Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy
Capricor Therapeutics (NASDAQ: CAPR) announced positive long-term data from its HOPE-2 open label extension trial for deramiocel, showing significant efficacy in treating Duchenne muscular dystrophy (DMD). The data, presented at the 2025 MDA Conference, demonstrated a 52% slowdown in disease progression over three years.
Key findings show patients treated with deramiocel experienced an average decline of 3.46 points in Performance of the Upper Limb (PUL 2.0) total score, compared to 7.19 points in the control group (p=0.019). The treatment effect improved yearly, with mean annual PUL 2.0 declines of 1.8, 1.2, and 1.1 points in Years 1, 2, and 3 respectively.
The drug maintained a favorable safety profile with no new safety signals. The FDA has accepted Capricor's Biologics License Application for DMD-associated cardiomyopathy, with a PDUFA date set for August 31, 2025.
Capricor Therapeutics (NASDAQ: CAPR) ha annunciato dati positivi a lungo termine dal suo studio di estensione open label HOPE-2 per deramiocel, mostrando un'efficacia significativa nel trattamento della distrofia muscolare di Duchenne (DMD). I dati, presentati alla Conferenza MDA 2025, hanno dimostrato un 52% di rallentamento nella progressione della malattia nel corso di tre anni.
I risultati chiave mostrano che i pazienti trattati con deramiocel hanno sperimentato un declino medio di 3.46 punti nel punteggio totale della Performance of the Upper Limb (PUL 2.0), rispetto a 7.19 punti nel gruppo di controllo (p=0.019). L'effetto del trattamento è migliorato annualmente, con declini medi annuali di PUL 2.0 di 1.8, 1.2 e 1.1 punti negli Anni 1, 2 e 3 rispettivamente.
Il farmaco ha mantenuto un profilo di sicurezza favorevole senza nuovi segnali di sicurezza. La FDA ha accettato la domanda di licenza biologica di Capricor per la cardiomiopatia associata alla DMD, con una data PDUFA fissata per il 31 agosto 2025.
Capricor Therapeutics (NASDAQ: CAPR) anunció datos positivos a largo plazo de su ensayo de extensión open label HOPE-2 para deramiocel, mostrando una eficacia significativa en el tratamiento de la distrofia muscular de Duchenne (DMD). Los datos, presentados en la Conferencia MDA 2025, demostraron un 52% de desaceleración en la progresión de la enfermedad durante tres años.
Los hallazgos clave muestran que los pacientes tratados con deramiocel experimentaron una disminución promedio de 3.46 puntos en la puntuación total de la Performance of the Upper Limb (PUL 2.0), en comparación con 7.19 puntos en el grupo de control (p=0.019). El efecto del tratamiento mejoró anualmente, con disminuciones promedio anuales de PUL 2.0 de 1.8, 1.2 y 1.1 puntos en los Años 1, 2 y 3, respectivamente.
El medicamento mantuvo un perfil de seguridad favorable sin nuevas señales de seguridad. La FDA ha aceptado la Solicitud de Licencia Biológica de Capricor para la cardiomiopatía asociada a la DMD, con una fecha PDUFA fijada para el 31 de agosto de 2025.
Capricor Therapeutics (NASDAQ: CAPR)는 deramiocel에 대한 HOPE-2 오픈 레이블 확장 시험에서 긍정적인 장기 데이터를 발표하며, 뒤셴 근육디스트로피(DMD) 치료에 있어 상당한 효능을 보여주었습니다. 2025년 MDA 컨퍼런스에서 발표된 이 데이터는 질병 진행 속도가 52% 느려짐을 보여주었습니다.
주요 발견은 deramiocel로 치료받은 환자들이 대조군의 7.19점에 비해 Performance of the Upper Limb (PUL 2.0) 총 점수에서 평균 3.46점 감소를 경험했다는 것입니다 (p=0.019). 치료 효과는 매년 개선되었으며, 1년, 2년, 3년 동안 각각 PUL 2.0의 평균 연간 감소는 1.8, 1.2, 1.1점이었습니다.
이 약물은 새로운 안전 신호 없이 우호적인 안전 프로파일을 유지했습니다. FDA는 DMD 관련 심근병증에 대한 Capricor의 생물학적 라이센스 신청을 수락했으며, PDUFA 날짜는 2025년 8월 31일로 설정되었습니다.
Capricor Therapeutics (NASDAQ: CAPR) a annoncé des données positives à long terme de son essai d'extension open label HOPE-2 pour deramiocel, montrant une efficacité significative dans le traitement de la dystrophie musculaire de Duchenne (DMD). Les données, présentées lors de la Conférence MDA 2025, ont démontré un ralentissement de 52% de la progression de la maladie sur trois ans.
Les résultats clés montrent que les patients traités avec deramiocel ont connu une diminution moyenne de 3,46 points dans le score total de la Performance of the Upper Limb (PUL 2.0), par rapport à 7,19 points dans le groupe témoin (p=0.019). L'effet du traitement s'est amélioré chaque année, avec des diminutions annuelles moyennes de PUL 2.0 de 1,8, 1,2 et 1,1 points respectivement au cours des Années 1, 2 et 3.
Le médicament a maintenu un profil de sécurité favorable sans nouveaux signaux de sécurité. La FDA a accepté la demande de licence biologique de Capricor pour la cardiomyopathie associée à la DMD, avec une date PDUFA fixée au 31 août 2025.
Capricor Therapeutics (NASDAQ: CAPR) hat positive Langzeitdaten aus seiner offenen HOPE-2-Erweiterungsstudie für deramiocel veröffentlicht, die eine signifikante Wirksamkeit bei der Behandlung der Duchenne-Muskeldystrophie (DMD) zeigen. Die Daten, die auf der MDA-Konferenz 2025 präsentiert wurden, zeigten eine 52%ige Verlangsamung des Krankheitsverlaufs über drei Jahre.
Wichtige Ergebnisse zeigen, dass Patienten, die mit deramiocel behandelt wurden, einen durchschnittlichen Rückgang von 3,46 Punkten im Gesamtscore der Performance of the Upper Limb (PUL 2.0) im Vergleich zu 7,19 Punkten in der Kontrollgruppe (p=0.019) erlebten. Der Behandlungseffekt verbesserte sich jährlich, mit durchschnittlichen jährlichen Rückgängen von PUL 2.0 von 1,8, 1,2 und 1,1 Punkten in den Jahren 1, 2 und 3.
Das Medikament wies ein günstiges Sicherheitsprofil auf, ohne neue Sicherheitszeichen. Die FDA hat den Antrag auf biologische Lizenz von Capricor für die mit DMD assoziierte Kardiomyopathie akzeptiert, mit einem PDUFA-Datum, das auf den 31. August 2025 festgelegt wurde.
- 52% reduction in disease progression over 3 years
- Improving treatment effect year over year (PUL 2.0 decline: 1.8 to 1.1 points)
- FDA accepted BLA with PDUFA date set for August 31, 2025
- Favorable long-term safety profile maintained
- Disease progression continues despite treatment (3.46 point decline in PUL 2.0)
Insights
The 3-year data from Capricor's HOPE-2 OLE trial represents a significant clinical breakthrough for deramiocel in DMD treatment. The
What's particularly compelling is the increasing treatment effect over time, with the annual decline in PUL 2.0 scores improving from 1.8 points in Year 1 to just 1.1 points by Year 3. This progressive enhancement suggests cumulative benefit rather than the efficacy plateau or diminishing returns typically observed with chronic therapies.
The evidence of potential disease-modifying activity is especially noteworthy. Patients experiencing a 1-year treatment gap still demonstrated slower disease progression (2.8 vs 3.7 points annually) compared to untreated patients. This suggests deramiocel may fundamentally alter disease biology rather than simply providing symptomatic relief.
With the FDA having already accepted Capricor's BLA for DMD-associated cardiomyopathy with an August 31, 2025 PDUFA date, this skeletal muscle preservation data strengthens the overall value proposition. Few therapies in development target both cardiac and skeletal muscle components of DMD, positioning deramiocel as potentially the most comprehensive approach to disease management if approved.
These results represent a potential paradigm shift in DMD management. The 3.46-point decline in PUL 2.0 over three years versus 7.19 points in untreated patients demonstrates meaningful preservation of upper limb function—directly translating to extended independence for patients in critical activities of daily living like eating, using digital devices, and personal hygiene.
The continued improvement in treatment effect across years (from 1.8 to 1.1 points annual decline) challenges our understanding of DMD progression. Typically, we expect accelerating functional loss as patients age, yet deramiocel appears to counter this trajectory, suggesting it may provide increasing protection as treatment continues.
Perhaps most clinically relevant is the apparent carry-over effect during treatment interruption. The slower decline rate during the 1-year gap (2.8 vs 3.7 points) suggests biological remodeling that persists beyond the treatment period—a true hallmark of disease modification rather than symptom management.
The favorable long-term safety profile is equally important. DMD patients often face treatment burden from multiple therapies with significant side effects. A well-tolerated option preserving both cardiac and skeletal muscle function could dramatically improve the risk-benefit calculation for patients and families navigating this progressive disease. If approved following the August PDUFA date, deramiocel could fundamentally alter how we approach DMD treatment sequencing and combination strategies.
--Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in
--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, 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; 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
FAQ
What are the 3-year efficacy results for Capricor's deramiocel (CAPR) in treating DMD?
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