Rilzabrutinib Achieves Primary Endpoint in LUNA 3 ITP Trial

Rilzabrutinib, an oral BTK inhibitor, has shown promising results in the LUNA 3 phase 3 clinical trial for the treatment of immune thrombocytopenia (ITP). The study revealed that the medication, administered at a dosage of 400 mg twice daily, successfully achieved its primary endpoint of a durable platelet response in adult patients with persistent or chronic ITP. This outcome was notably significant in a patient group that had been unresponsive to previous treatments, with participants having a median of four prior ITP therapies and a median baseline platelet count of 15,000/μL.

The LUNA 3 trial, which is still enrolling adolescent participants, is a randomized, multicenter study that compares rilzabrutinib against a placebo. The treatment period spans from 12 to 24 weeks under double-blind conditions, followed by a 28-week open-label phase and a 4-week safety follow-up or long-term extension. The primary endpoint focuses on the proportion of participants maintaining platelet counts of at least 50,000/μL for at least 8 out of the last 12 weeks of the blinded treatment phase without the need for rescue therapy.

Rilzabrutinib has been recognized by the US FDA with Fast Track Designation for ITP treatment, granted in November 2020, and has also received Orphan Drug Designation. The drug is part of Sanofi's immunology pipeline, which includes 12 potential medicines and vaccines, highlighting the company's commitment to developing next-generation treatments for immune disorders.

Houman Ashrafian, Executive Vice President and Head of Research and Development at Sanofi, emphasized the significance of the study's findings, stating that rilzabrutinib could be a groundbreaking oral, reversible BTK inhibitor offering substantial improvements for individuals with severe immune-mediated conditions like ITP. The results reflect Sanofi's dedication to rare blood diseases and the development of selective and effective small-molecule inhibitors.

ITP is a severe autoimmune disorder characterized by the destruction of platelets and impaired production, resulting in thrombocytopenia and an increased risk of severe bleeding events. Rilzabrutinib's dual action of reducing pathogenic autoantibodies and decreasing macrophage-mediated platelet destruction could address the root causes of ITP complications.

Rilzabrutinib's potential as a first- or best-in-class treatment for various immune-mediated diseases is currently under investigation. The drug utilizes Sanofi's TAILORED COVALENCY® technology to selectively inhibit the BTK target, which plays a crucial role in inflammatory pathways and disease processes. In addition to ITP, rilzabrutinib is being studied for its efficacy in treating asthma, chronic spontaneous urticaria, prurigo nodularis, IgG4-related disease, and warm autoimmune hemolytic anemia.

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