Rilzabrutinib

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.

Rilzabrutinib, a BTK inhibitor, showed significant efficacy in ITP patients, with 65% achieving platelet response vs. 33% on placebo. The study met primary and secondary endpoints, including durable platelet response, reduced bleeding, and improved quality of life. Rilzabrutinib is under regulatory review in the US and EU.

Rilzabrutinib, a BTK inhibitor, showed significant efficacy in a Phase 3 study for ITP, achieving platelet response in 65% of patients vs. 33% on placebo. It met primary and secondary endpoints, including durable platelet response, reduced bleeding, and improved quality of life. The safety profile was consistent with previous studies, with mild/moderate AEs like diarrhea and nausea. Rilzabrutinib is under regulatory review in the US and EU.

Recent studies presented at the ASH Annual Meeting highlight advancements in treating sickle cell disease (SCD) and immune thrombocytopenia (ITP). Key findings include a new oral medication for ITP showing durable platelet response, etavopivat reducing SCD pain episodes by nearly half, hydroxyurea benefits for HbSC SCD variant, safe fertility preservation for SCD patients, and promising early results from a base-edited gene therapy for SCD.

Rilzabrutinib, a BTK inhibitor, showed significant benefits in a phase 3 study for ITP patients, achieving durable platelet response, reducing bleeding, and improving quality of life. It's under regulatory review in the US and EU, marking a potential first-in-class treatment for ITP.

Rilzabrutinib, a BTK inhibitor, showed significant benefits in a phase 3 study for ITP patients, with 65% achieving platelet response vs. 33% on placebo. It reduced bleeding, need for rescue therapy, and improved quality of life. Currently under US and EU regulatory review, it offers hope for ITP treatment.

Rilzabrutinib showed significant benefits in a phase 3 study for ITP, with 65% achieving platelet response vs. 33% on placebo. It reduced bleeding, need for rescue therapy, and improved quality of life, supporting its potential as a first BTK inhibitor for ITP. Currently under US and EU regulatory review.