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X4 Pharmaceuticals to Present P-III Trial (4WHIM) Results of Mavorixafor for the Treatment of WHIM Syndrome at CIS 2023

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X4 Pharmaceuticals

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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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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