Normal service resumes in amyotrophic lateral sclerosis

Two recent FDA approvals in amyotrophic lateral sclerosis bucked the usual trend of disappointments in the neurodegenerative disease. But it was back to business as usual this week, with two stumbles in quick succession from Wave Life Sciences and Apellis. In fact, a look at an Evaluate Vantage analysis of the late-stage ALS pipeline, carried out almost a year ago, shows that over half of these projects have since failed. Some developers are pressing on, with Prilenia and Clene still seeing a future for their assets. Such an optimistic stance is no doubt helped by the fact that Biogen and Ionis’s Qalsody (tofersen) got accelerated approval for Sod1-mutated ALS despite the antisense therapy failing its phase 3 trial, Valor. Perhaps the most optimistic of all is Brainstorm Cell Therapeutics, which after FDA knockbacks chose to file its project Nurown under protest, setting up an advisory committee meeting; a date has not yet been set. No such drama from Wave and Apellis, which have thrown in the towel on WVE-004 and Empaveli respectively. Notably one dose of WVE-004 performed statistically worse than placebo, echoing findings with Biogen and Ionis’s similarly-acting BIIB078 last year.