Clinical Trials

The results demonstrate that the drug met its primary endpoint of durable platelet response in this indication, and its safety profile remained consistent with that reported in previous studies. Theclinicallyandstatisticallysignificant results were achieved in ‘a population of patients with primary ITP that had been refractory to prior therapy’, according to the company’s press release.

The trial also saw positive results on key secondary endpoints, which highlights the drug’s potential to provide meaningful benefits to patients with persistent and chronic ITP.

According to the company’s press release, ‘ITP is a serious, acquired autoimmune blood disorder characterised 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’.

Houman Ashrafian, executive vice president and head of research and development at 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. These pivotal results are a testament to our commitment and expertise in rare blood diseases and ability to build a portfolio of next-generation small-molecule inhibitors that are both more selective and optimised to deliver robust efficacy and safety outcomes as compared to existing therapies.”