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X4 Pharmaceuticals Completes Enrollment in Phase 3 Mavorixafor Trial in Patients with WHIM Syndrome
Final enrollment of 31 adult and pediatric patients exceeds initial target of 18-28 patients Top-line data expected in 4Q 2022; U.S. NDA filing anticipated in
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"Final enrollment of 31 adult and pediatric patients exceeds initial target of 18-28 patients Top-line data expected in 4Q 2022; U.S. NDA filing anticipated in 1Q 2023 BOSTON, Oct. 04, 2021 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4-targeted small molecule therapeutics to benefit patients with diseases of the immune system, announced today that it has completed enrollment in the ongoing pivotal Phase 3 clinical trial of its lead candidate, mavorixafor, in the treatment of patients with genetically confirmed WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a primary immunodeficiency caused by gain-of-function mutations in the CXCR4 gene. Thirty-one adult and pediatric patients have enrolled in the 4WHIM trial, which compares a once-daily, oral dose of mavorixafor to placebo across primary and secondary endpoints that include clinically relevant counts of neutrophils and lymphocytes, the frequency and severity of both infections and warts, as well as certain quality of life measurements. The trial was originally designed to enroll 18-28 patients. “This major milestone achievement for X4 is a hopeful step forward for the thousands of WHIM patients with no disease-modifying treatment options,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4. “We would like to especially recognize and thank the patient participants in the 4WHIM trial and their families and caregivers, our study investigators and their clinical teams, as well as our vendors, contractors, and employees who made this first-of-its-kind accomplishment possible.” Dr. Ragan continued: “As we now look ahead to announcing top-line data from the 4WHIM trial, expected in the fourth quarter of 2022, and to a possible regulatory filing in the first quarter of 2023, we are turning our focus towards pre-commercialization efforts, including further identifying WHIM patients and the physicians most likely to care for them. In addition, we look forward to the availability of new long-term data from our ongoing Phase 2 open-label extension trial in WHIM as well as new research and clinical data later this year that we anticipate will support broader market opportunities for mavorixafor across multiple indications.” About Mavorixafor and WHIM SyndromeWHIM (warts, hy...