Regeneron says Phase 3 NIMBLE trial met endpoints for cemdisiran in generalized myasthenia gravis
Regeneron said the Phase 3 NIMBLE trial of cemdisiran in generalized myasthenia gravis met primary and key secondary endpoints at week 24. The company said a U.S. regulatory application has been submitted and EU filings are planned for 2026.
Regeneron Pharmaceuticals said detailed positive results from the Phase 3 NIMBLE trial evaluating investigational cemdisiran in adults with generalized myasthenia gravis (gMG) were published in The Lancet and presented in an oral plenary session at the American Academy of Neurology Annual Meeting. The company said the trial met its primary and key secondary endpoints at week 24, with cemdisiran dosed subcutaneously every 12 weeks demonstrating rapid, deep and sustained disease control.
At week 24, cemdisiran versus placebo showed a 2.3-point placebo-adjusted MG-ADL improvement with p<0.001 and a 2.8-point placebo-adjusted QMG improvement with p=0.002. The company said 76.6% of cemdisiran patients achieved a 3-point or greater MG-ADL improvement and 48.4% achieved a 5-point or greater QMG improvement.
Cemdisiran is a novel siRNA therapeutic that durably reduces circulating levels of complement factor 5 (C5), allowing for every three months dosing. The company said cemdisiran could be the first siRNA to be approved for the treatment of gMG.
Regeneron said a U.S. regulatory application has been submitted and that EU filings are planned for 2026. The article also said one death from pneumonia occurred in the extension period.