Biogen and Ionis stumble again

Chalk up yet another disappointment in amyotrophic lateral sclerosis, and another for Ionis and Biogen. Today the partners said they were discontinuing BIIB078 after it failed to show a clinical benefit in a phase 1 trial. There is not much to go on, but worryingly patients receiving a high dose, 90mg, had worsening disease versus placebo; those on 60mg showed no benefit. Stifel analysts reckon this suggests dose-dependent toxicity, which could spell trouble for Ionis’s antisense approach – especially after its Roche-partnered Huntington’s project tominersen was linked with a similar phenomenon. And Biogen is particularly exposed to this technology, having paired up with Ionis on various CNS antisense projects. The Sod1 antisense tofersen is still in play in presymptomatic ALS, but its future looks dim after the failure of the Valor trial last year. While BIIB078 and tofersen were designed for specific genetic subtypes of ALS, the partners also have a potentially universal therapy, BIIB105, in development; and Ionis has a wholly owned phase 3 asset, jacifusen (ION363), for ALS with mutations in the FUS gene. Still, data on the latter are some way off. More immediately, an adcom this week will discuss the approval chances of Amylyx’s AMX0035.