Aug 26, 04:23 PM

Regeneron’s Cemdisiran and Pozelimab Combo Shows Positive Phase 3 Results in Myasthenia Gravis With Near-Complete C5 Suppression

Regeneron Pharmaceuticals announced positive results from its Phase 3 clinical trial evaluating a combination therapy for generalized myasthenia gravis (gMG). The therapy combines cemdisiran, a small interfering RNA (siRNA) targeting complement component 5 (C5), with pozelimab, a monoclonal antibody that suppresses circulating C5. The trial met its primary endpoint by significantly improving patients' activities of daily living, including functions such as talking and eating, and demonstrated near-complete C5 suppression with a favorable safety profile. These results position Regeneron to file for FDA approval in 2026. The success marks a notable advancement in the treatment of this rare autoimmune neuromuscular disorder amid a competitive landscape. Additionally, Regeneron’s cemdisiran therapy, used alone or in combination with its complement inhibitor Veopoz, further showed improvements in daily living activities for gMG patients. The company continues to express enthusiasm for its Factor XI candidates, which are viewed as promising but currently underappreciated. Alnylam Pharmaceuticals, a collaborator in this RNA-based therapeutic approach, is also preparing for upcoming investor presentations.

#Regeneron Pharmaceuticals #Regeneron #FDA #Veopoz #Factor XI #Alnylam Pharmaceuticals

Written with ChatGPT (GPT-4).

Regeneron’s Cemdisiran and Pozelimab Combo Shows Positive Phase 3 Results in Myasthenia Gravis With Near-Complete C5 Suppression

Sources

Additional media