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2 days ago
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Pharma's turbo spending spree on US manufacturing
This story was originally published on PharmaVoice. To receive daily news and insights, subscribe to our free daily PharmaVoice newsletter. Investments in U.S. drug manufacturing are skyrocketing. Pharma giants everywhere have announced massive, multi-year deals to build and expand facilities across the country as several market factors make the U.S. a favorable destination for producing branded drugs. The looming threat of U.S. tariffs remains a motivator for companies from around the globe. Earlier this month, President Donald Trump said tariffs as high as 200% on pharma products would be introduced 'very soon.' Other industry shifts, including the growing GLP-1 market and the demand for skilled workers in pharma's tech-heavy manufacturing environment, are also tipping the scales toward the U.S. Amid the investment boom, tens of billions of dollars are pouring into manufacturing hubs like Raleigh, North Carolina, and generating thousands of job openings for engineers, R&D personnel, operations professionals and more. Here's a look at the latest companies to hop on the U.S. manufacturing bandwagon. AstraZeneca makes its largest-ever single facility investment AstraZeneca joined the $50 billion club this month when it announced a sprawling manufacturing and R&D investment in the U.S. The size of the investment matches the other leading pledges from pharma companies this year — including Roche, Johnson & Johnson and Eli Lilly — to each pump $50 billion or more into U.S. facilities. CEO Pascal Soroit acknowledged that tariffs played a part in the U.K.-based company's decision to pump up its U.S. operations in a recent interview with Fortune, but also noted that Europe is 'losing ground' as a drug production destination by focusing on 'social benefits and managing costs.' AstraZeneca's plan includes $4 billion for a drug substance facility in Virginia — its largest single-facility manufacturing investment in the world. But the $50 billion in funds will stretch across the country and include an R&D expansion in Maryland, a continuous manufacturing expansion in Indiana, a next-generation cell therapy facility in Texas, a 'state of the art' R&D center in Massachusetts' biotech hub at Kendall Square and more. The American market for drugs also plays a starring role in the company's broader ambitions. All told, AstraZeneca aims to boost its total revenue from the $54 billion-mark it hit in 2024 to over $80 billion by 2030 — half of which it expects to be generated in the U.S., the company said. Biogen pours more into North Carolina Biogen is broadening its manufacturing footprint in North Carolina. The company said it will invest $2 billion into two campuses in the state's Research Triangle Park, Biogen's current hub for manufacturing. The fresh funds will expand Biogen's capacity for multiple modalities, including antisense oligonucleotides, while modernizing its operations with automation and AI. Biogen is still working to move past its Aduhelm debacle. The Eisai-partnered Alzheimer's drug notched a historic approval in 2021 but was later pulled from the market amid cost and efficacy controversies. Its other Alzheimer's treatment Leqembi has had a stronger showing on the market and has steadily risen in sales since last year. But Biogen has also been in cost-cutting mode since 2023 and expects revenue to decline this year. Going forward, the company is leaning on its pipeline, including treatments for Dravet syndrome, a rare form of epilepsy, to set the stage for future growth. Its latest manufacturing investment adds to the $10 billion it has funneled into North Carolina since setting up shop at Research Triangle Park in 1995. Thermo Fisher snags Sanofi facility to boost fill-finish chops Thermo Fisher Scientific is expanding its production prowess with the help of a growing Sanofi partnership. The companies said this month that Thermo Fisher is snapping up a sterile, fill-finish manufacturing site from the French pharma for an undisclosed sum. The move comes amid higher-than-expected profit last quarter for Thermo Fisher, one of the pharma industry's key suppliers of clinical research services and equipment. The new digs will help Thermo Fisher 'strengthen' its U.S. manufacturing capabilities for the broader pharma industry, the company stated. The latest move is also a part of the four-year, $2 billion commitment to U.S. manufacturing Thermo Fisher announced in April. Recommended Reading Tracking a moving target: Drugmakers brace for a looming tariff impact
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3 days ago
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Sarepta stock falls after Elevidys fails to get backing by EU regulators
-- Sarepta Therapeutics Inc. (NASDAQ:SRPT) stock declined 13% Friday after European regulators recommended against approving Elevidys, the company's gene therapy for Duchenne muscular dystrophy. The European Medicines Agency's drug advisory committee (CHMP) rejected Elevidys for children aged 3 to 7 years who are able to walk, the agency announced Friday. The therapy, which is marketed outside the U.S. by Roche Holding AG (SIX:ROG), was seeking approval for treating the rare genetic disease that causes progressive muscle weakness. This setback follows closely on the heels of the U.S. Food and Drug Administration's recent request for a pause on Elevidys use in the United States. Sarepta had already announced a temporary halt on U.S. shipments earlier this week. Roche has also paused shipments in jurisdictions that reference FDA approvals, further limiting the therapy's market reach. The Swiss pharmaceutical giant had been responsible for commercializing the treatment in markets outside the U.S. after partnering with Sarepta. Duchenne muscular dystrophy is a rare genetic disorder that primarily affects boys and leads to progressive muscle deterioration. The condition is ultimately fatal, with patients typically experiencing serious mobility limitations and shortened lifespans. The dual regulatory setbacks in both the U.S. and European markets represent significant challenges for Sarepta's commercialization strategy for Elevidys, which had been positioned as a breakthrough treatment option for the debilitating condition. Related articles Sarepta stock falls after Elevidys fails to get backing by EU regulators Victoria's Secret Exposed: The Warning Sign Behind the Stock's 52% Collapse Surge of 50% since our AI selection, this chip giant still has great potential Sign in to access your portfolio
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Sarepta Therapeutics Acknowledges CHMP Negative Opinion for ELEVIDYS in the European Union
Partner Roche will continue its dialogue with the European Medicines Agency to explore a potential path forward to make ELEVIDYS available to individuals living with Duchenne muscular dystrophy in the EU ELEVIDYS is the first and only disease-modifying gene therapy for Duchenne CAMBRIDGE, Mass., July 25, 2025--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, acknowledges that the Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization (CMA) for ELEVIDYS (delandistrogene moxeparvovec) in ambulatory individuals ages three to seven years for the treatment of Duchenne muscular dystrophy (DMD). "While we are disappointed by the CHMP's negative opinion, we understand the urgent need for continued dialogue and collaboration to bring transformative therapies to people with Duchenne who live with a relentless disease that steals their mobility, independence and ultimately life – often by early adulthood," said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta. "Following the initial FDA approval of ELEVIDYS on June 22, 2023, the therapy has subsequently received regulatory approval in several other countries. In the U.S., we are actively working with the FDA to address recent safety questions. We remain committed to working with regulators to address outstanding questions on safety so that people living with Duchenne have access to this important therapy." ELEVIDYS is the first and only approved gene therapy targeting the underlying cause of disease that has consistently demonstrated stabilization or slowing of DMD disease progression, with durable effects on functional and biological outcomes and muscle health. While the primary endpoint was not met in EMBARK after one year, ELEVIDYS showed clinically meaningful and statistically significant improvements across important secondary endpoints of functional outcome measures when compared to placebo. Longer term efficacy data were also submitted to EMA, including two-year results from the EMBARK study and three-year pooled efficacy analysis from three other ELEVIDYS studies that showed clinically meaningful improvements across key measures of motor function. One-year data from part one of the EMBARK study were published in Nature Medicine in October 2024, and results from year two were shared at this year's Muscular Dystrophy Association Clinical & Scientific Conference in Dallas. Quantitative muscle MR (magnetic resonance) outcomes from part 1 of EMBARK were published in JAMA Neurology in May 2025. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world. Regulatory approval and commercialization of ELEVIDYS in Japan is through Chugai Pharmaceuticals via its alliance with Roche. About ELEVIDYS (delandistrogene moxeparvovec-rokl)ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is indicated in U.S. for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age. For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory patients is approved under accelerated approval in the U.S. based on expression of ELEVIDYS micro-dystrophin (noted hereafter as "micro-dystrophin") in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). U.S. IMPORTANT SAFETY INFORMATION CONTRAINDICATION: ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene. WARNINGS AND PRECAUTIONS: Infusion-related Reactions: Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment. Once symptoms resolve, the infusion may be restarted at a lower rate. ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available. Discontinue infusion for anaphylaxis. Acute Serious Liver Injury: Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks. Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled. Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels). Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended. Immune-mediated Myositis: In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed. Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction. Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient's clinical presentation and medical history if these symptoms occur. Myocarditis: Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials. If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath. Advise patients to contact a physician immediately if they experience cardiac symptoms. Preexisting Immunity against AAVrh74: In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies. Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration. ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400. Adverse Reactions: The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia. Report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782). For further information, please see the full Prescribing Information. About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (Duchenne) and limb-girdle muscular dystrophies (LGMDs) and are building a robust portfolio of programs across muscle, central nervous system, and cardiac diseases. For more information, please visit or follow us on LinkedIn, X, Instagram and Facebook. Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website at We encourage investors and potential investors to consult our website regularly for important information about us. Forward-Looking StatementsThis statement contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "will," "may," "potential" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements relating to our future operations, research and development programs, discussions with regulators and the prospects for approvals or continued approvals, as applicable, of ELEVIDYS and the potential benefits and risks of ELEVIDYS. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: different methodologies, assumptions and applications we use to assess particular safety or efficacy parameters may yield different statistical results; our products or product candidates may be perceived as insufficiently effective, unsafe or may result in unforeseen adverse events; our products or product candidates may cause undesirable side effects that result in significant negative consequences; the possible impact of regulatory decisions by, and any halts imposed by, regulatory agencies on our business; and those risks identified under the heading "Risk Factors" in our most recent Annual Report on Form 10-K for the year ended December 31, 2024 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company, which you are encouraged to review. Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained herein. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law. Source: Sarepta Therapeutics, Inc. View source version on Contacts Investor Contact: Ian Estepan617-274-4052iestepan@ Media Contacts: Tracy Sorrentino617-301-8566tsorrentino@ Kara Hoeger617-710-3898KHoeger@
