Pozelimab

Hemolytic anemias include PNH, aHUS, and wAIHA. Anti-C5 therapy is key for PNH and aHUS. New treatments target upstream complement cascade components. Novel therapies for wAIHA include fostamatinib and sovleplenib.

ASH 2024 highlights include GSK's Blenrep Phase 3 data supporting re-market, J&J's Darzalex Faspro delaying progression in smoldering multiple myeloma, Arcellx's anito-cel showing comparable safety to Carvykti, and J&J/Legend's Carvykti improving survival outcomes. Kura Oncology's ziftomenib showed high response rates in leukemia, Eli Lilly's Jaypirca reduced disease progression risk, and Merck's zilovertamab vedotin achieved high complete response rates in lymphoma. Beam Therapeutics' BEAM-101 showed durable effects in sickle cell disease, Novo Nordisk's etavopivat reduced crises, and Bristol Myers Squibb's arlo-cel demonstrated durable responses in multiple myeloma. Galapagos' GLPG5101 showed encouraging cell therapy results, Orca Bio's Orca-T improved survival in blood cancers, and Sanofi's rilzabrutinib improved platelet response in thrombocytopenia. Regeneron's drug combo showed better disease control than Ultomiris in PNH.

Regeneron achieved significant trial results at the American Society for Hematology meeting, demonstrating better disease control in paroxysmal nocturnal hemoglobinuria with a combination therapy and potential best-in-class performance in follicular lymphoma with a bispecific antibody. Dr. Andres Sirulnik highlights Regeneron's science-driven approach and the company's focus on addressing difficult-to-treat problems in hematology.

Phase 3 trial data showed poze-cemdi, a first-in-class combination treatment, helped more PNH patients achieve and maintain LDH control compared to ravulizumab, with 96% achieving adequate LDH control and 93% achieving normalization.

Regeneron's Phase 3 trial shows pozelimab and cemdisiran (poze-cemdi) combination better controls lactate dehydrogenase (LDH) levels in paroxysmal nocturnal hemoglobinuria patients compared to ravulizumab, with four out of five patients previously on ravulizumab achieving LDH control after switching to poze-cemdi.

Regeneron Pharmaceuticals announced positive Phase 3 data showing pozelimab and cemdisiran (poze-cemdi) combination treatment achieved greater disease control in PNH patients compared to ravulizumab, with 4/5 patients switching from ravulizumab achieving LDH control.

Regeneron's pozelimab and cemdisiran (poze-cemdi) combination therapy for PNH showed superior LDH control vs. ravulizumab in a Phase 3 trial, with 96% of patients achieving adequate LDH control and 93% achieving normalization. Poze-cemdi offers a four-week subcutaneous delivery and is under investigation for other complement-mediated diseases, though not yet approved.

Phase 3 trial data showed poze-cemdi combo treatment improved LDH control in PNH patients compared to ravulizumab, with 96% achieving adequate LDH control and 93% achieving normalization. A registrational cohort is ongoing, comparing poze-cemdi against eculizumab.