Progress on a rare neurodegenerative therapy’s path toward pivotal testing, outlining company-aligned trial plans, study design choices, and upcoming development milestones.
Key Takeaways
- 50 mg twice-daily dose produced a 48% slowing of progression versus placebo
- Regulators agreed on a 12-month trial using the 11-item UMSARS Part I
- Pivotal trials plan about 200 patients randomized 1:1 and to start by year-end 2026