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Regeneron Advances In Rare Disease Race With Positive Autoimmune Muscle Disorder Trial Data

Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) on Tuesday shared data from the Phase 3 NIMBLE trial assessing investigational cemdisiran monotherapy in adults with generalized myasthenia gravis (gMG).

gMG is a rare, chronic autoimmune disease that causes weakness in voluntary muscles, affecting the eyes, face, neck, arms, legs, and breathing.

The trial met the primary and key secondary endpoints.

Cemdisiran is an siRNA that reduces circulating levels of complement factor 5 (C5) and, as monotherapy in this trial, was associated with an average of 74% inhibition of complement activity.

Cemdisiran’s Breakthrough: A Game Changer For GMG

The trial also assessed a combination of cemdisiran and pozelimab, a C5 antibody. The combination (cemdi-poze), which resulted in nearly 99% inhibition of complement activity, also met the primary and key secondary endpoints, though cemdisiran monotherapy was numerically better across these endpoints.

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Patients were randomized to receive subcutaneous administrations of: cemdisiran (600 mg) ...