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X4 Pharmaceuticals Announces Positive Phase 3 Results Showing Mavorixafor Reduced the Rate, Severity, and Duration of Infections vs. Placebo in Participants Diagnosed with WHIM Syndrome
~60% reduction in annualized infection rate seen in the mavorixafor arm vs. placebo (p75% reduction in the percentage of individuals experiencing severe
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"~60% reduction in annualized infection rate seen in the mavorixafor arm vs. placebo (p75% reduction in the percentage of individuals experiencing severe infections (Grade 3 or higher) in the mavorixafor group vs. placebo group >70% reduction in mean total days with infections (2 weeks on mavorixafor treatment vs. 7 weeks on placebo) Company on track to submit NDA for mavorixafor early in 2H 2023 following completion of pre-NDA meeting with FDA X4 webinar today at 4 pm ET to include live Q&A with clinical experts BOSTON, May 16, 2023 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel small molecule therapeutics to benefit patients with rare diseases of the immune system, is hosting a webinar today highlighting new data from its global, pivotal, 4WHIM Phase 3 clinical trial of once-daily, oral mavorixafor in individuals with WHIM syndrome. Following the announcement of positive top-line data from the 4WHIM trial in November 2022, today’s event focuses on the impact of mavorixafor on the rate, severity, and duration of infections in trial participants. “Today, we’re announcing that mavorixafor treatment demonstrated statistically significant and clinically meaningful improvements across a number of key infection metrics versus placebo in our pivotal, Phase 3 trial in those diagnosed with WHIM Syndrome,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “Given that frequent, severe, and long-lasting infections remain the most challenging burden for individuals with WHIM syndrome, we believe that demonstration of these clinical benefits in the 4WHIM trial are an important step in delivering a much-needed breakthrough for patients and their caregivers. We look forward to these data being presented at the upcoming annual meetings of the Clinical Immunology Society (CIS) and the European Hematology Association (EHA).” Dr. Ragan added, “We recently completed our pre-NDA meeting with the FDA and remain on track to submit for U.S. approval of mavorixafor for WHIM syndrome early in the second half of 2023.” Dr. Ragan concluded: “While we remain committed to delivering for the WHIM community, these meaningful results from the 4WHIM trial further bolster our commitment to advancing mavorixafor as quickly as possible as a potential treatment for th...