Cemdisiran Excels in Phase 3 Myasthenia Gravis Trial

Regeneron Pharmaceuticals reported positive topline results from the Phase 3 NIMBLE trial evaluating investigational cemdisiran, an siRNA targeting complement factor 5 (C5), as monotherapy for adults with generalized myasthenia gravis (gMG). The trial, involving patients with anti-acetylcholine receptor antibodies, randomized participants to receive cemdisiran (600 mg subcutaneously every 12 weeks), a cemdisiran-pozelimab combination (200 mg each every 4 weeks), or placebo.