Regeneron Reports Positive Phase 3 Data For Cemdisiran In Myasthenia Gravis

(RTTNews) - Regeneron Pharmaceuticals (REGN) reported positive Phase 3 results for cemdisiran in Generalized Myasthenia Gravis, with the NIMBLE trial meeting primary and key secondary endpoints at 24 weeks.

Cemdisiran-treated patients showed significant improvements in MG-ADL and QMG scores versus placebo, with placebo-adjusted gains of 2.3 points and 2.8 points, respectively.

Clinical benefits appeared within two weeks and were sustained through the study period. Notably, 76.6% of patients achieved =3-point MG-ADL improvement compared to 44.1% on placebo.

The investigational siRNA therapy targets complement factor C5 and is administered once every 12 weeks, offering a potentially more convenient treatment option.