Latest news with #Deramiocel
Yahoo
2 hours ago
- Business
- Yahoo
Capricor Therapeutics announces four-year data from HOPE-2 OLE study
Capricor Therapeutics (CAPR) announced four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy. The data will be featured in the session titled 'Therapies that Slow Progression' at the Parent Project Muscular Dystrophy 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada. After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF greater than45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function. Additionally, treatment continued to slow skeletal muscle disease progression, as measured by Performance of the Upper Limb, with patients experiencing a smaller average decline in the fourth year compared to the first year. Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See the top stocks recommended by analysts >> Read More on CAPR: Disclaimer & DisclosureReport an Issue FDA's Nicole Verdun placed on administrative leave, STAT reports Capricor Therapeutics announces ODD to Deramiocel Capricor treatment of Becker muscular dystrophy granted orphan designation Roth thinks Capricor Therapeutics drug could still be approved by August 31 Adobe reports Q2 beat, Meta invests in Scale AI: Morning Buzz Sign in to access your portfolio
Yahoo
a day ago
- Business
- Yahoo
Capricor Therapeutics Announces Positive 4-Year Data from HOPE-2 Open-Label Extension Study of Deramiocel in Duchenne Muscular Dystrophy
Four-year data show preservation of cardiac function, including LVEF Skeletal muscle disease progression continues to slow with extended treatment (PUL v2.0) Deramiocel's long-term safety profile remains favorable Results presented at PPMD's 2025 Annual Conference SAN DIEGO, June 20, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for rare diseases, today announced positive four-year safety and efficacy results from its ongoing HOPE-2 Open-Label Extension (OLE) study of Deramiocel, the Company's lead cell therapy candidate for Duchenne Muscular Dystrophy (DMD). The data will be featured in the session titled 'Therapies that Slow Progression' at the Parent Project Muscular Dystrophy (PPMD) 2025 Annual Conference, taking place June 21, 2025, in Las Vegas, Nevada. After four years of continuous treatment, Deramiocel-treated patients showed a median change of -0.5 points compared to baseline. Further, a subgroup analysis of patients with baseline LVEF >45% showed an even greater clinical benefit, supporting early intervention with Deramiocel to potentially preserve cardiac function. Additionally, treatment continued to slow skeletal muscle disease progression, as measured by Performance of the Upper Limb (PUL v2.0), with patients experiencing a smaller average decline in the fourth year (0.6 points) compared to the first year (1.8 points). Together, these findings suggest that extended treatment with Deramiocel may help attenuate the progression of DMD over time. Deramiocel continues to maintain a favorable safety profile throughout the study. 'Cardiomyopathy remains one of the leading causes of mortality in Duchenne and addressing this aspect of the disease is critical to improving outcomes,' said Pat Furlong, Founding President and CEO of PPMD. 'The long-term data from the HOPE-2 OLE study are encouraging, particularly in demonstrating cardiac stabilization over four years. Deramiocel represents an important therapeutic approach and we support continued progress through the regulatory pathway to ensure that treatments targeting both heart and muscle function are available to our community as quickly as possible.' 'These four-year data reinforce the strength and durability of Deramiocel's clinical benefit and favorable safety profile across both cardiac and skeletal muscle function,' said Linda Marbán, Ph.D., CEO of Capricor Therapeutics. 'With our BLA under priority review and several key regulatory steps now completed, we are executing with focus and urgency as we move toward potential approval. Our continued dialogue with the FDA remains on track with no evidence of any delays. We thank the patients, families, and clinicians who have been instrumental in advancing this program.' The PPMD Annual Conference is the largest international event focused on advancing research, clinical trials, and standards of care for Duchenne and Becker muscular dystrophies. Capricor's presentation will be available following the conference in the publications section of the Company's was a randomized, double-blind, placebo-controlled Phase 2 study of Deramiocel in boys and young men with Duchenne Muscular Dystrophy (DMD). Patients received intravenous infusions of either Deramiocel (150 million cells) or placebo every three months. The results were published in The Lancet. Following study completion, all patients entered a treatment gap phase for approximately 392 days (range: 239–567). Eligible patients then enrolled in the HOPE-2 OLE study, receiving Deramiocel every three months. The OLE study previously met its primary endpoint at one year with statistically significant improvement on the PUL v2.0 (p=0.02). Now in its fifth year, the HOPE-2 OLE study remains ongoing, with participants continuing to be monitored for safety, cardiac function, and upper limb Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles. It is caused by the absence of functional dystrophin, a key structural protein in muscle cells. DMD affects approximately 15,000 individuals in the United States and primarily impacts boys. Over time, deterioration of the heart muscle leads to cardiomyopathy and heart failure, which is the leading cause of death in DMD. There is no cure, and treatment options remain (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs), a rare population of cardiac cells that have been shown in preclinical and clinical studies to exert potent immunomodulatory and anti-fibrotic actions in the 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 to adopt a healing, rather than a pro-inflammatory phenotype. CDCs have been investigated in more than 250 peer-reviewed scientific publications and administered to over 250 human subjects across multiple clinical trials. Deramiocel has received Orphan Drug Designation for the treatment of Duchenne Muscular Dystrophy (DMD) from both the U.S. FDA and the European Medicines Agency (EMA). In addition, it has been granted Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S., Advanced Therapy Medicinal Product (ATMP) designation in Europe, and Rare Pediatric Disease Designation from the FDA, which may qualify Capricor for a Priority Review Voucher upon approval. For Becker Muscular Dystrophy (BMD), Deramiocel has also received Orphan Drug Designation from the Therapeutics (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 anti-fibrotic actions in the preservation of cardiac and skeletal muscle function in muscular dystrophies 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 and follow Capricor on Facebook, Instagram and 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 potential that required regulatory inspections may be delayed or not be successful which would delay or prevent product approval; 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, 2024, as filed with the Securities and Exchange Commission on March 26, 2025, and in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, as filed with the Securities and Exchange Commission on May 14, 2025. 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 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 (IND) and is not yet approved for any indications. Neither BMD nor any of Capricor's exosome-based candidates have been approved for clinical Media Contact:Raquel ConaKCSA Strategic Communications rcona@ Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@
Yahoo
12-06-2025
- Business
- Yahoo
Capricor says FDA inspection brought Form 483 with several observations
Capricor Therapeutics (CAPR) announced the completion of the Food and Drug Administration's pre-license inspection of its San Diego manufacturing facility for Deramiocel, the company's lead cell therapy candidate with a biologics license application under FDA review for potential approval in the treatment of Duchenne muscular dystrophy. The inspection concluded with a Form 483 containing several observations. Capricor said it has submitted its responses to the FDA, none of which required material changes to the cGMP process or facility. 'The observations were primarily related to routine quality systems and documentation practices. The Company is confident that the facility will meet the necessary requirements to support product licensure and, pending approval, commercial launch,' Capricor added. The FDA informed Capricor of its intent to hold the Advisory Committee meeting on July 30, although that date is pending confirmation by the FDA. 'At the time of the mid-cycle review, no significant issues or major deficiencies were noted. A late-cycle meeting is planned for mid-July 2025,' the company added. The BLA for Deramiocel remains under priority review with a Prescription Drug User Fee Act action date of August 31. Easily unpack a company's performance with TipRanks' new KPI Data for smart investment decisions Receive undervalued, market resilient stocks right to your inbox with TipRanks' Smart Value Newsletter Published first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>> See today's best-performing stocks on TipRanks >> Read More on CAPR: Disclaimer & DisclosureReport an Issue Largest borrow rate increases among liquid names Capricor Therapeutics participates in a conference call with Maxim Capricor Therapeutics Approves 2025 Equity Incentive Plan MongoDB and Asana downgraded: Wall Street's top analyst calls Capricor Therapeutics initiated with a Buy at Roth Capital
Yahoo
17-03-2025
- Business
- Yahoo
Capricor Therapeutics Announces Positive Data Demonstrating Long-Term Efficacy of Deramiocel for the Treatment of Duchenne Muscular Dystrophy
--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 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 ConaKCSA Strategic Communications rcona@ Capricor Company Contact:AJ Bergmann, Chief Financial Officerabergmann@ in to access your portfolio
