X4 Pharmaceuticals to Present P-III Trial (4WHIM) Results of Mavorixafor for the Treatment of WHIM Syndrome at CIS 2023
Shots:
- The P-III trial (4WHIM) evaluating mavorixafor (400mg, qd) vs PBO & enrolled 31 patients aged ≥12yrs. The results showed a ~60% reduction in annualized inf. rate, patients experienced ≤1 inf./yr. vs 4.5 on PBO, grade ≥3 inf. (7% vs 29%)
- 75% reduction who experienced sev. inf., single grade 3 inf. during the first 3mos. while no serious inf. after 3mos. & the frequency of sev. inf. in those on PBO remained unchanged over the 52wk. trial period
- ≥70% reduction in total days with inf. (2 vs 7wks.), 40% lower total inf. score, reductions in upper & lower respiratory tract and skin inf., 90% continued to receive mavorixafor in an ongoing OLE study. The company plans to submit the NDA for mavorixafor early in H2’23, upon completion of the pre-NDA meeting with the US FDA
Ref: X4 Pharmaceuticals | Image: X4 Pharmaceuticals
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