Regeneron Pharmaceuticals, licensing siRNA drug cemdisiran from Alnylam, announced positive Phase III trial outcomes in generalized myasthenia gravis. As a monotherapy, cemdisiran significantly reduced complement factor 5 levels and improved clinical activity scores. The firm plans to pursue regulatory approval in early 2026. The therapy addresses a rare autoimmune disease characterized by progressive muscle weakness, and its success highlights advancements in RNA-based therapeutics within neuromuscular disorders.