Latest news with #LindaMarbán
Yahoo
05-05-2025
- Business
- Yahoo
Capricor Therapeutics Announces Completion of Mid-Cycle Review Meeting with FDA on Deramiocel for the Treatment of Duchenne Muscular Dystrophy Cardiomyopathy
-Company remains on track for PDUFA target action date of August 31, 2025- -Advisory committee meeting to be held in advance of target action date- SAN DIEGO, May 05, 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 the completion of a mid-cycle review meeting with the U.S. Food and Drug Administration (FDA) for the Company's Biologics License Application (BLA) seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. During the meeting, FDA stated that no significant deficiencies have been identified by the Review Committee and that the package is on track for a Prescription Drug User Fee Act (PDUFA) action date of August 31, 2025. The FDA has also confirmed its intent to hold an advisory committee meeting, although an official date has not yet been set. 'The successful completion of our mid-cycle review meeting along with the upcoming advisory committee meeting represents major milestones on the path towards approval of deramiocel,' said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. 'Deramiocel is a first-in-class cellular therapy with the potential to halt or slow the progression of DMD-cardiomyopathy, and we are pleased to have the opportunity to present the efficacy and safety data to the advisory committee. We have been actively preparing for an advisory committee meeting, and we look forward to providing the physician and patient perspectives to highlight the weight of evidence supporting the transformative potential of deramiocel in treating DMD-cardiomyopathy.' The BLA submission is supported by Capricor's cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials compared to patient level data from an FDA-funded and published dataset on the natural history of DMD-cardiomyopathy and potential biomarkers of disease progression. Efficacy from the ongoing HOPE-3 study is not part of this BLA package 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 (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 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 the subject of over 200 peer-reviewed scientific publications and have been administered to over 250 human subjects across several clinical trials. Deramiocel for the treatment of DMD, has received Orphan Drug Designation from the FDA and European Medicines Agency (EMA). The regulatory pathway for deramiocel is supported by RMAT (Regenerative Medicine Advanced Therapy Designation) in the U.S. and the Advanced Therapy Medicinal Product (ATMP) Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD, Capricor would be eligible to receive a Priority Review Voucher (PRV) based on its previous receipt of a rare pediatric disease 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 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 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. 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. None 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@ in to access your portfolio
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
Yahoo
04-03-2025
- Business
- Yahoo
Capricor Therapeutics Announces FDA Acceptance and Priority Review of its Biologics License Application for Deramiocel to Treat Duchenne Muscular Dystrophy
--FDA assigns PDUFA target action date of August 31, 2025-- --If approved, deramiocel would be first therapy to treat Duchenne muscular dystrophy cardiomyopathy-- --At this time, FDA has not identified any potential review issues with the BLA-- SAN DIEGO, March 04, 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 the U.S. Food and Drug Administration ('FDA') has accepted for review its Biologics License Application ('BLA') seeking full approval for deramiocel, an investigational cell therapy, as a treatment for patients diagnosed with Duchenne muscular dystrophy ('DMD') cardiomyopathy. Additionally, the FDA granted the BLA Priority Review with a Prescription Drug User Fee Act ('PDUFA') target action date of August 31, 2025 and at this time, the FDA has not identified any potential review issues. "We are thrilled to announce the acceptance of our BLA bringing us one step closer to providing this first-in-class treatment for Duchenne-cardiomyopathy, a condition for which there are no approved therapies" said Linda Marbán, Ph.D., Chief Executive Officer of Capricor. 'If our application is successful, we expect deramiocel to be a lifelong treatment, administered quarterly, with the potential to be widely adopted across the DMD-cardiomyopathy treatment landscape. We want to extend our appreciation to the patients, their families and advocates who continue to work with Capricor and to the FDA for its commitment to accelerating treatments for DMD.' The BLA submission is supported by Capricor's existing cardiac data from its Phase 2 HOPE-2 and HOPE-2 Open Label Extension ('OLE') trials compared to natural history data from an FDA-funded and published dataset on the implications of DMD cardiomyopathy and potential biomarkers of disease progression. The FDA also informed the Company they have not yet decided whether an Advisory Committee meeting is needed in relation to this application. 'Deramiocel has shown in multiple clinical trials attenuation of DMD-cardiomyopathy, which is currently one of the leading causes of death in those with DMD,' commented Craig McDonald, M.D., national PI and University of California, Davis, Professor and Department of Physical Medicine and Rehabilitation Chair. 'Based on the totality of the safety and efficacy data deramiocel has shown, this potential approval offers patients a first-in-class therapeutic for DMD-cardiomyopathy.' The FDA grants Priority Review to applications for medicines that, if approved, provide significant improvements in the safety or effectiveness of the treatment of a serious condition. Deramiocel for the treatment of DMD has received Orphan Drug Designation from the FDA and European Medicines Agency ('EMA'). The regulatory pathway for deramiocel is supported by RMAT ('Regenerative Medicine Advanced Therapy Designation') in the U.S. and the Advanced Therapy Medicinal Product ('ATMP') Designation in the European region. In addition, if Capricor were to receive FDA marketing approval for deramiocel regarding the treatment of DMD by September 30, 2026, Capricor would be eligible to receive a Priority Review Voucher ('PRV') based on its previous receipt of a rare pediatric disease designation. About Deramiocel Deramiocel (also referred to as 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, antifibrotic and regenerative actions in dystrophinopathy and heart failure. 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 demonstrate immunomodulatory, antifibrotic, and regenerative actions specifically tailored for dystrophinopathies and heart disease. 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
