Rilzabrutinib

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.

Sanofi's Phase III LUNA 3 trial of rilzabrutinib for persistent or chronic immune thrombocytopenia (ITP) met its primary endpoint, showing a 23% durable platelet response in therapy-treated adults. The study involved a 24-week treatment period with rilzabrutinib 400mg twice daily or placebo, followed by a 28-week open-label period. A 65% platelet response rate was observed in the therapy group versus 33% in the placebo group, with significant improvements in secondary endpoints like decreased bleeding and need for rescue therapy. The therapy is under regulatory review in the EU and US.

Sanofi's phase 3 LUNA 3 study of rilzabrutinib, an oral BTK inhibitor for immune thrombocytopenia (ITP), showed durable platelet response in 23% of patients vs. 0% in placebo. Rilzabrutinib also demonstrated significant improvements in bleeding reduction, rescue therapy use, and quality of life measures.

Sanofi's Phase III LUNA 3 trial of rilzabrutinib for immune thrombocytopenia met primary endpoint, showing 23% durable platelet response. Rilzabrutinib demonstrated a 65% platelet response rate, compared to 33% for placebo, with significant improvements in secondary endpoints and quality of life measures. The therapy is under regulatory review in the EU and US.

Sanofi presents positive phase III results for rilzabrutinib in treating persistent or chronic ITP at ASH annual meeting, showing rapid and durable platelet response, reduced bleeding, and improved quality of life.

Rilzabrutinib, an oral BTK inhibitor, demonstrated safety and efficacy in boosting platelet counts in heavily pretreated ITP patients, achieving a durable platelet response in 23% vs. 0% placebo at week 25. The study met secondary endpoints, showing reduced fatigue, rescue medication use, and bleeding events. Rilzabrutinib may offer a safer treatment option for ITP compared to first-generation BTK inhibitors.

Sanofi-Aventis announced positive LUNA 3 phase 3 study results for rilzabrutinib in adults with persistent or chronic ITP, showing 65% platelet response vs. 33% with placebo. Rilzabrutinib met primary and secondary endpoints, including durable platelet response, reduced bleeding, and fewer rescue therapies. Safety profile consistent with previous studies; under regulatory review in the US and EU.

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.