Cemdisiran

Regeneron's poze-cemdi combo showed superior LDH control and normalization in PNH patients vs. ravulizumab in the ACCESS-1 trial. Poze-cemdi, combining an antibody and siRNA targeting C5, demonstrated complete complement inhibition, with a safety profile consistent with approved C5 inhibitors. Results support its continued development for PNH and other complement-mediated diseases.

Regeneron's pozelimab and cemdisiran combo shows promise in PNH treatment, outperforming AstraZeneca's Ultomiris in LDH control. With 96% achieving adequate LDH control vs. Ultomiris's 80%, and 93% normalizing LDH levels vs. 65%, the subcutaneous treatment could transform PNH therapy. Regeneron and Alnylam are exploring its use in other diseases.

Pozelimab combined with cemdisiran shows superior control of intravascular hemolysis in PNH patients compared to Ultomiris, with 96% achieving target LDH levels versus 80%. The combination offers potential for self-administration with infrequent dosing, indicating a promising treatment for PNH and other complement-mediated diseases.

Pozelimab combined with cemdisiran shows superior control of intravascular hemolysis in PNH patients compared to Ultomiris, with lower LDH levels and potential for self-administration. Ongoing Phase 3 trials and extensions aim to confirm its efficacy and safety, offering hope for better PNH treatment.

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.

The pozelimab and cemdisiran (poze-cemdi) combination showed superior phase 3 results in the ACCESS-1 trial for PNH, outperforming ravulizumab in disease control and LDH normalization. It offers potential for self-administration with infrequent dosing. Safety profiles were consistent with approved C5 inhibitors, with ongoing studies against eculizumab.

Regeneron Pharmaceuticals reported positive phase 3 trial results for pozelimab plus cemdisiran in treating paroxysmal nocturnal haemoglobinuria (PNH), showing superior efficacy over standard treatments. The combination achieved higher rates of LDH control and normalization, indicating significant progress in PNH treatment and potential for broader application in complement-mediated diseases.

Phase 3 trial data showed the poze-cemdi combination treatment achieved better disease control in PNH patients, measured by LDH levels, compared to ravulizumab. Poze-cemdi, combining an antibody and siRNA targeting C5, demonstrated superior LDH control and normalization, with potential for self-administration, indicating a promising treatment approach for PNH and other complement-mediated diseases.