Full Press Release Details
Positive Phase 1/2 study results of rilzabrutinib in people with immune thrombocytopenia
published in The New England Journal of Medicine
PARIS April 14, 2022 - Positive results from the Phase 1/2 dose-finding study evaluating the safety, pharmacokinetics
and clinical activity of rilzabrutinib, an investigational oral Bruton s tyrosine kinase (BTK) inhibitor, in adults with heavily pre-treated immune thrombocytopenia (ITP) were published in the New
England Journal of Medicine. Results demonstrate treatment with rilzabrutinib led to a rapid and durable increase in platelet count and support an acceptable safety profile. Sanofi is investigating the safety and efficacy of twice daily
rilzabrutinib (400 mg) for adults and adolescents with chronic ITP in the ongoing Phase 3 clinical study LUNA 3, initiated in April 2021.
Director of clinical hematology at Massachusetts General Hospital and
professor of medicine at Harvard Medical School, lead author of the study
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.
Dietmar Berger, M.D., Ph.D.
Global Head of Clinical Development and Chief Medical Officer, Sanofi
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 U.S. Food and
Drug Administration (FDA) for treatment of ITP in November of 2020 and was previously granted orphan drug designation. Rilzabrutinib is being investigated in multiple clinical trials across a range of diseases including immunological and
inflammatory diseases.
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:
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.
Rilzabrutinib is an oral
Bruton s tyrosine kinase inhibitor incorporating Sanofi s TAILORED COVALENCY technology being investigated for the treatment of immune-mediated diseases, including ITP. BTK is an
intracellular signaling molecule involved in innate and adaptive immune responses related to certain immune-mediated diseases. By inhibiting BTK, rilzabrutinib has the potential to target the underlying disease pathogenesis.
Rilzabrutinib is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority.
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