Capricor Therapeutics Presents at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
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Insights
The recent announcement by Capricor Therapeutics regarding the HOPE-2 study results for CAP-1002 in treating Duchenne muscular dystrophy (DMD) presents significant clinical advancements. The data indicating that CAP-1002 can potentially slow disease progression by 64% and improve cardiac function in 67% of patients is a promising development in the field of rare disease treatment. These results are particularly notable given the lack of effective treatment options currently available for DMD, a condition characterized by rapid progression and early mortality.
From a research perspective, the use of CAP-1002, which operates through immunomodulatory, anti-inflammatory and anti-fibrotic actions, may represent a breakthrough in addressing both skeletal and cardiac muscle decline in DMD. The 4.9-point difference in the Performance of the Upper Limb (PUL v2.0) scale between treated patients and the placebo group, with a p-value of 0.021, suggests a clinically meaningful impact on patients' quality of life and ability to perform daily activities.
Furthermore, the consistent safety profile over 24 months of treatment supports the potential for long-term administration of CAP-1002, which is critical for chronic conditions such as DMD. As the company moves towards discussions with the FDA regarding expedited approval pathways, the robustness of these results will be under scrutiny, particularly the durability of the treatment effect and its translation into tangible patient benefits.
The positive results from Capricor's HOPE-2 study could have substantial implications for the company's financials and investor sentiment. As a biotechnology firm focused on rare diseases, Capricor operates in a high-risk but potentially high-reward sector, where successful clinical trial outcomes can lead to significant stock price movements and partnership opportunities.
The announcement of the 24-month data could act as a catalyst for Capricor's stock (NASDAQ: CAPR), as it not only demonstrates sustained efficacy but also strengthens the case for expedited regulatory pathways. Such pathways could lead to earlier market entry and revenue generation for CAP-1002. Additionally, the data might attract potential partnerships or buyout interest from larger pharmaceutical companies seeking to expand their rare disease portfolios.
Investors will likely monitor the upcoming presentation of three-year results and any subsequent regulatory developments closely. The company's ability to secure FDA approval and subsequently commercialize CAP-1002 will be pivotal in determining its financial trajectory. However, it is essential to note that the biotech sector is volatile and investment risks remain high until regulatory approval is secured and commercialization is achieved.
The economic implications of Capricor's CAP-1002 for the treatment of DMD are multifaceted. On one hand, the potential to slow disease progression and improve cardiac function could reduce the long-term healthcare costs associated with managing DMD, a disease that often requires intensive medical care, including surgeries and cardiac interventions.
Should CAP-1002 gain FDA approval, it may command a premium price due to its orphan drug status and the lack of effective treatments for DMD. The cost-effectiveness of the therapy will be a point of analysis, considering the direct treatment costs against the potential to reduce secondary healthcare expenditures and improve patient quality of life.
It is also worth considering the broader economic impact, such as the potential for patients to maintain a higher degree of independence for longer periods, which has implications for caregivers and the labor market. The societal value of innovative treatments like CAP-1002 extends beyond immediate healthcare savings, encompassing the enhancement of patients' lives and the potential to contribute economically and socially.
Presentation Highlights Positive 24-Month Results from HOPE-2 Open Label Extension Study of CAP-1002 in Duchenne Muscular Dystrophy
SAN DIEGO, March 06, 2024 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment and prevention of rare diseases, announced today that the Company will present the positive 24-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 MDA Clinical and Scientific Conference which is taking place in Orlando, Florida from March 3-6, 2024.
Key results from the study, include:
- CAP-1002 is an investigational cell therapy which aims to slow disease progression through immunomodulatory, anti-inflammatory and anti-fibrotic actions with the goal of potentially improving the rate of decline in skeletal and cardiac muscle function in patients with DMD.
- Skeletal muscle function as measured by the Performance of the Upper Limb (PUL v2.0) showed a mean PUL v2.0 decline after 24-months of treatment with CAP-1002 was 2.8 points versus a 7.7 point decline on average observed over 24-months in the placebo patient group.
- Delta change=4.9 points; p=0.021
- The average rate of decline in CAP-1002 treated patients showed an attenuation of disease progression by approximately
64%
- CAP-1002 revealed clinically meaningful improvements in ameliorating cardiac function.
- Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in
67% of patients, compared to a steady decline in a comparable natural history population
- Cardiac function as measured by left ventricular ejection fraction (LVEF%) by MRI at the 24-month timepoint, improved in
- CAP-1002 treatment during the OLE portion of the study continues to yield a consistent favorable 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 24-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.
“We are pleased to be presenting our positive long-term open label extension data at this year’s MDA Conference,” said Linda Marbán, Ph.D., chief executive officer of Capricor. “DMD is a progressive disease associated with loss of function over time and there is a critical need for treatment options to slow the rate of decline and preserve upper limb function to enable key activities of daily living. These two-year results suggest that CAP-1002 has the potential to slow the decline of DMD progression and speaks to the potential long-term benefit for patients. At this time, as these patients enter their fourth year of CAP-1002 continuous treatment, we plan to discuss options for potential expedited approval pathways with the FDA and in connection with these efforts, look forward to sharing the three-year results in the second quarter of this year.”
Podium Presentation in Clinical Trial Updates Session
| Title: | Long term safety and efficacy of CAP-1002 in late-stage patients with DMD: A new treatment approach to target skeletal and cardiac muscle pathogenesis |
| Date: | Wednesday, March 6, 2024, 11:45 a.m. – 12:00 p.m. ET |
For more information on the MDA conference, please visit www.mdaconference.org.
About the 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. 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 24-months. These 24-month results were presented previously at the 2023 Parent Project Muscular Dystrophy (PPMD) Annual Conference.
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 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 and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2023, as filed with the Securities and Exchange Commission on November 14, 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.
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 Officer
abergmann@capricor.com
858.727.1755
FAQ
What are the key results from the HOPE-2 open-label extension study of CAP-1002 in Duchenne Muscular Dystrophy?
How did skeletal muscle function change after 24 months of treatment with CAP-1002?
What improvements were seen in cardiac function with CAP-1002 treatment?
What is the safety profile of CAP-1002 in the HOPE-2 OLE study?
What is the significance of the 24-month results for CAP-1002 in DMD treatment?
