2 study presented?

The results of the HOPE-2 study were presented on March 23, 2023, at the Muscular Dystrophy Association Clinical & Scientific Conference.

What is the next step for Capricor after HOPE-2?

Capricor is moving forward with the HOPE-3 Phase 3 trial for CAP-1002, aiming for broader clinical validation.

What is Duchenne muscular dystrophy (DMD)?

Duchenne muscular dystrophy is a severe genetic disorder causing progressive muscle weakness and inflammation, primarily affecting boys and young men.