Douglas Macpherson's questions to X4 Pharmaceuticals Inc (XFOR) leadership • Q4 2024
Question
Douglas Macpherson from H.C. Wainwright asked for commentary on commercialization learnings, whether new patients beyond the trial cohort are on therapy, details on ex-U.S. partnership milestones, and current patient population estimates for WHIM and chronic neutropenia.
Answer
Mark Baldry (Executive) confirmed that all U.S. trial patients are on commercial product and new demand is building through physician engagement. Paula Ragan (Executive) noted the next key ex-U.S. milestone is a potential EU approval in H1 2026 and reiterated patient estimates of ~1,000 diagnosed WHIM patients and a target of 15,000 severe CN patients in the U.S.