Otsuka/Ionis ALS drug clears Phase 3 primary endpoint, eyes expedited review
TL;DR Summary
A Phase 3 trial of ulefnersen, the RNA-targeted ALS drug from Ionis and Otsuka, met its primary endpoint in the rare FUS-ALS form, with 73 of 89 enrolled patients in the primary analysis showing improved outcomes on functional and survival measures and favorable safety. Secondary endpoints and a biomarker signal also favored the drug, supporting discussions with regulators about expedited approval. The rarity of FUS-ALS limits market opportunity; Ionis will collect royalties on sales after a $10 million upfront license, with analysts forecasting modest long-term revenue compared with other Ionis programs.
- Otsuka heads to FDA as Ionis-partnered ALS drug hits primary phase 3 endpoint Fierce Biotech
- Otsuka Establishes Ulefnersen Early Access Program (EAP) for Eligible People Living with FUS-ALS BioSpace
- Ionis Pharmaceuticals Says Ulefnersen Demonstrates Favorable Safety And Tolerability Profile TradingView
- Ionis drug meets primary goal in rare ALS gene mutation trial Investing.com
- ALS Association Statement on Positive Phase 3 Results for Ulefnersen in FUS-ALS PR Newswire
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