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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.

Core Viewpoint - Capricor Therapeutics Inc. received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for Deramiocel, indicating that the application cannot be approved in its current form due to insufficient evidence of effectiveness and the need for additional clinical data [1][2]. Group 1: FDA Response and Requirements - The FDA stated that the BLA does not meet the statutory requirement for substantial evidence of effectiveness and that additional clinical data is needed [2]. - The CRL referenced outstanding items in the Chemistry, Manufacturing and Controls (CMC) section, which Capricor claims to have addressed in prior communications, but the FDA did not review these materials due to the timing of the CRL issuance [3]. Group 2: Next Steps and Company Plans - The FDA confirmed that it will restart the review clock upon resubmission and offered Capricor the opportunity to request a Type A meeting to discuss the path forward [4]. - Capricor plans to submit data from the Phase 3 HOPE-3 clinical trial to provide additional evidence of effectiveness, with topline results expected in the third quarter of 2025 [5]. Group 3: Market Reaction - Following the news, CAPR stock is down 37.4% at $7.14 during the premarket session [6].

Group 1 - U.S. stock futures are lower, with Dow futures down approximately 300 points [1] - Frequency Electronics, Inc. reported fourth-quarter earnings of 34 cents per share, an increase from 28 cents per share year-over-year [1] - Frequency Electronics' sales rose to $19.986 million from $15.576 million [1] Group 2 - Frequency Electronics shares fell 5.4% to $21.44 in pre-market trading [2] - Capricor Therapeutics, Inc. shares declined 44% to $6.39 following a regulatory update on Deramiocel BLA for Duchenne Muscular Dystrophy [4] - Youlife Group Inc. shares fell 23.2% to $3.84 after an 82% drop on Thursday [4] - Civista Bancshares, Inc. shares dipped 12.5% to $21.72 after announcing a public offering of common shares [4] - Resolute Holdings Management, Inc. shares fell 9.5% to $36.01 after a previous gain of over 3% [4] - Scage Future shares decreased by 7.7% to $6.19 [4] - Albemarle Corporation shares fell 4% to $71.67 after a 5% gain on Thursday [4]

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

Core Viewpoint - Pomerantz LLP is investigating claims of potential securities fraud or unlawful business practices by Capricor Therapeutics, Inc. and its officers or directors [1]. Group 1: Company Developments - On May 5, 2025, Capricor announced that the FDA confirmed its intent to hold an advisory committee meeting regarding the Biologics License Application for deramiocel, a treatment for Duchenne muscular dystrophy cardiomyopathy [3]. - Following this announcement, Capricor's stock price dropped by $3.00 per share, or 29.13%, closing at $7.30 per share on May 6, 2025 [4]. - On June 20, 2025, it was reported that the FDA canceled the advisory committee meeting due to concerns about the drug's efficacy and safety, leading to a further decline in Capricor's stock price by $3.68 per share, or 30.82%, closing at $8.26 per share [4]. Group 2: Legal and Investigative Actions - Pomerantz LLP, a firm specializing in corporate, securities, and antitrust class litigation, is leading the investigation into Capricor's practices [5]. - The firm has a history of recovering significant damages for victims of securities fraud and corporate misconduct [5].

Company Overview - Capricor Therapeutics (CAPR) shares increased by 20.4% to $9.25 in the last trading session, following a period of 25.1% loss over the past four weeks, indicating a significant recovery in stock performance [1] - The company is focused on developing Deramiocel, a cell therapy candidate aimed at treating Duchenne Muscular Dystrophy-associated cardiomyopathy [2] Regulatory Updates - Capricor provided positive regulatory updates regarding its Biologics License Application (BLA) for Deramiocel, with the FDA indicating that an Advisory Committee meeting is not required at this time [2] - The BLA is currently under Priority Review, with a target action date set for August 31, 2025, which may enhance investor confidence [2] Financial Performance Expectations - The company is expected to report a quarterly loss of $0.43 per share, reflecting a year-over-year decline of 22.9% [3] - Revenue projections for the upcoming quarter are estimated at $2.89 million, down 27.2% compared to the same quarter last year [3] Earnings Estimate Trends - The consensus EPS estimate for Capricor has remained unchanged over the last 30 days, suggesting stability in earnings expectations [4] - The stock's price movement is typically correlated with trends in earnings estimate revisions, indicating that future price strength may depend on any changes in these estimates [4] Industry Context - Capricor operates within the Zacks Medical - Products industry, which includes other companies such as Myomo, Inc. (MYO) [4] - Myomo's consensus EPS estimate has seen a significant change of -23.5% over the past month, indicating potential challenges within the same industry [5]

Core Insights - Capricor Therapeutics Inc is advancing its Biologics License Application (BLA) for Deramiocel, aimed at treating Duchenne Muscular Dystrophy (DMD)-associated cardiomyopathy [1][3] - DMD affects around 15,000 individuals in the U.S., primarily boys, due to the absence of functional dystrophin [2] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not necessary at this time, with the BLA under Priority Review and a PDUFA target action date set for August 31, 2025 [3][4] - Capricor has been informed of a potential Advisory Committee meeting on July 30, 2025, pending FDA confirmation, with no significant issues noted during the mid-cycle review [4] Company Performance - CEO Linda Marbán stated that the company has successfully met all key regulatory milestones, including a pre-license inspection and mid-cycle review [5] - Capricor recently presented four-year data from its HOPE-2 Open-Label Extension study, showcasing one of the longest treatment datasets in DMD, focusing on cardiac and skeletal muscle function [6] Market Reaction - Following these updates, CAPR stock experienced a 20% increase, reaching $9.22 [6]

Core Insights - The article discusses the use of cookies to enhance user experience and analyze website traffic [1] Group 1 - The company utilizes cookies for improving user experience [1] - Data collected through cookies may be shared with analytics partners [1] - Consent is required from users to store cookies on their devices [1]

Core Insights - Capricor Therapeutics is advancing its lead cell therapy candidate, Deramiocel, for the treatment of Duchenne Muscular Dystrophy (DMD) associated cardiomyopathy, with a Biologics License Application (BLA) under Priority Review by the FDA, targeting an action date of August 31, 2025 [1][2][3] Regulatory Updates - The FDA has indicated that an Advisory Committee meeting is not required at this time, and the BLA remains under Priority Review [2][7] - A late-cycle review meeting is scheduled for mid-July 2025 [7] Clinical Data - Capricor presented four-year data from its HOPE-2 Open-Label Extension study at the 2025 Parent Project Muscular Dystrophy Conference, showing sustained cardiac function and clinical benefits from long-term Deramiocel treatment [3][7] Product Information - Deramiocel (CAP-1002) consists of allogeneic cardiosphere-derived cells (CDCs) that have demonstrated immunomodulatory and anti-fibrotic actions in preserving cardiac and skeletal muscle function in DMD [5][8] - The product has received multiple designations, including Orphan Drug Designation and Regenerative Medicine Advanced Therapy designation from the FDA [6][10] Market and Collaboration - Capricor has entered into an exclusive commercialization agreement for Deramiocel in the United States and Japan with Nippon Shinyaku Co., Ltd., pending regulatory approval [10]