Key Points A third-party manufacturing facility has apparently been flagged by the FDA. The plant is owned by pharmaceutical industry peer Novo Nordisk. 10 stocks we like better than Scholar Rock › A potential delay in an important submission was the news item driving Scholar Rock's (NASDAQ: SRRK) share price down as the trading week kicked off. On Monday, the biotech's shares fell by more than 13% as a result, contrasting unfavorably with the 1.6% gain of the S&P 500 (SNPINDEX: ^GSPC). A concerni ...

Larimar Therapeutics Program Update September 2025 1 pro Forward-Looking Statements This presentation contains forward-looking statements that are based on the beliefs and assumptions of Larimar Therapeutics, Inc. ( "Company") and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward-looking statements, including but not limited to Larimar's ability to develop and commercialize nomlabofusp (CTI-1601) and any other ...

Core Viewpoint - The Gross Law Firm is notifying shareholders of Capricor Therapeutics, Inc. regarding a potential class action lawsuit due to misleading statements related to the company's drug deramiocel for treating cardiomyopathy associated with Duchenne muscular dystrophy [1][3]. Summary by Relevant Sections Company Information - Capricor Therapeutics, Inc. is involved in developing cell therapy candidates, specifically deramiocel, aimed at treating cardiomyopathy linked to Duchenne muscular dystrophy [3]. Allegations - The complaint alleges that Capricor provided investors with misleading information about deramiocel's potential to secure a Biologics License Application (BLA) from the FDA while concealing adverse facts regarding the drug's safety and efficacy data from its Phase 2 HOPE-2 trial [3]. - Following the announcement of a Complete Response Letter (CRL) from the FDA on July 11, 2025, which denied the BLA due to insufficient evidence of effectiveness and the need for additional clinical data, Capricor's stock price fell from $11.40 to $7.64 per share [3]. Class Action Details - The class period for the lawsuit is defined as October 9, 2024, to July 10, 2025, with a deadline for shareholders to register for the class action set for September 15, 2025 [4]. - Shareholders who register will receive updates on the case's progress through a portfolio monitoring software [4]. Law Firm Information - The Gross Law Firm is a nationally recognized class action law firm dedicated to protecting investors' rights against deceit and fraud [5].

Core Insights - Inovio Pharmaceuticals' shares increased by 20.6% following FDA's acceptance of a rolling submission timeline for the biologics license application (BLA) for INO-3107, aimed at treating recurrent respiratory papillomatosis (RRP) in adults [1][5] - The company plans to finalize the BLA submission in the coming months and seeks priority review, targeting FDA acceptance by the end of 2025 [1][2] - INO-3107 is designed to elicit a targeted T cell response against HPV-6 and HPV-11, which cause RRP, and has received breakthrough therapy and orphan drug designations in the U.S. [3][5] Company Developments - Inovio's BLA submission is supported by data from a completed phase I/II study that demonstrated the candidate's safety, tolerability, immunogenicity, and efficacy in patients with HPV-related RRP [6] - The FDA has indicated that the completed study could support a BLA filing under the accelerated approval program, negating the need for a pivotal phase III study [6] - A confirmatory study for INO-3107 will be initiated, enrolling 100 patients across the U.S. [7] Industry Context - Precigen, Inc. recently received FDA approval for Papzimeos, a treatment for RRP, which also holds breakthrough therapy and orphan drug designations [8] - Precigen's Papzimeos was approved based on data from a pivotal phase I/II study, which met primary safety and efficacy endpoints, eliminating the need for a confirmatory study [10] - Year-to-date, Inovio's shares have risen by 34.4%, significantly outperforming the industry average growth of 3.2% [4]

Core Viewpoint - Capricor Therapeutics, Inc. is facing a class action lawsuit due to allegations of misleading statements regarding its lead cell therapy candidate, deramiocel, which was intended for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD) [3][4]. Group 1: Allegations and Impact - The lawsuit claims that Capricor provided investors with overly positive information about deramiocel's potential to obtain a Biologics License Application (BLA) from the FDA while concealing adverse facts related to its Phase 2 HOPE-2 trial data [3][4]. - Following the announcement of a Complete Response Letter (CRL) from the FDA on July 11, 2025, which denied the BLA due to insufficient evidence of effectiveness and the need for additional clinical data, Capricor's stock price dropped from $11.40 to $7.64 per share [3][4]. Group 2: Class Action Details - The class period for the lawsuit is defined as October 9, 2024, to July 10, 2025, and shareholders are encouraged to register for participation by September 15, 2025 [3][4]. - Shareholders who register will receive updates on the case's progress and can seek lead plaintiff status without any cost or obligation [4]. Group 3: Law Firm Information - The Gross Law Firm, which is handling the case, is a nationally recognized class action law firm dedicated to protecting investors' rights against deceit and fraud [5]. - The firm aims to ensure companies adhere to responsible business practices and seeks recovery for investors who suffered losses due to misleading statements [5].

Core Viewpoint - GSK's Blenrep combination therapy has been voted against by the FDA's Oncologic Drugs Advisory Committee (ODAC) due to an unfavorable benefit/risk profile, indicating that the risks currently outweigh the benefits for treating relapsed or refractory multiple myeloma [1][8]. GSK's Stock Performance - Following the ODAC announcement, GSK's shares fell by 4.7% [2]. - Year-to-date, GSK's shares have increased by 7.8%, contrasting with a 0.6% decline in the industry [6]. Regulatory Context - The ODAC's opinion is significant as the FDA typically considers it in their final decision, which is expected on July 23 [3][8]. - The Blenrep combination therapy has already been approved in the UK and Japan but faces regulatory challenges in the United States [8][11]. - Regulatory applications for the Blenrep combination are under review in the EU and other countries [9]. Clinical Study Background - The biologics license application (BLA) for the Blenrep combination was based on data from the III DREAMM-7 and DREAMM-8 studies, which met their primary endpoints of progression-free survival (PFS) [7]. - However, Blenrep was previously withdrawn from the U.S. market after failing to meet the primary endpoint in the phase III DREAMM-3 study [10].

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.