European Investor
21 Apr 2026, 18:58
Regeneron reported positive Phase 3 results for its investigational therapy cemdisiran in patients with generalized myasthenia gravis (gMG), demonstrating rapid, deep, and sustained disease control. The NIMBLE trial met both its primary and key secondary endpoints at 24 weeks, showing significant improvements in daily functioning and muscle strength compared to placebo.
Patients receiving cemdisiran experienced clinically meaningful benefits within two weeks, with effects sustained throughout the study period. The therapy, administered subcutaneously every 12 weeks, also offers a reduced treatment burden compared to existing options.
The safety profile was generally consistent, with most adverse events mild to moderate and no new major safety concerns identified.
Regeneron has already submitted a U.S. regulatory application and plans additional filings in other regions, positioning cemdisiran as a potential first siRNA-based treatment for gMG.
Globe Newswire
Patients receiving cemdisiran experienced clinically meaningful benefits within two weeks, with effects sustained throughout the study period. The therapy, administered subcutaneously every 12 weeks, also offers a reduced treatment burden compared to existing options.
The safety profile was generally consistent, with most adverse events mild to moderate and no new major safety concerns identified.
Regeneron has already submitted a U.S. regulatory application and plans additional filings in other regions, positioning cemdisiran as a potential first siRNA-based treatment for gMG.
Globe Newswire
