Top-line data from a phase II trial of Italfarmaco SpA's givinostat in men with Becker muscular dystrophy found it failed to affect a significant change in total muscle fibrosis vs. placebo, the study's primary endpoint. But whether the outcome reflected a shortcoming of the drug or a function of the rare condition's clinical profile remained uncertain, with the company placing its bets on the latter.
CEO John Leonard said Intellia Therapeutics Inc. plans “to share information on a cohort-by-cohort basis, so we get a consistent readout” and, as the year goes on, longer-term follow-up findings will emerge from the phase I trial with the company’s lead in vivo genome editing candidate, NTLA-2001.
A new study from researchers at the Washington University School of Medicine In St. Louis demonstrated evidence of a long-lasting immune response, possibly one that could last for years, from receiving Pfizer Inc.’s mRNA COVID-19 vaccine.
LONDON – New data have arrived to allay concerns about shortages of COVID-19 vaccines disrupting dosing schedules, and also answering the question of whether third – booster – doses will restore waning immunity and be effective against viral variants.
Swiss biotech Polyphor AG is facing an uncertain future after a pivotal phase III study of the company’s balixafortide in HER2-negative breast cancer showed the drug did not improve response rates compared with standard therapy alone.
Fulcrum Therapeutics Inc.’s phase IIb data with losmapimod in facioscapulohumeral muscular dystrophy (FSHD) brought renewed hope for patients in what historically has proved a challenging therapeutic space. Though the firm’s oral p38 mitogen-activated protein kinase inhibitor missed its primary biomarker endpoint – changes in DUX4-driven gene expression – other indicators of benefit in the study called ReDUX4 painted a bright picture.
Wall Street must wait a while longer to find out if Gilead Sciences Inc. will exercise its $275 million option for Arcus Biosciences Inc.’s TIGIT binder, domvanalimab. Meanwhile, investors took heart from an optimistic – albeit vague – interim report on the phase II ARC-7 trial.
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