About this content About Emily Jarvie Emily began her career as a political journalist for Australian Community Media in Hobart, Tasmania. After she relocated to Toronto, Canada, she reported on business, legal, and scientific developments in the emerging psychedelics sector before joining Proactive in 2022. She brings a strong journalism background with her work featured in newspapers, magazines, and digital publications across Australia, Europe, and North America, including The Examiner, The Advocate, ...

Summary of Capricor Therapeutics (CAPR) Update Call Company Overview - **Company**: Capricor Therapeutics - **Focus**: Development of Daramyacel, a cell therapy candidate for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) Key Points Regulatory Update - Capricor received a Complete Response Letter (CRL) from the FDA regarding the Biologics License Application (BLA) for Daramyacel, indicating that the application does not meet the requirements for substantial evidence of effectiveness and requires more clinical data [4][5] - The review clock has been stopped until a complete response is submitted, and Capricor does not believe a new BLA will be required [5][41] - Capricor is preparing a written response to the CRL and has initiated engagement with the FDA for further discussions [5][10] Clinical Data and Efficacy - The BLA was supported by data from the HOPE-two trial, which demonstrated statistically significant benefits in cardiac function [7][8] - The HOPE-two open label extension study showed significant results in left ventricular ejection fraction with a p-value of 0.0079 [9] - Ongoing Phase III HOPE-three clinical trial is seen as a key opportunity to address the FDA's request for additional clinical data [10] - HOPE-three has completed a twelve-month evaluation of 104 DMD patients, with data expected in Q3 2025 [10] Manufacturing and Quality Control - Capricor is committed to resolving outstanding issues in the chemistry, manufacturing, and controls (CMC) section of the application [11] - The company believes that the issues raised by the FDA are resolvable in a timely manner [11] Industry Context - The FDA's decision is viewed as concerning, especially given the high unmet medical need in DMD and the innovative nature of the therapy [6][12] - Capricor emphasizes the importance of using long-term extension data and real-world evidence in evaluating therapies for rare diseases like DMD [12] Support from Medical Community - A group of leading DMD physician scientists submitted a letter to the FDA expressing support for the Daramyacel clinical program [13] - The letter highlighted their extensive experience in treating DMD and their belief in the efficacy and safety of Daramyacel [13] Future Outlook - Capricor remains committed to working collaboratively with the FDA and is optimistic about the potential for Daramyacel to receive approval [14][15] - The company aims to provide updates as they achieve clarity with the FDA regarding the path forward [69] Financial and Strategic Considerations - Capricor maintains its priority review status and is focused on addressing the FDA's concerns without needing a new BLA [48] - The partnership with NS Pharma remains strong, with continued commitment to commercialization in the U.S. despite the CRL setback [58] Additional Insights - The FDA's cancellation of the Public Advisory Committee meeting without explanation raised concerns about the review process [6] - Capricor's approach to statistical analysis in clinical trials has been a point of contention with the FDA, particularly regarding the use of parametric versus nonparametric models [30][36] This summary encapsulates the critical updates and insights from the Capricor Therapeutics conference call, highlighting the company's current challenges and future strategies in the context of regulatory approval for Daramyacel.

Capricor Therapeutics Inc. CAPR stock is trading lower on Friday. The company received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Deramiocel, the company’s lead cell therapy candidate for cardiomyopathy associated with Duchenne muscular dystrophy (DMD).In the CRL, the FDA stated that it had completed its application review but could not approve the BLA in its current form.The agency specifically cited that the BLA d ...

NEW YORK, July 11, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Sarepta Therapeutics, Inc. ("Sarepta" or the "Company") (NASDAQ: SRPT) of a class action securities lawsuit.CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Sarepta investors who were adversely affected by alleged securities fraud between June 22, 2023 and June 24, 2025. Follow the link below to get more information and be contacted by a member of our team:https://zlk.com/pslra-1/sarepta-lawsuit-submission- ...

Summary of BridgeBio Pharma (BBIO) Update / Briefing July 11, 2025 Company Overview - **Company**: BridgeBio Pharma (BBIO) - **Focus**: Development of therapies for genetic diseases, specifically Limb Girdle Muscular Dystrophy (LGMD) Industry Context - **Disease**: Limb Girdle Muscular Dystrophy (LGMD), particularly LGMD2IR9FKRP related - **Patient Population**: Approximately 7,000 patients in the U.S. and EU with a focus on those with significant cardiac and respiratory involvement Key Points and Arguments 1. **Therapeutic Development**: The Phase III trial for BVP-four eighteen is fully enrolled, with interim data expected later this year to support an accelerated approval strategy [9][10][24] 2. **Clinical Trials**: The Phase II study has shown promising results, with a focus on increasing glycosylated alpha dystroglycan levels and reducing serum creatine kinase levels, indicating muscle injury reduction [30][40] 3. **FDA Engagement**: Positive discussions with the FDA regarding the use of glycosylated alpha dystroglycan as a surrogate endpoint for accelerated approval [45][48] 4. **Market Opportunity**: The company aims to be the first to market with a disease-modifying oral therapy, addressing a significant unmet need in the LGMD patient population [51][55] 5. **Patient Engagement**: Efforts to increase patient identification through partnerships with advocacy organizations and genetic testing programs [53][54] Additional Important Content 1. **Genetic Understanding**: The understanding of LGMD has evolved from clinical symptoms to identifying specific genetic mutations, particularly the FKRP gene associated with LGMD2IR9 [24][19] 2. **Clinical Impact**: The therapy aims to stabilize muscle function and potentially improve quality of life for patients, with a focus on slowing disease progression rather than just symptomatic relief [62][75] 3. **Safety Profile**: BVP-four eighteen has been well tolerated in trials, with only minor gastrointestinal side effects reported [38][82] 4. **Regulatory Strategy**: The company maintains a conservative approach to regulatory approval, emphasizing the need for flexibility given the lack of existing therapies for this patient population [80][81] Conclusion - BridgeBio Pharma is positioned to potentially deliver a first-of-its-kind therapy for LGMD, with ongoing clinical trials and strong regulatory engagement paving the way for future developments in the treatment of this genetic disorder. The focus on patient outcomes and safety, combined with a clear understanding of the disease's genetic basis, underscores the company's commitment to addressing significant unmet medical needs in the LGMD community.

NEW YORK, July 11, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in PepGen Inc. ("PepGen Inc." or the "Company") (NASDAQ: PEPG) of a class action securities lawsuit.CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of PepGen Inc. investors who were adversely affected by alleged securities fraud between March 7, 2024 and March 3, 2025. Follow the link below to get more information and be contacted by a member of our team:https://zlk.com/pslra-1/pepgen-inc-lawsuit-submission-form ...

NEW YORK, July 11, 2025 /PRNewswire/ -- Levi & Korsinsky, LLP notifies investors in Rocket Pharmaceuticals, Inc. ("Rocket" or the "Company") (NASDAQ: RCKT) of a class action securities lawsuit.CLASS DEFINITION: The lawsuit seeks to recover losses on behalf of Rocket investors who were adversely affected by alleged securities fraud between February 27, 2025 and May 26, 2025. Follow the link below to get more information and be contacted by a member of our team:https://zlk.com/pslra-1/rocket-pharmaceuticals-i ...

HOUSTON, July 11, 2025 (GLOBE NEWSWIRE) -- FibroBiologics, Inc. (Nasdaq: FBLG) (“FibroBiologics”), a clinical-stage biotechnology company with 275+ patents issued and pending with a focus on the development of therapeutics and potential cures for chronic diseases using fibroblasts and fibroblast-derived materials, today announced that Founder and Chief Executive Officer, Pete O’Heeron, will present at the Advanced Wound Care Summit USA in Boston, Massachusetts, on July 15, 2025. The presentation will includ ...

Now Read This: Photo via Shutterstock Loading... U.S. stock futures were lower this morning, with the Dow futures falling around 300 points on Friday. Shares of Frequency Electronics, Inc. FEIM fell sharply in today's pre-market trading after the company reported results for the fourth quarter. Frequency Electronics reported earnings of 34 cents per share for the fourth quarter, up from 28 cents per share in the year-ago period. The company's sales rose to $19.986 million from $15.576 million. Frequency Ele ...

Capricor Therapeutics shares plunged more than 60% after US regulators rejected the company’s treatment for a deadly muscle disorder https://t.co/pLLiQDiV7i ...