Etavopivat

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.

Darzalex reduced smoldering multiple myeloma progression risk by 51%; BEAM-101 increased fetal hemoglobin in sickle cell patients; zilovertamab vedotin achieved complete responses in lymphoma; etavopivat halved sickle cell pain crises; anito-cel showed strong response in multiple myeloma, with potential to surpass existing therapies.

Etavopivat reduced annualized vaso-occlusive crises rate vs. placebo in sickle cell disease, increased hemoglobin levels, reduced fatigue, and improved hemolysis markers, according to HIBISCUS trial data presented at ASH Annual Meeting and Exposition.

Novo’s PKR activator, Etavopivat, reduced vaso-occlusive events by nearly 50% in Phase II sickle cell disease patients, with delayed VOC occurrence and improved blood biomarkers.

Sanofi's rilzabrutinib shows promise in treating immune thrombocytopenia, while Novo Nordisk's etavopivat reduces sickle cell pain crises. Beam Therapeutics' CRISPR therapy for sickle cell yields consistent results, though fertility preservation remains a concern.

Five studies at the 66th ASH Annual Meeting present new treatments for sickle cell disease (SCD) and immune thrombocytopenia (ITP), including a first-in-class oral medication for ITP, experimental drugs for SCD reducing pain episodes and improving red blood cell health, hydroxyurea benefits for HbSC variant, and a base-edited gene therapy showing robust results in early trials.

Novo’s PKR activator, Etavopivat, in Phase II reduced vaso-occlusive events by nearly 50% in sickle cell disease patients, with delayed VOC occurrence and improved blood biomarkers.

Etavopivat, in the phase 2 HIBISCUS trial, showed improvements in vaso-occlusive crises, fatigue, hemolysis, and hemoglobin levels in sickle cell disease patients, with a significant reduction in VOCs in per-protocol analysis. The drug's novel mechanism of action as an erythrocyte pyruvate kinase activator suggests potential benefits for SCD treatment.

Novo Nordisk announced 52-week results from the phase 2 HIBISCUS study of etavopivat in sickle cell disease, showing a trend toward reduced vaso-occlusive crises and increased hemoglobin response. Results will be confirmed in the phase 3 HIBISCUS trial.