Core Viewpoint - Rosen Law Firm is reminding investors who purchased Sarepta Therapeutics, Inc. securities between June 22, 2023, and June 24, 2025, of the upcoming lead plaintiff deadline on August 25, 2025, for a class action lawsuit [1]. Group 1: Class Action Details - Investors who purchased Sarepta securities during the specified Class Period may be entitled to compensation without any out-of-pocket fees through a contingency fee arrangement [2]. - A class action lawsuit has already been filed, and those wishing to serve as lead plaintiff must act by August 25, 2025 [3]. - Investors can join the class action by visiting the provided link or contacting the law firm directly for more information [6]. Group 2: Law Firm Credentials - Rosen Law Firm emphasizes the importance of selecting qualified counsel with a successful track record in securities class actions, highlighting its own achievements in this area [4]. - The firm has secured significant settlements for investors, including over $438 million in 2019 alone, and has been consistently ranked among the top firms for securities class action settlements [4]. Group 3: Case Specifics - The lawsuit alleges that Sarepta made false and misleading statements regarding its gene therapy product, ELEVIDYS, which posed significant safety risks and failed to detect severe side effects during trials [5]. - The adverse events related to ELEVIDYS treatment led to a halt in recruitment and dosing in trials, attracting regulatory scrutiny and increasing risks around the therapy's approvals [5].

SAN FRANCISCO, July 17, 2025 (GLOBE NEWSWIRE) -- When, on May 27, 2025, Rocket Pharmaceuticals (NASDAQ: RCKT) revealed that the FDA put a clinical hold on its Phase 2 pivotal trial for RP-A501, an experimental therapy intended to treat patients with a rare gene disorder called Danon disease, investors in the company’s shares saw their investment crash over 60% that day. Danon disease is a multi-organ lysosomal-associated disorder leading to early death due to heart disorder. Since the May 27 announcement, a ...

Key Takeaways AstraZeneca's anselamimab missed the primary endpoint in both phase III CARES studies for AL amyloidosis. The drug showed a clinically meaningful benefit in a prespecified subgroup, but details remain undisclosed. AZN plans a full analysis of the data and will present findings at an upcoming medical meeting.AstraZeneca (AZN) reported disappointing results from two late-stage studies that evaluated its investigational light chain depleter antibody, anselamimab, in certain patients with a rare ...

OSE Immunotherapeutics Launches Newsletter for Individual Shareholders NANTES, France, July 17, 2025, 6:30pm CET – OSE Immunotherapeutics SA (ISIN: FR0012127173; Mnemo: OSE), a biotech company dedicated to developing first-in-class assets in immuno-oncology (IO) and immuno-inflammation (I&I) that address the unmet patient needs, today launched the inaugural edition of its Newsletter, designed specifically for individual shareholders and investors in France and internationally. " This year, OSE Immunotherape ...

NEW YORK, July 17, 2025 (GLOBE NEWSWIRE) -- Class Action Attorney Juan Monteverde with Monteverde & Associates PC (the “M&A Class Action Firm”), has recovered millions of dollars for shareholders and is recognized as a Top 50 Firm in the 2024 ISS Securities Class Action Services Report. The firm is headquartered at the Empire State Building in New York City and is investigating Onconetix, Inc. (NASDAQ: ONCO) related to its merger with Ocuvex Therapeutics, Inc. Upon completion of the proposed transaction, e ...

Core Viewpoint - Sarepta Therapeutics is experiencing a stock price increase following an update on the label expansion efforts for Elevidys, its gene therapy for Duchenne muscular dystrophy, despite the FDA's request for a black box warning regarding acute liver injury and failure [1][10]. Label Expansion and FDA Interaction - The FDA has requested a black box warning for Elevidys, which Sarepta agrees to, resolving material issues with the ambulant portion of the label [2][10]. - A Sarepta committee is reviewing cases of acute liver failure and is exploring enhanced immunosuppressive regimens for non-ambulant patients [3][4]. Financial Restructuring - Sarepta announced a strategic restructuring plan aimed at prioritizing programs and achieving approximately $400 million in annual cost reductions, with a workforce reduction of 36% impacting around 500 employees [5]. - The restructuring is projected to lower average annual non-GAAP R&D and SG&A expenses to between $800 million and $900 million starting in 2026 [5]. Financial Performance - Preliminary financial highlights for Q2 2025 show total net product revenue of $513 million, with Elevidys contributing $282 million [6]. - As of June 30, the company reported cash and investments of approximately $850 million [6]. Strategic Focus - The company is shifting its focus towards chronically administered therapies for neurodegenerative and pulmonary diseases, pausing several gene therapy programs for limb-girdle muscular dystrophy [7]. - Sarepta plans to submit a Biologics License Application for SRP-9003 for LGMD type 2E/R4 later this year [7]. Management Changes - In conjunction with the restructuring, Sarepta appointed Ian Estepan as President and Chief Operating Officer and Ryan Wong as CFO [9]. Analyst Insights - Analysts from William Blair suggest that the FDA's request for a black box warning indicates that complete removal of Elevidys from the market is unlikely, leading to increased investor confidence in the company's ability to manage long-term debts [10]. - There are expectations of short-term challenges for Elevidys as patients and doctors exhibit hesitation, with cautious investor sentiment until early results from the ENDEAVOR trial are available [10][11].

Core Insights - Sarepta Therapeutics (SRPT) announced a strategic restructuring plan aimed at pipeline reprioritization and long-term financial sustainability, resulting in a more than 30% surge in shares during after-market trading [1] Cost-Cutting and Workforce Reduction - The restructuring plan is expected to save approximately $400 million annually starting in 2026, with a workforce reduction of 36%, equating to around 500 employees, projected to save nearly $120 million per year [2] - The company anticipates generating around $300 million in annual savings through pipeline reprioritization [2] Pipeline Focus - Sarepta will pause the development of most gene-therapy candidates for limb-girdle muscular dystrophy (LGMD) but will continue with SRP-9003 for LGMD type 2E/R4, with a regulatory filing to the FDA expected before year-end [3] - The focus has shifted to siRNA programs acquired from Arrowhead Pharmaceuticals, including four clinical-stage candidates: SRP-1001 (FSHD), SRP-1002 (IPF), SRP-1003 (DM1), and SRP-1004 (SCA2) [4] - Initial data from studies on SRP-1001 and SRP-1003 is anticipated before year-end, and three preclinical programs, including SRP-1005 for Huntington's disease, are set to enter clinical development early next year [5] Financial Expectations - Sarepta expects to incur severance and one-time charges of $32 million to $37 million but anticipates saving over $100 million in annual costs this year [6] - For full-year 2026, the company projects combined adjusted R&D and SG&A expenses to be in the range of $800-$900 million [6] Stock Performance and Market Sentiment - The restructuring aims to address setbacks from safety concerns surrounding Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), which has seen Sarepta's shares decline 85% year-to-date compared to a 2% decline in the industry [7] - The stock price surge reflects investor optimism regarding the restructuring plan's potential to stabilize the company's financial profile and growth trajectory [7] Safety Concerns and Regulatory Actions - Elevidys faces a black box warning due to safety issues linked to two patient deaths from acute liver failure in non-ambulatory DMD patients, leading to a suspension of dosing for this group [10] - Sarepta is developing a new protocol with enhanced immunosuppression to improve the safety of Elevidys administration and plans to submit these findings to the FDA [11] Preliminary Financial Results - Sarepta reported preliminary net product revenues of $513 million for Q2 2025, exceeding estimates, with $282 million attributed to Elevidys [13][14] - The preliminary combined adjusted R&D and SG&A expenses for the quarter were around $294 million, with cash and short-term investments totaling approximately $850 million as of June 2025 [14]

Company Overview - Proactive is a financial news publisher that provides fast, accessible, informative, and actionable business and finance news content to a global investment audience [2] - The company has a team of experienced news journalists who produce independent content across various financial markets [2] Market Focus - Proactive specializes in medium and small-cap markets while also covering blue-chip companies, commodities, and broader investment stories [3] - The content delivered includes insights across sectors such as biotech and pharma, mining and natural resources, battery metals, oil and gas, crypto, and emerging digital and EV technologies [3] Technology Adoption - Proactive is recognized for its forward-looking approach and enthusiastic adoption of technology to enhance workflows [4] - The company utilizes automation and software tools, including generative AI, while ensuring that all content is edited and authored by humans [5]

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, ...

Harmony Biosciences (HRMY) Conference July 17, 2025 10:00 AM ET Speaker0Good morning, everyone, and welcome to HC Wainwright at Home, our virtual fireside chat series. I'm Patrick Trucchio, a senior health care analyst at HCW. Today, we're joined by Jeffrey Dano, president and chief executive officer Kumar Boudur, Chief Medical and Scientific Officer Sandeep Tapadia, Chief Financial Officer and Adam Zeski, Chief Commercial Officer of Harmony Biosciences. Harmony's commercial franchise is anchored by Wakix o ...