New Delhi, Aug. 28 -- Regeneron Pharmaceuticals, together with Alnylam Pharmaceuticals, has reported strong results from a Phase III clinical trial of its investigational therapy cemdisiran for generalized myasthenia gravis (gMG), an autoimmune neuromuscular disorder that causes muscle weakness and fatigue. The pivotal NIMBLE trial achieved both its primary and secondary endpoints, marking an important step toward bringing a new treatment option to patients.

Trial Outcomes The trial tested cemdisiran as a monotherapy, administered as a subcutaneous injection once every three months. Patients achieved a 2.30-point improvement versus placebo on the Myasthenia Gravis Activities of Daily Living (MG-ADL) score, a validated measure of daily fu...