STAT+: Biogen drug for rare form of ALS fails pivotal study — another setback for fatal disease with few treatments
Biogen reported disappointing results Sunday from a late-stage clinical trial involving its experimental treatment for a rare, genetically defined form of amyotrophic lateral sclerosis, or Lou Gehrig’s disease.
In the Phase 3 study, the Biogen drug called tofersen failed to slow the neurologic and functional decline of ALS patients compared to a placebo. The drug showed some improvements on other measures of disease progression, the company said, although interpreting those secondary results is challenging because the study’s main goal was not met.
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