Biogen Announces Topline Results from Phase 3 VALOR Study and OLE of Tofersen in SOD1 Amyotrophic Lateral Sclerosis

Biogen Announces Topline Results from Phase 3 VALOR Study and OLE of Tofersen in SOD1 Amyotrophic Lateral Sclerosis

Biogen announced complete results from the phase 3 VALOR study of tofersen (BIIB067), an antisense drug under development for patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). Tofersen did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). However, trends favoring tofersen were seen across multiple secondary and exploratory measures of biologic activity and clinical function. Biogen is presenting the data on October 18 at the American Neurological Association 2021 Annual Meeting.  

Study Details

VALOR was a 28-week Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety and tolerability, pharmacodynamic, and biomarker effects of tofersen 100 mg in adults with ALS associated with a SOD1 mutation. A total of 108 patients were randomized (n=72 to tofersen 100 mg and n=36 to placebo). Sixty of these participants met the study’s protocol-defined enrichment criteria for rapid disease progression, comprising the primary analysis population (“faster progressing”). Forty-eight participants did not meet these prognostic enri...

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