Fresh hope for tofersen in ALS

After missing the primary endpoint in the six-month Phase III VALOR study last year, 12-month data from the open-label follow-up of tofersen suggest longer treatment with the Biogen antisense therapy in SOD1-mutant ALS may better reduce disease progression.

Amyotrophic lateral sclerosis patients who initiated treatment at the start of the study had less disease progression than patients who initiated treatment six months later at the start of the open-label extension, with a 3.5-point difference (p=0.027) in clinical function as measured by Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score, and a 9.2% predicted difference in respiratory function as measured by slow vital capacity (p=0.016), Biogen Inc. (NASDAQ:BIIB) said. ...