X4 Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Corporate Update

X4 Pharmaceuticals

Tue, March 17, 2026 at 7:15 AM EDT 7 min read

- Global 4WARD Phase 3 Chronic Neutropenia Trial On Track to Complete Enrollment in Q3 2026 -

- Positive Opinion from European Medicines Agency Recommending Marketing Authorization of Mavorixafor for the Treatment of WHIM Syndrome in the European Union -

- Balance Sheet Provides Cash Runway through 2028 -

BOSTON, March 17, 2026 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company focused on improving the lives of people with rare hematology diseases, today reported financial results for the fourth quarter and year ended December 31, 2025 and provided a corporate update.

“Our focus remains on our pivotal 4WARD trial in chronic neutropenia as we execute our strategy to deliver life-changing therapies to people living with rare blood diseases,” said Adam Craig, M.D., Ph.D., Executive Chairman of X4 Pharmaceuticals. “We have sharpened our operational focus and implemented targeted initiatives to drive continued progress in 4WARD with enrollment on track for completion in the third quarter of this year. Additionally, we were pleased to receive the positive opinion from the European Medicines Agency recommending the potential European approval of mavorixafor in WHIM syndrome, which would be the first and only therapy in Europe to treat this devastating condition.”

Recent Accomplishments and Updates

• Deployed a series of proactive measures to increase enrollment in the global 4WARD Phase 3 trial of oral, once-daily mavorixafor (with or without G-CSF) in patients with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia, including:

  • Expanding the number of current active clinical trial sites to over 100, including approximately 20 in the U.S.;

  • Enhancing our global Medical Affairs activities to increase Medical Science Liaison (MSL) field engagement and site interaction to educate physicians on our 4WARD trial and mavorixafor’s potential in treating chronic neutropenia;

  • Establishing a patient referral pathway for physicians to assist them in finding trial sites for their chronic neutropenia patients; and

  • Investing in database mining to identify potential participants.

• European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization of mavorixafor the treatment of WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome in the European Union (EU). The positive opinion will now be reviewed by the European Commission (EC) with a final approval decision anticipated in the second quarter of 2026.

• Cash runway through 2028 due to recent equity financings and operating expense reductions.