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Sarepta's Flagship Gene Therapy Gets the FDA's Most Severe Warning After Two Boys Die
Two boys with Duchenne muscular dystrophy died of acute liver failure after receiving Sarepta's gene therapy Elevidys, prompting the FDA to add a black box warning and strip the drug's non-ambulatory indication entirely. The company is now cutting roughly 500 jobs (36% of its workforce) to save $400 million annually, while quarterly U.S. revenue has already dropped to about $100 million, down sharply from its peak. Several pipeline programs are being paused or shelved as Sarepta fights to meet its 2027 financial obligations.
Why it matters: This is the gene therapy field's most serious safety crisis to date. Combined with similar liver toxicity deaths linked to Novartis's Zolgensma, it raises uncomfortable questions about whether the AAV viral vectors underlying most gene therapies can ever fully escape this risk.
Read more →Deals and M&A
Supernus and Indivior Merge to Build a $2.2 Billion Brain-Drug Giant
Two mid-cap pharma companies are combining in an all-stock merger of equals that would create a CNS powerhouse spanning addiction, ADHD, depression, and Parkinson's. Indivior shareholders pocket a $1 billion special cash dividend before closing. The merged company projects $125 million in annual cost synergies, but mergers of equals have a notoriously mixed track record in pharma.
Read more →Sandoz Drops $322 Million on a Shanghai Biosimilar Shopping Spree
The world's largest standalone biosimilar company licensed up to ten products from Shanghai's Henlius Biotech, including copies of cancer drug Erbitux and cholesterol blockbuster Repatha. The deal could push Sandoz's pipeline to 46 assets, reinforcing its post-Novartis strategy and the broader trend of Western pharma sourcing its next wave of biologics from China.
Read more →Sentynl Bets Up to $475 Million on a Lung Disease Most Doctors Miss
Rare disease specialist Sentynl licensed alvelestat from Mereo BioPharma through a clever option-to-license structure, targeting the first oral treatment for alpha-1 antitrypsin deficiency lung disease. The Phase 3 trial could begin in early 2027, aiming to reach up to 100,000 underdiagnosed Americans. The deal's architecture lets Sentynl walk away if the data disappoint.
Read more →A Werewolf Just Became a Painkiller Company (Seriously)
Werewolf Therapeutics, a failing cancer immunotherapy company, agreed to a $150 million reverse merger with Ambros Therapeutics and its non-opioid painkiller for complex regional pain syndrome. The oversubscribed financing was led by RA Capital and Janus Henderson. Ambros shareholders will own 71.7% of the combined company, making this a public listing in disguise.
Read more →Clinical and Regulatory
Capricor's Duchenne Drug Faces Likely FDA Rejection as Activist Investors Storm the Boardroom
An FDA advisory committee voted 9-to-3 against Capricor's cell therapy for Duchenne muscular dystrophy, and activist investor Kaos Capital is simultaneously demanding a full governance overhaul. The PDUFA date is August 22, and analyst price targets range widely, reflecting what is essentially a coin flip with a ticker symbol.
Read more →A Polish Company Nobody Knows Just Matched Ozempic in an Early Study
Celon Pharma's semaglutide copycat, Reduzek, hit bioequivalence against both Ozempic and Wegovy in a 91-person Canadian study. The strategic choice of Canada was deliberate: Novo Nordisk's semaglutide patent there has lapsed. U.S. patents hold until at least 2032, but the global GLP-1 pricing war just quietly started.
Read more →The Target Big Pharma Couldn't Crack for 20 Years Just Got Its First Drug Filing
China's NMPA accepted a priority review filing for opamtistomig, a bispecific antibody from Leads Biolabs that targets both PD-L1 and the long-elusive 4-1BB pathway. If approved for advanced neuroendocrine carcinoma, it would be the first 4-1BB therapy to reach market after two decades of industry failures involving liver toxicity and weak potency.
Read more →Policy and Industry
Inside Kennedy's Latest Move to Reshape Federal Vaccine Policy
A new HHS document reveals RFK Jr.'s plans to create entirely new categories for vaccine recommendations and offer single-vaccine alternatives to the MMR combo shot. It follows 18 months of systematic changes, including firing all 17 ACIP members and canceling $500 million in mRNA vaccine funding. A 30-day comment period closes in late September, but the direction is already clear.
Read more →The $2 Billion Biotech Factory That's Shutting Half Its Lights Off
National Resilience closed six U.S. manufacturing sites after a separate lease-holding affiliate filed for bankruptcy—a case a court later dismissed for lack of good faith—keeping only its Cincinnati facility (which makes Eli Lilly's GLP-1 blockbusters) and Toronto operations. The company raised over $2 billion since 2020 to build domestic biomanufacturing capacity, but the biotech funding winter left those shiny new factories without enough customers.
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