Sanofi has announced positive results from their pivotal LUNA 3 study, a phase 3 clinical trial of rilzabrutinib, the company’s investigational oral therapy developed to treat persistent or chronic primary immune thrombocytopenia (ITP).
Primary ITP is a rare autoimmune disorder that causes a significant reduction in platelets, which are necessary for clotting. Symptoms of ITP include the appearance of petechiae (clusters of red spots where capillaries below the skin have bled), excessive bruising, nose bleeds and prolonged bleeding of the gums after dental procedures. In addition, women living with ITP may experience heavy menstrual bleeding. The disorder can lead to increased fatigue and impact overall quality of life. In the most severe cases, ITP can even become life-threatening.
According to a new Sanofi press release, LUNA 3, phase 3 data has shown that the desired platelet response was achieved in 65% of patient participants receiving rilzabrutinib compared to 33% of those on placebo. Sanofi reported that the primary study endpoint was met, with rilzabrutinib demonstrating durable platelet response in 23% of ITP adult patients compared to 0% on the placebo group. The secondary clinical study endpoints were additionally met, including reduced bleeding, number of weeks with platelet response, the need for rescue therapy use, and improved physical fatigue and quality of life measures.
“People living with immune thrombocytopenia who cannot tolerate or do not respond to medications aimed at raising platelet counts are at risk of uncontrolled bleeding and often endure side effects from steroids and other available therapies. A significant percentage of these patients also suffer from severe fatigue and an impaired quality of life,” said study author David Kuter, MD. Dr. Kuter, director of clinical hematology at Massachusetts General Hospital and a Professor of Medicine at Harvard University went on to say that “I’m encouraged by the robust therapeutic effects I’ve seen in patients of the LUNA 3 study across all aspects of the disease, including clinically meaningful and sustained improvements in platelet count, quality of life metrics, reduction in bleeding, and a favorable safety profile.”
These results were presented during the 66th American Society of Hematology meeting, which was held December 7-10, 2024, in San Diego, California.
The U.S. Food and Drug Administration granted rilzabrutinib fast track designation back in the fall of 2020. The drug is currently under regulatory review in both the U.S. and the E.U.
Read the complete press release for more information about rilzabrutinib and the LUNA 3 study.
Writer: Jonathan Angarola, Senior Manager of Medical Programs & Information
