Capricor Therapeutics reported a 52% slowing of disease progression in DMD patients treated with deramiocel over three years.
Quiver AI Summary
Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can significantly slow the progression of Duchenne muscular dystrophy (DMD). Over three years, patients treated with deramiocel experienced a 52% decrease in the decline of upper limb function compared to a matched external group. The findings highlight a consistent reduction in disease progression over time and demonstrate a favorable safety profile for the treatment. Capricor's CEO emphasized the importance of these results for enhancing the quality of life for patients with DMD. The company is seeking full FDA approval for deramiocel, which targets the cardiomyopathy associated with DMD, with a decision expected by August 31, 2025.
Potential Positives
- Positive data from the HOPE-2 clinical trial shows a 52% slowing of disease progression in patients with Duchenne muscular dystrophy (DMD), indicating the potential long-term efficacy of deramiocel.
- The data presentation at the MDA Conference enhances the visibility and credibility of Capricor Therapeutics in the medical community, potentially attracting interest from investors and partners.
- The acceptance of the Biologics License Application (BLA) by the FDA for deramiocel indicates progress towards regulatory approval, which could significantly impact the company's market position and revenue potential.
- Deramiocel demonstrates a favorable safety profile, with no new safety signals identified, reinforcing its therapeutic viability and potential for future use in clinical settings.
Potential Negatives
- The positive results presented may not be sufficient to guarantee regulatory approval from the FDA, as indicated by the upcoming Prescription Drug User Fee Act target action date of August 31, 2025.
- There are inherent risks associated with the forward-looking statements, implying uncertainty about the drug's future performance and market acceptance.
- Deramiocel remains an investigational drug and has not yet received approval for any indications, highlighting the ongoing challenges in bringing the product to market.
FAQ
What is deramiocel and its role in DMD treatment?
Deramiocel is Capricor's lead asset, an allogeneic cell therapy that aims to slow disease progression in Duchenne muscular dystrophy (DMD).
How effective is deramiocel in slowing DMD progression?
Deramiocel achieved a 52% reduction in disease progression over three years, as indicated by the Performance of the Upper Limb (PUL 2.0) scores.
What were the safety outcomes for deramiocel?
Deramiocel was well tolerated with no new safety signals, maintaining a favorable long-term benefit-risk profile throughout the study.
When is the FDA decision expected for deramiocel?
The FDA's target action date for Capricor's Biologics License Application for deramiocel is set for August 31, 2025.
Where can I find the study's poster presentation?
The poster presentation will be available on the publications section of Capricor's website following the MDA conference.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
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Full Release
--Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease--
--Data Presented at the 2025 Muscular Dystrophy Association (MDA) Conference--
SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) --
Capricor Therapeutics
(NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, today announced positive long-term data from its ongoing HOPE-2 open label extension (“OLE”) clinical trial, demonstrating the potential of the Company’s lead asset, deramiocel, to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”). The data is presented as a late breaking poster at this year’s
Muscular Dystrophy Association Clinical and Scientific Conference
, which began on March 16 and runs through March 19 in Dallas, Texas.
In a cohort-matched external comparator analysis, the study showed that patients treated with deramiocel over three years experienced an average decline in Performance of the Upper Limb (PUL 2.0) total score of 3.46 points, compared to a 7.19-point decline in the external comparator group (p=0.019). This equates to a 52 percent slowing of disease progression, reinforcing deramiocel’s potential long-term therapeutic durability.
Additional findings include:
Treatment effect increases year over year –
Patients on deramiocel showed a reduction in disease progression, with a mean annual PUL 2.0 decline of 1.8 points in Year 1, 1.2 points in Year 2 and 1.1 points in Year 3.
Potential disease-modifying effects
– During a 1-year gap of treatment, those originally randomized to deramiocel showed a slower rate of decline (2.8 points per year) compared to untreated patients (3.7 points per year).
Favorable safety profile
– Deramiocel was well tolerated with no new safety signals identified and continues to maintain a favorable long-term benefit-risk profile.
“For patients and families battling DMD, time is muscle. This data showing long term preservation of skeletal muscle function reinforces that deramiocel is not just slowing the disease—it is rewriting the trajectory of what’s possible for those living with Duchenne,” said Linda Marbán, Ph.D., CEO of Capricor. “We have continued to see durable, long-term benefits, enabling patients to maintain skeletal muscle and cardiac function when decline was once inevitable.”
Capricor recently announced the acceptance by the U.S. Food and Drug Administration (“FDA”) of its Biologics License Application (“BLA”) for the cardiomyopathy associated with DMD with a Prescription Drug User Fee Act (“PDUFA”) target action date set for August 31, 2025, seeking full approval of its application.
A copy of the poster presentation will be made available on the
publications
section of the Capricor website following the meeting.
About Deramiocel
Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (“CDCs”), a population of stromal cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and antifibrotic actions in preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. CDCs act by secreting extracellular vesicles known as exosomes, which target macrophages and alter their expression profile so that they adopt a healing, rather than a pro-inflammatory, phenotype. CDCs have been the subject of over 100 peer-reviewed scientific publications and have been administered to over 200 human subjects across several clinical trials.
About Duchenne Muscular Dystrophy
DMD is a devastating genetic disorder characterized by progressive weakness and chronic inflammation of the skeletal, heart and respiratory muscles with mortality at a median age of approximately 30 years. It is estimated that DMD occurs in approximately one in every 3,500 male births and that the patient population is estimated to be approximately 15,000-20,000 in the United States. DMD pathophysiology is driven by the impaired production of functional dystrophin, which normally functions as a structural protein in muscle. The reduction of functional dystrophin in muscle cells leads to significant cell damage and ultimately causes muscle cell death and fibrotic replacement. In DMD patients, heart muscle cells progressively die and are replaced with scar tissue. This cardiomyopathy eventually leads to heart failure, which is currently the leading cause of death among those with DMD. Treatment options are limited and there is no cure.
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, deramiocel, an allogeneic cardiac-derived cell therapy. Extensive preclinical and clinical studies have shown deramiocel to exert potent immunomodulatory and antifibrotic actions in preservation of cardiac and skeletal muscle function in dystrophiopathies such as DMD. Deramiocel is currently in late-stage development for the treatment of Duchenne muscular dystrophy. Capricor is also harnessing the power of its exosome technology, using its 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
,
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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; potential future agreements; 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, and in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the Securities and Exchange Commission on November 14, 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 an agreement for the exclusive commercialization and distribution of deramiocel (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. Deramiocel 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 Company Contact:
AJ Bergmann, Chief Financial Officer
abergmann@capricor.com
858.727.1755
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