Ionis/Otsuka ALS Drug Hits Primary Goal in Late-Stage Trial
IONS is trading near its 52-week low of $44.2 (5.0% above the low).
Summary
Ulefnersen, Ionis's partnered ALS drug with Otsuka, met its primary endpoint in a Phase 3 trial for FUS-ALS, a rare inherited form of the disease with no approved treatments. The drug improved function and survival versus placebo, reduced nerve damage markers, and showed a favorable safety profile. Ionis Pharmaceuticals says ulefnersen demonstrates favorable safety and tolerability profile. This is a major pipeline win for Ionis, which has faced recent setbacks including the eplontersen ATTR-CM failure and pelacarsen miss. The companies plan to pursue accelerated approval with the FDA and other regulators, potentially bringing the first targeted therapy for FUS-ALS to market. An early access program has also been launched, underscoring the unmet need and commercial urgency.
At the time of this announcement, IONS was trading at $46.42 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $7.5B. The 52-week trading range was $44.20 to $86.74. This news item was assessed with positive market sentiment and an importance score of 9 out of 10. Source: Reuters.