Regeneron’s Cemdisiran Meets Phase III Goals in Myasthenia Gravis Trial

Regeneron’s experimental siRNA therapy, cemdisiran, successfully met the main objectives in a Phase III trial for adults with generalized myasthenia gravis. In the 24‑week study, cemdisiran monotherapy led to a 74 percent reduction in complement protein C5 levels and delivered measurable improvement in patients’ daily activities such as talking and eating. When combined with Regeneron’s approved C5 blocker pozelimab (Veopoz), C5 suppression increased to nearly 99 percent, though cemdisiran alone performed slightly better on key disease‑specific outcomes. No patients discontinued treatment due to adverse events. The company now plans to submit an FDA marketing application in early 2026. This development positions cemdisiran as a promising competitor in the field, potentially offering at‑home dosing and strong efficacy compared to existing treatments.