On September 23, Ionis Pharmaceuticals rose 5.9% overnight, trading at $48.45/share, with turnover of $259,700.

On the news front, Ionis Pharmaceuticals and Otsuka Pharmaceutical announced that the Phase 3 Fusion study of ulefnersen successfully met its primary endpoint, demonstrating statistically significant improvement in survival and function in patients with FUS gene mutation-associated amyotrophic lateral sclerosis (ALS). The trial also met key secondary endpoints, significantly reducing a critical nerve damage biomarker and delaying death or the need for permanent ventilation.

Ulefnersen is an antisense oligonucleotide therapy designed to intervene in the disease process at its genetic root. If ultimately approved, it would become the first potential disease-modifying therapy for FUS-ALS, filling an unmet medical need in this rare disease indication. The companies stated they plan to engage with the FDA and other regulatory authorities regarding potential expedited approval pathways. Separately, the FDA PDUFA target action date for the company's another drug, Zilganersen for Alexander disease, was also set for September 22.

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