Core Insights - NS Pharma, a subsidiary of Nippon Shinyaku, has received FDA acceptance for the Biologics License Application (BLA) for deramiocel, a cell therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with a target action date of August 31, 2025 [1][2] - The FDA has granted Priority Review status for deramiocel, indicating a potential expedited review process [1] - NS Pharma is exclusively responsible for the commercialization and distribution of deramiocel in the U.S. under an agreement with Capricor Therapeutics [2] About Deramiocel - Deramiocel is composed of allogeneic cardiosphere-derived cells (CDCs), which have demonstrated immunomodulatory, antifibrotic, and regenerative properties in preclinical and clinical studies [3] - CDCs function by secreting exosomes that target macrophages, promoting a healing phenotype rather than a pro-inflammatory one [3] - Over 100 peer-reviewed publications have focused on CDCs, which have been administered to more than 200 human subjects in various clinical trials [3] About Duchenne Muscular Dystrophy - Duchenne muscular dystrophy primarily affects males and leads to progressive weakness and loss of skeletal, cardiac, and respiratory muscles [4] - Early symptoms include delayed motor skills, with significant mobility loss often requiring wheelchair use by adolescence [4] - Cardiac and respiratory complications typically arise in the teenage years, posing serious health risks [4] About Capricor Therapeutics - Capricor Therapeutics is a biotechnology company focused on cell and exosome-based therapeutics for rare diseases [5] - The company is developing its proprietary StealthX™ platform for targeted delivery of therapeutics, including oligonucleotides and proteins [5] About NS Pharma - NS Pharma is a wholly owned subsidiary of Nippon Shinyaku Co., Ltd., dedicated to advancing treatments for rare diseases [6]

Core Insights - Capricor Therapeutics is participating in one-on-one investor meetings and providing a corporate update at Leerink's Global Healthcare Conference 2025 from March 10-12, 2025 [1][2] - The company will present updates on its lead program, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD) along with other scientific and corporate updates [2] Company Overview - Capricor Therapeutics, Inc. is focused on developing cell and exosome-based therapeutics aimed at treating rare diseases, with deramiocel being its lead product candidate [3] - Deramiocel is an allogeneic cardiac-derived cell therapy that has shown immunomodulatory, antifibrotic, and regenerative actions, specifically for dystrophinopathies and heart disease [3] - The company is also utilizing its proprietary StealthX™ platform for preclinical development in areas such as vaccinology and targeted delivery of therapeutics [3] Commercialization Agreement - Capricor has entered into an exclusive commercialization and distribution agreement for deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5][6]

Core Insights - Capricor Therapeutics is participating in one-on-one investor meetings and providing a corporate update at Leerink's Global Healthcare Conference 2025 from March 10-12, 2025 [1][2] - The company will present updates on its lead program, deramiocel, for the treatment of Duchenne muscular dystrophy (DMD) [2][5] - Capricor is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel being a key product candidate in late-stage development [3] Company Overview - Capricor Therapeutics, Inc. is a biotechnology company listed on NASDAQ (CAPR) that aims to redefine treatment for rare diseases through innovative therapies [3] - The lead product candidate, deramiocel, is an allogeneic cardiac-derived cell therapy showing immunomodulatory, antifibrotic, and regenerative actions [3] - The company is also developing exosome technology using its proprietary StealthX™ platform for various therapeutic applications [3] Strategic Partnerships - Capricor has entered into an exclusive commercialization and distribution agreement for deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [5]

Core Insights - The article discusses the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities in the biotech sector [1][2]. Group 1 - The service offers a library of over 600 biotech investing articles, a model portfolio of more than 10 small and mid-cap stocks, and live chat features for investors [2]. - A promotional offer is available for new subscribers, including a two-week free trial and a discounted annual subscription price of $399, which represents a 33.50% discount compared to the monthly rate of $49 [1]. Group 2 - The author of the article has no current stock or derivative positions in any of the companies mentioned, nor plans to initiate any positions in the next 72 hours [3]. - The article emphasizes that past performance is not indicative of future results, and no specific investment recommendations are provided [4].

Core Points - The FDA has accepted Capricor Therapeutics Inc's Biologics License Application for deramiocel, aimed at treating Duchenne muscular dystrophy (DMD) cardiomyopathy [1] - The FDA granted Priority Review status with a target action date of August 31, 2025 [1] - No potential review issues have been identified by the FDA [2] Company and Industry Summary - The BLA submission is supported by data from Capricor's Phase 2 HOPE-2 and HOPE-2 Open Label Extension trials, which were compared to natural history data [3] - The FDA has not yet determined if an Advisory Committee meeting will be necessary for this application [3] - Deramiocel has received Orphan Drug Designation from both the FDA and the European Medicines Agency [4] - If approved by September 30, 2026, Capricor could receive a Priority Review Voucher due to its previous rare pediatric disease designation [4] - CAPR stock increased by 6.33% to $14.54 following the news [4]

Core Insights - The FDA has accepted Capricor Therapeutics' Biologics License Application (BLA) for deramiocel, targeting Duchenne muscular dystrophy (DMD) cardiomyopathy, with a PDUFA action date set for August 31, 2025 [1][2] - If approved, deramiocel would be the first therapy specifically for DMD cardiomyopathy, a condition currently lacking approved treatments [1][2] - The BLA is supported by data from Capricor's Phase 2 clinical trials, indicating the potential effectiveness of deramiocel in treating DMD cardiomyopathy [2][3] Company Overview - Capricor Therapeutics is focused on developing cell and exosome-based therapeutics for rare diseases, with deramiocel as its lead product candidate [7] - The company has received Orphan Drug Designation from the FDA and EMA for deramiocel, which is also recognized under the Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S. [3] - Capricor has entered into an exclusive commercialization agreement for deramiocel with Nippon Shinyaku Co., Ltd. for the U.S. and Japan markets, pending regulatory approval [9] Product Details - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have shown immunomodulatory, antifibrotic, and regenerative properties in clinical studies [4] - The therapy is designed to be administered quarterly and aims to provide a lifelong treatment option for patients with DMD cardiomyopathy [2][3] - Clinical trials have demonstrated deramiocel's ability to attenuate DMD cardiomyopathy, which is a leading cause of death in DMD patients [3] Disease Context - Duchenne muscular dystrophy (DMD) is a severe genetic disorder affecting approximately 15,000-20,000 individuals in the U.S., characterized by progressive muscle weakness and inflammation [5][6] - The median age of mortality for DMD patients is around 30 years, with heart failure being the leading cause of death due to the disease's impact on heart muscle cells [5][6]

Group 1 - Capricor Therapeutics submitted its Biologics License Application (BLA) for deramiocel, targeting patients with Duchenne muscular dystrophy (DMD) cardiomyopathy [1] - The company highlighted that deramiocel is slowing the progression of cardiac dysfunction in DMD patients [1]

SAN DIEGO, Feb. 10, 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 that the Company is scheduled to participate in the following upcoming investor conferences. Event:Oppenheimer 35thAnnual Healthcare Life Sciences Conference (Virtual)Date:February 12, 2025 from 12:00-12:30 p.m. ETWebcast LinkClick here Event:Barclays 27thAnnual Global Healthcare Conferenc ...

Capricor Therapeutics (CAPR) closed the last trading session at $13.47, gaining 7.6% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $41.50 indicates a 208.1% upside potential.The mean estimate comprises six short-term price targets with a standard deviation of $18.52. While the lowest estimate of $25 indicates an 85.6% increase from the current price level, the most optimistic ana ...

Shares of Capricor Therapeutics (CAPR) gained 8.4% on Thursday following the completion of the submission of a biologics license application (BLA) to the FDA seeking full approval for deramiocel to treat patients diagnosed with Duchenne muscular dystrophy (DMD) cardiomyopathy. CAPR’s lead product candidate and only clinical pipeline asset, deramiocel, is an investigational, allogeneic cardiac-derived cell therapy. A final decision of the FDA is anticipated in the second half of 2024.Capricor initiated its r ...