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X4 Pharmaceuticals Announces Positive Results from Completed Six-Month Phase 2 Trial of Mavorixafor in Chronic Neutropenia (CN)
Mavorixafor durably and meaningfully elevated participants’ mean absolute neutrophil counts (ANC) Mavorixafor enabled substantial reductions in G-CSF dosing

About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Mavorixafor durably and meaningfully elevated participants’ mean absolute neutrophil counts (ANC) Mavorixafor enabled substantial reductions in G-CSF dosing while maintaining mean ANC at normal levels Phase 2 study results and new analysis confirming functionality of neutrophils in sub-study participants bolster confidence in achieving success in Phase 3 4WARD trial Company conference call and webcast today at 8:00 am ET BOSTON, Nov. 13, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced positive new clinical data from its now completed Phase 2 clinical trial evaluating mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia (CN). An analysis of final data from the six-month study showed that once-daily oral mavorixafor durably and meaningfully increased participants’ mean absolute neutrophil counts (ANC). In addition, when tested in combination with injectable granulocyte colony-stimulating factor (G-CSF), mavorixafor treatment enabled clinicians to substantially reduce G-CSF dosing while maintaining normal mean ANC levels. “Since the U.S. approval of G-CSF to treat severe chronic neutropenia, there has remained a significant unmet need for an optimal treatment in terms of long-term efficacy, safety, and route of administration,” said Teresa Tarrant, M.D., Associate Professor of Medicine, Rheumatology, and Immunology at Duke University School of Medicine. “I am encouraged by these Phase 2 results showing that mavorixafor not only increased circulating neutrophils across study participants, but also that these neutrophils were functional in a sub-study population. The larger Phase 3 mavorixafor CN trial will expand on these data, and I remain optimistic about this potential much-needed innovation for patients with chronic neutropenia.” “We could not be more pleased with the results from the six-month Phase 2 study of mavorixafor in CN, which are consistent with our earlier findings that mavorixafor durably and meaningfully increased ANC in this population,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “Importantly, we gained insights into the potential real-world use of mavorixafor in CN, should we ultimately obtain approval. These data not o...