Shares of Sarepta Therapeutics Inc. (NASDAQ:SRPT) plunged to $19.91, down $16.27, or almost 45%, on word of a second death by acute liver failure (ALF) with Elevidys (delandistrogene moxeparvovec), the approved gene therapy for patients with Duchenne muscular dystrophy (DMD). Both ALF cases have turned up in non-ambulatory DMD patients. The drug is partnered globally by Sarepta with Roche AG, (OTCMKTS: RHHBY) shares of which were trading at $40.89, down 43 cents. Elevidys was given the U.S. FDA’s full green light about a year ago, and the first ALF death surfaced in March of this year.

For $795M, Sage is added to Supernus’ CNS quiver

Supernus Pharmaceuticals Inc. is buying Sage Therapeutics Inc. for about $795 million. The deal brings Supernus, already firmly in the CNS market, the only U.S. FDA-approved oral treatment for postpartum depression. Zurzuvae (zuranolone), approved in August 2023, brought Sage $36.1 million in net sales for 2024 and another $13.8 million in the first quarter of 2025. Supernus already has Qelbree for attention deficit hyperactivity disorder in for those ages 6 to 17 years, the subcutaneous apomorphine infusion device Onapgo for motor fluctuations in adults with advanced Parkinson’s disease and Gocovri for levodopa-induced dyskinesia. Sage’s stock (NASDAQ:SAGE) surged at midday, with shares selling at $9.10 each, a boost of 35%.

Astrazeneca makes $5.3B drug R&D pact with CSPC

CSPC Pharmaceutical Group Ltd. announced June 13 that it struck a potential $5.33 billion deal with Astrazeneca plc to develop novel preclinical small-molecule candidates using CSPC’s AI-driven drug development platform. The strategic cooperation with Astrazeneca is one of three $5 billion deals that CSPC hinted at earlier this month on the Hong Kong stock exchange, the company confirmed to BioWorld June 16.

‘Be Part of Research,’ UK government urges with NHS app

The U.K. government has announced the latest measures to speed up and expand clinical trials, launching “Be Part of Research,” a central national register where people can search and sign up to take part in studies. The register will be part of the National Health Service (NHS) smartphone app, which is used to book appointments, get reminders and receive test results. At present, there are 20 million users, and with £50 million (US$68 million) of planned new investment in the system, this is due to increase. As Be part of Research evolves, the intention is that the NHS app will automatically match registered patients based on their health data, and send push notifications to their phones when relevant new trials open.

BIO 2025: Building biosecurity through public-private partnership

The COVID-19 pandemic sent the world into a tailspin, raising ongoing concerns about biosecurity, a subject that encompassed the better part of the morning June 14, the first day of the Biotechnology Innovation Organization’s annual conference in Boston. Developing vaccines and therapeutics to combat a virus that has killed more than 7 million people worldwide in the past five years, according to the World Health Organization, is one thing; supplying those products globally and throughout the U.S. is quite another, a feat often dependent on access to materials sourced in China and India. Another major concern is pulling together the funding and public-private partnerships needed to incentivize companies to conduct R&D before an emergency situation arises. “There are alarming gaps in antiviral R&D that if not corrected will leave us extremely vulnerable” to another pandemic, Susan Dentzer, president and CEO of America’s Physician Group, told attendees.

Wojcicki, TTAM win 23andme bidding

The 23andme saga may be coming full circle with founder Anne Wojcicki regaining control in a bidding battle against Regeneron Pharmaceuticals Inc. TTAM Research Institute, a nonprofit benefit organization led by Wojcicki bid $305 million, topping Regeneron's bid of $265 million.

Also in the news

Astrazeneca, Astria, Beam, Bioarctic, Biocryst, Biorestorative, Celldex, Centivax, CSPC, Courage, Enliven, Incyte, Indaptus, Ionis, Johnson & Johnson, Kancera, Keymed, Merck, Moderna, Parallel, Quince, Rebel, Silence, Specialised, Sumitomo, Tonix, Urogen, Xediton