Biogen’s Experimental Drug Shows Promise for Genetic Form of ALS

Amyotrophic lateral sclerosis (ALS) is a devastating disease with no approved treatments. Diagnosis is tantamount to a death sentence and researchers are desperately searching for some kind of therapy. Although there have been numerous failed treatments, Biogen believes it might be on the right track. “ALS is a disease you really want to make a difference in,” Toby Ferguson, M.D., Ph.D, Senior Medical Director for Neuromuscular Research and Early Development at Biogen told BioSpace. Earlier this month, Biogen announced interim results from a Phase I/II study of tofersen, an antisense oligonucleotide the company licensed from Ionis Pharmaceuticals that is being studied as a potential treatment of ALS patients who have a confirmed superoxide dismutase 1 (SOD1) mutation. The interim results showed both a proof-of-biology and proof-of-concept, which supported the start of a Phase III clinical trial to confirm the efficacy and safety of tofersen in that patient population, Ferguson said. Ferguson said this was exciting news as it showed the potential of tofersen to target the genetic driver of this form of ALS.