FDA Backs Smaller Phase 3 Trial for X4's Chronic Neutropenia Drug
XFOR sits 48% above its 52-week low of $2.8 on light trading volume (0.2× avg).
Summary
X4 Pharmaceuticals announced a positive FDA Type C meeting outcome for its 4WARD Phase 3 trial of mavorixafor in chronic neutropenia. The FDA agreed to reduce the trial's sample size from 176 to 126 participants while maintaining adequate statistical power for the co-primary endpoints. The agency also indicated the proposed safety database would be acceptable to support a potential sNDA. This de-risks the pivotal program and accelerates the timeline: enrollment is now expected to complete by year-end 2026, with topline data in H1 2028. The company's cash runway extends into 2029, so the trial is fully funded. The reduced sample size lowers costs and shortens the path to a potential second indication for mavorixafor, which is already approved as XOLREMDI in the U.S. for WHIM syndrome.
At the time of this announcement, XFOR was trading at $4.14 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $400.6M. The 52-week trading range was $2.80 to $4.83. This news item was assessed with positive market sentiment and an importance score of 8 out of 10. Source: GlobeNewswire.