X4 Pharmaceuticals (NASDAQ:XFOR) reported positive results from its ongoing Phase 2 trial of mavorixafor for WHIM syndrome.
WHIM stands for warts; hypogammaglobulinemia, or low antibody levels; infections; and myelokathexis, or low white blood cell levels. The syndrome is an inherited immunodeficiency disease caused by mutations in the CXCR4 receptor gene. Mavorixafor is an orally administered CXCR4 receptor antagonist.
The trial demonstrated sustained, dose-dependent increases in white blood cells and absolute neutrophil and lymphocyte counts. Higher doses of mavorixafor increased absolute neutrophil counts by at least 4.5-fold, compared with lower doses.
The trial also met two secondary clinical endpoints of fewer infections and reduced numbers of cutaneous warts.
“We are extremely encouraged by the positive therapeutic profile of mavorixafor emerging from this study, including improvements in key biomarkers and clinical symptoms at the higher doses,” president and CEO, Paula Ragan, said in a statement.
“We view these data as a significant de-risking event for our ongoing and pivotal Phase 3 clinical trial, from which we expect top-line data in 2022,” she added.
