Clinical TrialApr 21, 2026, 10:32 AM
Regeneron's Cemdisiran gMG trial meets endpoints; US filing submitted
AI Summary
Regeneron Pharmaceuticals announced detailed positive results from the Phase 3 NIMBLE trial for investigational cemdisiran in adults with generalized myasthenia gravis (gMG). The trial met its primary and key secondary endpoints at week 24, demonstrating rapid, robust, and sustained efficacy with convenient quarterly subcutaneous administration. A U.S. regulatory application has been submitted, positioning cemdisiran as a potential first-in-class siRNA for gMG.
Key Highlights
- Cemdisiran achieved a 4.5-point MG-ADL improvement vs. 2.2 for placebo (p<0.001) at week 24.
- 76.6% of cemdisiran patients had a ">=3-point MG-ADL improvement vs. 44.1% for placebo.
- Cemdisiran showed a 4.2-point QMG improvement vs. 1.5 for placebo (p=0.002) at week 24.
- 48.4% of cemdisiran patients had a ">=5-point QMG improvement vs. 19% for placebo.
- Clinically meaningful improvements in MG-ADL and QMG occurred within 2 weeks.