On September 23, Ionis Pharmaceuticals rose 5.9% in pre-market trading, trading at $48.46 per share, with turnover of $87,100.

On the news front, the company and Roche announced that an interim analysis of the Phase 3 study of their experimental kidney disease drug sefaxersen in adults with primary IgA nephropathy met its primary endpoint, achieving a statistically significant and clinically meaningful reduction in proteinuria compared with placebo at week 37. The study will continue to evaluate changes in kidney function over two years.

This follows closely on the heels of another major readout: Ionis and Otsuka Pharmaceutical reported that ulefnersen successfully met the primary endpoint in its Phase 3 Fusion trial, significantly improving survival and function in patients with FUS-mutated ALS. The trial also met key secondary goals, lowering a key nerve damage marker and delaying death or permanent ventilation need. If approved, ulefnersen would be the first disease-modifying therapy for FUS-ALS. Additionally, the FDA target review date for Zilganersen in Alexander disease was set for September 22, adding a further regulatory catalyst.

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