On September 29, Ionis Pharmaceuticals rose 5.15% in regular trading, trading at $46.82/share, with turnover of $83.55 million. The rally was driven by two consecutive Phase 3 trial successes within a week, compounding the positive momentum from an earlier FDA approval.

On September 22, Ionis and Otsuka announced that ulefnersen met its primary endpoint in the Phase 3 Fusion study, significantly improving survival and function in patients with FUS-mutant amyotrophic lateral sclerosis. The trial also met key secondary goals, lowering a nerve damage marker and delaying death or the need for permanent ventilation. One day later, Ionis and Roche reported that sefaxersen achieved a statistically significant reduction in proteinuria in the Phase 3 IMAgINATION trial for primary IgA nephropathy at week 37, with a consistent safety profile.

These back-to-back successes, combined with the FDA approval of zilganersen for Alexander disease earlier in September, have effectively repaired investor sentiment that was damaged in July when the eplontersen Phase 3 trial in amyloid cardiomyopathy failed to meet its primary endpoint, which had sent shares down over 20% at the time.

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