Three and a half years after Biogen and Ionis Pharmaceuticals won approval for Qalsody to treat a genetic form of ALS, Ionis with partner Otsuka Pharmaceutical is back with another clinical win that could offer hope to patients with a different subtype of the disease.
Ulefnersen, an antisense oligonucleotide therapy, showed a statistically significant improvement versus placebo in functional impairment and survival in 73 patients with a genetic subtype of ALS caused by mutations in the fused in sarcoma (FUS) gene, meeting the Phase 3 FUSION trial’s primary objective.