Business
X4 Pharmaceuticals Announces Positive Interim Clinical Data from Ongoing Six-Month Phase 2 Trial of Mavorixafor in Chronic Neutropenia (CN) and Initiation of Pivotal Phase 3 CN Trial
100% of evaluable participants at Month 6 achieved target absolute neutrophil count (ANC) increase with once-daily, oral mavorixafor +/- stable-dose G-CSF as
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"100% of evaluable participants at Month 6 achieved target absolute neutrophil count (ANC) increase with once-daily, oral mavorixafor +/- stable-dose G-CSF as of the interim analysis data cut-off date Durable mean ANC levels above the lower limit of normal for CN were achieved for participants on mavorixafor monotherapy at Months 3 and 6 Company webinar today at 8:00 am ET will detail the interim clinical results and feature clinical experts in the treatment of CN BOSTON, June 27, 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 ongoing Phase 2 clinical trial evaluating the safety and efficacy of mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia (CN). An interim analysis of data from the ongoing six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (ANC) both as a monotherapy and in combination with stable doses of injectable granulocyte colony-stimulating factor (G-CSF), the only therapy approved in the U.S. for severe chronic neutropenia. Today the company also announced that it is currently screening patients for enrollment into its global, pivotal Phase 3 clinical trial, the 4WARD study, evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants. “For the first time, we have demonstrated the ability of mavorixafor monotherapy to durably and meaningfully increase ANC in people living with chronic neutropenia,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “In addition, we were pleased to see that once-daily mavorixafor used in combination with G-CSF also led to meaningful and sustained ANC increases and was generally well tolerated, further supporting the design of our newly initiated Phase 3 4WARD clinical trial, which will study the use of mavorixafor alone and with G-CSF in people with CN deali...