▎WuXiEdited by Kant Content Team

Today, Sanofi announced positive results from the Phase 3 LUNA clinical trial of its potential "first-in-class" oral BTK inhibitor rilzabrutinib in the abstracts presented at this year's American Society of Hematology (ASH) Annual Meeting. The data showed,23% of adult patients with immune thrombocytopenia (ITP) reached the primary endpoint, while none of the patients in the placebo group met the criteria. Sanofi is preparing to submit a regulatory application to the FDA for the use of this therapy in ITP treatment.

In the LUNA 3 trial, a total of 133 adults were randomly assigned to receive rilzabrutinib treatment.Analysis showed that 86 patients (65%) in the rilzabrutinib group achieved platelet response, compared with only 23 patients (33%) in the placebo group.The primary endpoint was durable platelet response, defined as achieving a platelet count of 50,000/μL or more for at least 8 weeks during the last 12 weeks of the 24-week double-blind treatment period without the need for rescue therapy.The analysis shows,Thirty-one patients (23%) in the rilzabrutinib group reached this endpoint, compared with none of the 69 patients in the placebo group (P<0.0001).

Treatment with Rilzabrutinib also improved several secondary endpoints, including a 53% reduction in the need for urgent rescue therapy compared to placebo, as well as improvements in physical fatigue.In terms of safety, the incidence of adverse events was similar between the rilzabrutinib and placebo groups; however, one patient with multiple risk factors experienced a treatment-related grade 3 peripheral embolism, or arterial blockage.

Rilzabrutinib is an oral, reversible covalent BTK inhibitor that has the potential to become a "first-in-class" and "best-in-class" therapy for various immune-mediated diseases.BTK is expressed in B cells, mast cells, and other innate immune system cells, playing a crucial role in inflammatory pathways and the pathological processes of various immune-mediated diseases. This drug was acquired by Sanofi through its $3.7 billion acquisition of Principia Biopharma in 2020.

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References:

[1] 5 Efficacy and Safety of Oral Bruton Tyrosine Kinase Inhibitor (BTKi) Rilzabrutinib in Adults with Previously Treated Immune Thrombocytopenia (ITP): A Phase 3, Placebo-Controlled, Parallel-Group, Multicenter Study (LUNA 3). Retrieved November 5, 2024 from https://ash.confex.com/ash/2024/webprogram/Paper199718.html

[2] Sanofi reveals positive data for BTK inhibitor from $3.7B Principia takeover. Retrieved November 5, 2024 from https://endpts.com/ash24-sanofi-reveals-positive-phase-3-data-for-btk-inhibitor-from-3-7b-principia-takeover/

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