Press Release

Rilzabrutinib LUNA 3 phase 3 study met primary endpoint in immune thrombocytopenia - Seite 2

ITP is a serious, acquired autoimmune blood disorder characterized by autoantibody-mediated platelet destruction and impaired platelet production, leading to thrombocytopenia (low platelet counts of less than 100,000/μL) and an increased risk of life-threatening bleeding episodes (like intracranial hemorrhage). In addition, patients with ITP often experience significant quality-of-life impairments such as fatigue and cognitive dysfunction. With its dual mechanisms of action that reduce production of pathogenic autoantibodies and decrease macrophage mediated platelet destruction, rilzabrutinib could address the underlying mechanisms responsible for a wide range of ITP complications. 

About LUNA 3
LUNA 3 (NCT04562766) is a randomized, multicenter, phase 3 study evaluating the efficacy and safety of rilzabrutinib vs placebo in adult and adolescent patients with persistent or chronic ITP. Patients received either oral rilzabrutinib 400 mg twice a day or placebo through a 12- to 24-week double-blind treatment period, followed by a 28-week open-label treatment, and then a 4-week safety follow-up or long-term extension period. The adolescent part of the study is ongoing and still recruiting.

The primary endpoint is durable platelet response defined as the proportion of participants able to achieve platelet counts at or above 50,000/μL for for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. Secondary endpoints include the number of weeks with and time to platelet responses, rescue therapy use, and physical fatigue and bleeding score.

Detailed results of the LUNA 3 phase 3 study will be presented at a medical congress later this year.

Rilzabrutinib is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

About Rilzabrutinib
Rilzabrutinib is an oral, reversible, covalent BTK inhibitor that has the potential to be a first- or best-in-class treatment of several immune-mediated diseases. BTK, expressed in B cells, mast cells and other cells from the innate immune system, plays a critical role in inflammatory pathways and multiple immune-mediated disease processes. With the application of Sanofi’s TAILORED COVALENCY technology, rilzabrutinib can selectively inhibit the BTK target.