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Biogen Prepares More Doubtful Phase III Results For FDA Scrutiny, This Time In ALS

Will Hope To Ride On Coattails Of Amylyx’s Thumbs Up

 
• By Andrew McConaghie

After the Aduhelm controversy, Biogen has another neurodegenerative disease drug which so far can only show efficacy against surrogate biomarkers – will the FDA be swayed again this time?

Biogen
Even if approved, tofirsen looks unlikely to be a big earner for Biogen, which is depending on other late-stage candidates to revive its fortunes.

Biogen is preparing to talk to the US Food and Drug Administration about its amyotrophic lateral sclerosis (ALS) candidate tofersen, and is hopeful of approval, even though the drug failed its pivotal study.

The company first announced disappointing topline data from its Phase III VALOR study evaluating tofersen for the treatment of a subtype of the disease, superoxide dismutase 1 (SOD1) ALS in October 2021. These showed the drug failed to produce a significant improvement versus placebo on the primary endpoint of the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) at 28 weeks in faster-progressing SOD1-ALS patients

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