X4 Pharma’s mavorixafor action positive in rare immune deficiency disorder

X4 Pharmaceuticals (XFOR +3.5%) announces results from an open-label Phase 2 extension study evaluating lead candidate mavorixafor in patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, a rare inherited immune deficiency disorder caused by mutations in a gene that encodes a protein called CXCR4. The data were virtually presented at EHA.

Sustained dose-dependent increases in white blood cells, neutrophil count and lymphocyte count were achieved which correlated with fewer infections and reduced numbers of warts. Specifically, yearly infection events dropped from 4.63 to 2.27 while the number of warts dropped by an average of 75%.

On the safety front, mavorixafor, a small molecule inhibitor of CXCR4, was well-tolerated over periods of two years or more without serious treatment-related adverse events.

A Phase 3 study is in process. Topline data should be available in 2022.

https://seekingalpha.com/news/3582669-x4-pharmas-mavorixafor-shows-positive-action-in-rare-immune-deficiency-disorder