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X4 Pharmaceuticals Announces Positive Preliminary Data from Ongoing Phase 1b Clinical Trial of Mavorixafor in Waldenström’s Macroglobulinemia as Published in EHA Abstract
Initial data from first cohort of patients indicate mavorixafor plus ibrutinib to be well tolerated, with encouraging decreases in serum IgM observed These
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Initial data from first cohort of patients indicate mavorixafor plus ibrutinib to be well tolerated, with encouraging decreases in serum IgM observed These and additional preliminary Phase 1b data to be presented in an e-Poster on June 11 at the 2021 European Hematology Association (EHA) Annual Congress BOSTON, Mass., May 12, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today disclosed the first efficacy and safety data from a small cohort of patients in its ongoing Phase 1b clinical trial of its lead candidate mavorixafor in combination with ibrutinib in patients with Waldenström’s macroglobulinemia. These data are included in an abstract published today and selected to be presented as an e-Poster at this year’s European Hematology Association (EHA) Annual Congress, taking place virtually from June 9-17, 2021. About the Phase 1b Clinical Trial The Phase 1b, open-label, multicenter, single-arm study (NCT04274738) is being conducted in the U.S., Germany, and Greece. The trial examines intra-patient dose escalation, safety, pharmacokinetics, and pharmacodynamics of mavorixafor in combination with ibrutinib in patients greater than 18 years of age with a diagnosis of Waldenström’s macroglobulinemia and confirmed MYD88L265P and CXCR4WHIM genetic mutations.In the Phase 1 study, patients are initiated on oral doses of mavorixafor 200 mg and ibrutinib 420 mg once-daily. Mavorixafor escalation to 400 mg occurs after 28 days if no dose-limiting toxicities are observed, and to 600 mg after 400 mg is deemed safe. Patients are followed for adverse events and change from baseline in serum IgM, pharmacokinetics, and pharmacodynamic markers, including peripheral white blood cell counts (WBCs). About Waldenström’s Macroglobulinemia and Mavorixafor Waldenström’s macroglobulinemia (WM) is a rare B-cell lymphoproliferative disorder characterized by increased Immunoglobulin M (IgM)-secreting cells and increased risk of developing symptomatic hyperviscosity syndrome. Patients with the double MYD88L265P and CXCR4WHIM mutation treated with ibrutinib (a Bruton's tyrosine kinase (BTK) inhibitor) typically experience delayed response, inferior depth of response, and/or shorter progression-free survival.Mavo...