X4 Pharmaceuticals Inc（XFOR）のニュースとイベントXFORX4 Pharmaceuticals Inc

X4 Pharmaceuticals Reports Q3 Revenue of $2.88M, Beating Consensus

Reports Q3 revenue $2.88M, consensus $2.3M. "With topline data from both our global 4WARD trial and our new 4STEPS trial expected in the first quarter of 2028, we believe mavorixafor is uniquely positioned to transform the treatment landscape for patients with primary chronic neutropenia," said Adam Craig, Executive Chairman of X4 Pharmaceuticals. "We have made significant progress enrolling our 4WARD trial and remain on track to complete our target enrollment of 126 participants by the end of this year. At our recent, well-received Investor Day, we outlined the design of our Phase 2b 4STEPS trial designed to evaluate how to safely reduce G-CSF dosage after starting mavorixafor while maintaining stable neutrophil counts. We also shared new market research supporting the significant commercial opportunity in this disease targeting 15,000 to 30,000 patients in the United States, subject to FDA labeling."

X4 Pharmaceuticals Aligns with FDA on 4WARD Trial Sample Size

X4 Pharmaceuticals announced the outcome of its Type C Meeting with the FDA related to the global 4WARD Phase 3 clinical trial. The purpose of the meeting was to gain alignment on a revised sample size and associated statistical power and the size of the safety database to support a supplemental new drug application for mavorixafor for the treatment of chronic neutropenia in 4WARD. The FDA agreed with the company's analysis that the reduction in sample size for the 4WARD trial from 176 to 126 participants maintains adequate overall power for the co-primary endpoints of the reduction in annualized infection rate and positive absolute neutrophil count response. In addition, the FDA indicated that the proposed safety database would be acceptable to support a potential sNDA.