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Second-generation oral Bruton tyrosine kinase (BTK) inhibitor tirabrutinib demonstrated sustained efficacy in treatment-na&iuml;ve (TN) and relapsed/refractory (R/R) Waldenstr&ouml;m&rsquo;s macroglobulinemia (WM), according to two-year follow-up data from a phase II study presented at the 2021 American Society of Hematology Annual Meeting.Tirabrutinib, designed to improve the safety and efficacy of ibrutinib with a more favorable toxicity profile, was approved in Japan in 2020 for TN or R/R WM. Results from an open-label, prospective, single-arm phase II study conducted at 19 facilities in Japan, previously reported with data cutoff on August 28, 2019, provided the basis for this approval.The present report details results from a phase II study that enrolled 18 patients with TN WM and nine with R/R WM. Major response rate (MRR) as assessed by an independent review committee (IRC). Secondary endpoints were overall response rate (ORR), time to major response (TTMR), duration of response (DOR), progression-free survival (PFS), overall survival (OS), and safety.Participants with serum IgM &ge;500 mg/dL received tirabrutinib 480 mg under fasting conditions once daily until disease progression or unacceptable toxicity.Median follow-up was 23.8 months in the TN cohort and 25.4 months in the R/R arm. A total of five patients discontinued treatment because of adverse events (AEs), including atypical mycobacterial infection (n = 1), progressive disease (n = 1), physician&rsquo;s discretion (n = 1), and withdrawal by subject (n = 2). MRR assessed by IRC was 94.4% in the TN group and 88.9% in the R/R group.IRC-assessed ORR was 94.4% in the TN arm and 100% in the R/R cohort. In both cohorts, the rate of very good partial response or greater was 33.3%. In the TN group, median TTMR was 1.9 months (range = 1-20.3) compared with 2.1 months (range = 1-3.7) in the R/R arm. Across both treatment groups, median PFS, OS, and DOR were not reached. The two-year PFS rates were 94.4% and 88.9% in the TN and R/R cohorts, respectively. In patients who remained on treatment, continued reductions in IgM were observed.Across both treatment groups, the most common AEs were rash (44.4%), neutropenia (33.3%), and nasopharyngitis (25.9%). The most common grade &ge;3 AEs were neutropenia (22.2%), lymphopenia (18.5%), and leukopenia (11.1%). One new grade &ge;3 treatment-related AE of hypertriglyceridemia was observed during the follow-up period.

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Extended Follow-Up Data on Tirabrutinib in Treatment-Naïve and Relapsed/Refractory Waldenström Macroglobulinemia

ASH Annual Meeting and Exposition 2021

Second-generation oral Bruton tyrosine kinase (BTK) inhibitor tirabrutinib demonstrated sustained efficacy in treatment-naïve (TN) and relapsed/refractory (R/R) ...

Extended Follow-Up Data on Tirabrutinib in Treatment-Naïve and Relapsed/Refractory Waldenström

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