Sanofi has announced positive results for rilzabrutinib in a phase 3 study that achieved its primary endpoint of durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP), an autoimmune blood disorder.
The randomised, multicentre LUNA 3 study results will be presented at a medical congress later this year.
Affecting approximately four in every 100,000 people every year, ITP is a serious condition characterised by autoantibody-mediated platelet destruction and impaired platelet production, which leads to thrombocytopenia and an increased risk of life-threatening bleeding episodes.
The phase 3 study has been evaluating the efficacy and safety of rilzabrutinib versus placebo in adult and adolescent patients with persistent or chronic ITP who either received oral rilzabrutinib 400mg twice daily or placebo.
Rilzabrutinib is an oral, reversible, covalent BTK inhibitor that, in combination with Sanofi’s Tailored Covalency technology, can selectively inhibit the BTK target, offering the potential to be a first- or best-in-class treatment for several immune-mediated diseases.
The study met its primary endpoint after demonstrating that a significantly higher proportion of patients with primary ITP who had been refractory to prior therapy receiving rilzabrutinib achieved durable platelet response versus placebo, with a median of four prior ITP therapies and a median baseline platelet count of 15,000/μL.
In addition, positive key secondary endpoint results underscored the potential for rilzabrutinib to deliver clinically meaningful benefits for patients living with persistent and chronic ITP.
Houman Ashrafian, executive vice president, head of research and development, Sanofi, commented: “The results of this study reinforce rilzabrutinib’s potential to be a first-in-class oral, reversible BTK inhibitor that can provide clinically meaningful improvements for people living with severe immune-mediated diseases like ITP.”
In February, Sanofi presented positive results from the phase 2 RILECSU study, which showed that rilzabrutinib demonstrated a reduction in itch severity and significantly improved disease activity in adults with moderate-to-severe chronic spontaneous urticaria (CSU), a common and distressing skin condition.
In addition, rilzabrutinib is being evaluated as a treatment for other diseases, including asthma, prurigo nodularis, IgG4-related disease and warm autoimmune haemolytic anaemia.