Shares of Ionis Pharmaceuticals fell 12% after the company said a study for tofersen missed its primary endpoint, as reported in Morningstar.
Ionis said its partner Biogen reported topline results from a placebo-controlled study of tofersen being evaluated for people with superoxide dismutase 1 amyotrophic lateral sclerosis (SOD1-ALS).
Tofersen missed its primary endpoint, but trends favouring tofersen were seen across multiple secondary and exploratory measures of disease activity and clinical function.
Biogen, which licensed the drug from Ionis in 2018, said early tofersen initiation led to less decline across multiple measures including motor function, respiratory function, muscle strength, and quality of life in people with SOD1-ALS.
Biogen will expand eligibility for its ongoing early access program to all people with SOD1-ALS in countries where programs are permitted by local regulations and future access may be secured.
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