Stop me if you think you’ve heard this one before. A Biogen neurodegenerative disease prospect has failed a pivotal study, but the Big Biotech has latched onto biomarker data and “trends favoring” the treatment as it heads into talks with the FDA. Will it be enough to get the candidate to market?
The drug this time around is tofersen, an antisense prospect licensed from Ionis Pharmaceuticals, and the indication is a subset of amyotrophic lateral sclerosis (ALS). With only a tiny fraction of ALS patients having the genetic mutations targeted by tofersen, the financial stakes for Biogen are much lower than with Aduhelm, with Jefferies analysts putting peak sales at up to $300 million.