X4 Pharmaceuticals Highlights Clinical Progress and Market Opportunity for Mavorixafor in Primary Chronic Neutropenia During Virtual Investor Day
Globe Newswire•29/10/2026•16:30 ET
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Key Highlights
- ➤15,000 symptomatic U.S. patients identified as base-case PCN market opportunity
- ➤30,000 U.S. patients represent potential expansion opportunity, subject to FDA labeling
- ➤4WARD Phase 3 target enrollment of 126 participants expected in Q4 2026
- ➤4STEPS Phase 2b trial initiation expected in Q1 2027
- ➤Topline data from both trials expected in Q1 2028
Expert Statements
Adam Craig, Executive Chair of X4 Pharmaceuticals
“PCN is a serious, chronic disease with no new therapy approved in more than 30 years, and today we outlined a clear path to potentially bring the first once-daily oral therapy to people living with this disease.”
Adam Craig, Executive Chair of X4 Pharmaceuticals
“Our 4WARD Phase 3 trial is designed to clearly measure the clinical benefit of mavorixafor, and we remain on track to fully enroll the study by year end.”
Adam Craig, Executive Chair of X4 Pharmaceuticals
“Our planned 4STEPS Phase 2b trial is designed to complement 4WARD by evaluating whether granulocyte colony-stimulating factor (G-CSF) dosage can be reduced or discontinued after initiating mavorixafor.”
Adam Craig, Executive Chair of X4 Pharmaceuticals
“Our most recent commercial research supports a U.S. market opportunity of approximately 15,000 patients, with the potential to grow to approximately 30,000.”
Adam Craig, Executive Chair of X4 Pharmaceuticals
“With data from both trials expected in Q1 2028, we look forward to a milestone-rich next 18 months.”
- Base Case U.S. Market Opportunity Estimated at 15,000 Patients, With Potential to Expand to Approximately 30,000 Patients -
On Track for Completion of 4WARD Phase 3 Target Enrollment in Q4 2026 -
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