- Results published today in the
- Data support an acceptable safety profile
- Rilzabrutinib is an investigational oral Bruton tyrosine kinase inhibitor (BTKi) for the treatment of ITP, a rare acquired autoimmune disorder in which platelets are destroyed or damaged and for which there are limited treatment options
Director of clinical hematology at
"Currently, there are no standard treatment recommendations for ITP patients with multiple relapses. Despite advances in treatment options over the years, some patients remain refractory to existing therapies and durable remission remains elusive. The Bruton's tyrosine kinase is a critical signaling molecule in the immune system that is involved in certain immune-mediated diseases, and our research suggests that targeting BTK may represent a promising approach to addressing the underlying cause of ITP."
ITP is an acquired autoimmune blood disorder characterized by low platelet count (thrombocytopenia) resulting from immune-mediated platelet destruction and impairment of platelet production. A decrease in platelet counts - whether temporary or persistent - can predispose a person to a higher risk of bleeding, hospitalization, fatigue, impaired quality of life, and even death. The incidence of ITP increases with age and is more common over the age of 60.
Global Head of Clinical Development and Chief Medical Officer,
"We are pleased to share these encouraging early clinical results through this publication. These findings demonstrate a clinically meaningful response in difficult-to-treat ITP patients who received a median of four prior ITP therapies. Moreover, the overall study population, which also included less refractory patients, showed a numerically higher response. Rilzabrutinib could become a first-in-class BTK inhibitor therapy with the potential to increase platelet counts quickly and durably for people with ITP."
Rilzabrutinib was granted Fast Track Designation by the
Phase 1/2 Study Results
The global Phase 1/2 adaptive, open-label, dose-finding study evaluated rilzabrutinib in 60 people with ITP with a median age of 50 years (range, 19-74). Patients had received a median of four different ITP therapies previously. Initial doses could be 200 mg once daily, 400 mg once daily, 300 mg twice daily (600 mg/day), or 400 mg twice daily (800 mg/day). The median platelet counts at the start of the study were 15×109/L, indicating a very low platelet count and high risk of bleeding. The primary endpoint measured the number of participants who achieved at least two consecutive platelet counts of >=50×109/L and an overall platelet count increase of >=20×109/L from the start of treatment without requiring rescue medication.
Study results showed:
- Overall, 24 of 60 people enrolled in the study at any dose achieved the primary endpoint. Of the 45 people who initiated rilzabrutinib at 400 mg twice daily, 18 met the primary endpoint.
- Median time to first platelet count of at least 50×109/L was rapid at 11.5 days, which was maintained in patients with primary platelet response for a mean of 65% of weeks during the 24-week treatment period.
- 52% of participants experienced at least one treatment related adverse event, all of which were grade 1 or 2; the most common adverse events were diarrhea (32%), nausea (30%), and fatigue (10%).
- There were no grade 3 or higher treatment-related adverse events or serious adverse events.
Rilzabrutinib Clinical Program
The safety and efficacy of rilzabrutinib in ITP are being evaluated in the ongoing randomized, double-blind, Phase 3 LUNA 3 study in adults and adolescents (aged >=12 years) with persistent/chronic ITP. In addition, phase 2 studies are ongoing to evaluate rilzabrutinib as a potential therapy for the autoimmune condition IgG4 disease and immunological diseases, including asthma, atopic dermatitis, chronic spontaneous urticaria and warm autoimmune hemolytic anemia.
About Rilzabrutinib
Rilzabrutinib is an oral Bruton's tyrosine kinase inhibitor incorporating
Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
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Arnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.com
Corentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com
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