ANA 2021 – Ionis rocked by tofersen flop

The amyotrophic lateral sclerosis field has had more than its fair share of disappointments. The latest one came yesterday, with the failure of Biogen and Ionis’s Sod1-targeted project tofersen in the phase 3 Valor study.

Bulls are clinging to a decrease seen in a biomarker of axonal damage, neurofilament light chain, with some believing that this could pave the way to an accelerated approval. Even sellside analysts poured cold water on this idea, but the companies do have another shot with an ongoing trial in presymptomatic ALS.