ASH 2024: Regeneron presents pozelimab + cemdisiran head-to-head data in PNH

US biotech Regeneron Pharmaceuticals (Nasdaq: REGN) on Sunday announced positive updated Phase III data of an exploratory cohort from the ACCESS-1 trial investigating its first-in-class pozelimab and cemdisiran (poze-cemdi) combination treatment against ravulizumab, a standard-of-care complement factor 5 (C5) inhibitor, in patients with paroxysmal nocturnal hemoglobinuria (PNH).

Results were shared during an oral session at the American Society of Hematology (ASH) 2024 Annual Meeting and support continued development of poze-cemdi in PNH, including in a separate registrational cohort, as well as in other complement-mediated diseases. Poze-Cemdi is a first-in-class combination of an antibody and an siRNA targeting C5: pozelimab is a fully human monoclonal antibody designed to block the activity of C5, while cemdisiran is an investigational siRNA therapeutic that reduces circulating levels of C5, the company noted.

In August last year, the US Food and Drug Administration (FDA) approved Veopoz (pozelimab-bbfg) injection for the treatment of adult and pediatric patients aged one year and older with CD55-deficient protein-losing enteropathy (PLE), also known as CHAPLE disease. Cemdisiran is being developed with Alnylam (Nasdaq: ALNY).