Tofersen never was going to be a large-market, major revenue-driving drug for Biogen, Inc. But with multiple setbacks over the last several months plus commercial challenges, disappointing Phase III results for the drug in amyotrophic lateral sclerosis (ALS) patients with a superoxide dismutase 1 (SOD1) gene mutation is another badly timed setback for the company and its partner, Ionis Pharmaceuticals, Inc.
Top-line results from the Phase III VALOR clinical trial and an open-label extension study of tofersen – an antisense oligonucleotide that binds to SOD1 mRNA to prevent production of the SOD1 protein – were presented on 17 October during the American Neurological Association (ANA) annual meeting
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