Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
Alzheon Inc. has raised $100 million in a series E financing round to push its oral drug candidate for early Alzheimer’s disease (AD), ALZ-801 (valiltramiprosate), through a late-stage, Apolloe4 study.
Quoted biotech investor Syncona Ltd. is still struggling with the fallout from one of the worst bear markets on record, reporting its net asset value fell from £1.25 billion (US$1.58 billion) to £1.24 billion in its 2023-24 financial results on June 20. That is despite a restructuring toward more mature companies, with 70% of the portfolio now invested in clinical-stage assets.
The African Vaccine Manufacturing Accelerator is up and running, with more than $1 billion already pledged to the effort that’s aimed at supporting the sustainable growth of Africa's manufacturing base and contributing to the African Union's goal of producing 60% of the vaccines required by African countries by 2040.
Now that it has some experience with interchangeable biosimilars under its belt, the U.S. FDA is proposing an update to its interchangeability guidance that could eliminate the need for switching studies in some instances.
A nearly two-year-old partial clinical hold has been lifted by the U.S. FDA on PTC Therapeutics Inc.’s pivotal phase II study in Huntington’s disease. The agency had paused enrollment in October 2022, saying it wanted more data on PTC-518, an orally bioavailable small-molecule splicing modifier, before enrollment could continue.
Gilead Sciences Inc. tallied a “clear win,” said Barclays analyst Carter Gould, in the phase III interim analysis showing that the Foster City, Calif.-based company’s twice-yearly injectable HIV-1 capsid inhibitor, lenacapavir, yielded 100% efficacy as an HIV blocker for cisgender women.
China’s National Medical Products Administration (NMPA) approved Dizal Pharmaceutical Co. Ltd.’s golidocitinib for treating adults with relapsed or refractory peripheral T-cell lymphoma whose disease has progressed or was refractory to at least one prior systemic therapy.
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