Regeneron's Cemdisiran Shows Promise in Myasthenia Gravis Trial

Regeneron Pharmaceuticals has announced a successful Phase 3 trial for its RNA-based therapy cemdisiran, developed with Alnylam Pharmaceuticals, for the treatment of generalized myasthenia gravis, a rare autoimmune disorder. Cemdisiran, as a monotherapy, demonstrated improved clinical outcomes compared to the current standard of care, including a significant reduction in symptom severity. The trial results support the potential of cemdisiran as a treatment option for patients with this debilitating condition.

Regeneron Pharmaceuticals (NASDAQ: REGN) has announced positive results from a Phase III clinical trial evaluating cemdisiran, an investigational RNA interference (siRNA) therapy for generalized myasthenia gravis. The study, conducted in collaboration with Alnylam Pharmaceuticals (NASDAQ: ALNY), demonstrated that cemdisiran, either as a standalone treatment or in combination with Veopoz, Regeneron’s complement inhibitor, significantly improved patients’ ability to perform daily activities [1].

Generalized myasthenia gravis is characterized by muscle weakness caused by disruptions in communication between nerves and muscles. Cemdisiran targets the complement system, which plays a role in the disease’s progression. In the trial, patients receiving cemdisiran showed measurable improvements in their capacity to carry out routine tasks compared to baseline assessments [1].

The Phase III NIMBLE study randomized patients to receive cemdisiran 600 mg every three months, cemdisiran 200 mg and pozelimab 200 mg every month, or placebo every month. The monotherapy arm of cemdisiran met the primary endpoint, showing a 2.3-point placebo-adjusted improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score [2].

The trial results are significant as they support the potential of cemdisiran as a treatment option for patients with this debilitating condition. Regeneron plans to submit regulatory filings for cemdisiran in 2026 based on these results [1].

References:
[1] https://www.geneonline.com/regeneron-reports-phase-iii-trial-success-for-cemdisiran-in-treating-generalized-myasthenia-gravis/
[2] https://www.nasdaq.com/articles/regenerons-phase-3-nimble-study-cemdisiran-generalized-myasthenia-gravis-meets-primary