Cemdisiran Achieves 76.6% Responder Rate; U.S. Filing Submitted
Regeneron’s Phase 3 NIMBLE trial of subcutaneous cemdisiran every 12 weeks met its primary endpoint with a placebo-adjusted 2.3-point MG-ADL improvement and 76.6% responder rate at 24 weeks, and showed a 2.8-point QMG gain (p<0.002). Regeneron filed its U.S. regulatory application in Q1 2026 and plans EU submissions.
1. Phase 3 NIMBLE Trial Results
In the NIMBLE trial, adults with gMG receiving subcutaneous cemdisiran every 12 weeks (n=64) achieved a least-squares mean MG-ADL improvement of 4.5 points versus 2.2 for placebo (placebo-adjusted 2.3 points, p<0.001) and a QMG improvement of 4.2 versus 1.5 (placebo-adjusted 2.8 points, p=0.002), with rapid benefits within two weeks and sustained through 24 weeks.
2. Safety and Tolerability
During the double-blind period, 69.2% of cemdisiran patients experienced treatment-emergent adverse events versus 77.1% on placebo, most mild to moderate. Infection rates were 27% versus 40%, no serious or meningococcal infections occurred, and there were no treatment discontinuations due to adverse events in the cemdisiran arm.
3. Regulatory Filings
Regeneron submitted its U.S. regulatory application for cemdisiran in Q1 2026 and plans additional filings in the European Union later in 2026. The company will host a virtual roundtable on April 22 to discuss its C5 complement program.