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X4 Pharmaceuticals Initiates Phase 1b Clinical Trial of Mavorixafor in Combination with Ibrutinib for the Treatment of Waldenström’s Macroglobulinemia (WM)
– Trial to assess safety and tolerability of mavorixafor in combination with ibrutinib in WM patients with MYD88/CXCR4 double mutation associated with
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Trial to assess safety and tolerability of mavorixafor in combination with ibrutinib in WM patients with MYD88/CXCR4 double mutation associated with treatment resistance –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nX4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, today announced the initiation of a Phase 1b clinical trial of mavorixafor (X4P-001) in combination with ibrutinib (Imbruvica®) for the treatment of Waldenström’s macroglobulinemia (WM), a rare form of non-Hodgkin’s lymphoma. Mavorixafor, X4’s lead therapeutic candidate, is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4.\n\n\n“There is a significant unmet medical need for patients living with WM who have CXCR4 mutations. The development of a therapeutic CXCR4 antagonist such as mavorixafor represents a very important advance for targeted therapy of this disease,” said Steven Treon, M.D., Ph.D., FACP, FRCP, Director of the Bing Center for Waldenström's Macroglobulinemia and Professor of Medicine at Harvard Medical School.\n\n\n“The CXCR4 mutation, which is present in approximately 30 to 40 percent of patients with WM, is known to play an important role in treatment resistance, and is associated with higher rates of disease burden, making the CXCR4 pathway a critical therapeutic target for patients with WM,” said Christian Buske, M.D., Director of the Institute of Experimental Cancer Research and Attending Physician and Professor of Medicine at the University of Ulm.\n\n\nThe Phase 1b multi-center, open-label, dose-escalation, clinical trial is designed to assess the safety and tolerability of mavorixafor in combination with ibrutinib in patients with WM who have acquired a “gain of function” mutation in CXCR4 in addition to the MYD88 mutation, which is a hallmark of WM diagnosis.1 In addition, the trial is designed to measure changes in serum immunoglobulin M (IgM) and hemoglobin (Hgb) from baseline, both biomarkers are key elements of clinical response in WM patients.2,3 The clinical trial is expected to enroll approximately 12-18 patients.\n\n\n“Having established proof of concept for mavorixafor in WHIM patients, we believe that there is a compelling case to evaluate mavorixafor’s same mechanism ...