X4 reports Phase II data for WHIM syndrome candidate

X4 Pharmceuticals Inc. (Cambridge, Mass.) reported preliminary data from five evaluable patients with warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome in the open-label, dose-escalation Phase II portion of the Phase II/III X4P-001-MKKA trial showing that once-daily oral X4P-001-RD dose-dependently increased neutrophil and lymphocyte counts from baseline. X4P-001-RD was well tolerated with no severe adverse events reported. Data were presented at the American Society of Hematology meeting in Atlanta in December.

The placebo-controlled, U.S. and Australian trial is evaluating safety and absolute neutrophil and lymphocyte counts as its primary endpoints. Secondary endpoints include frequency and severity of infections, area of wart-involved skin, severity of genital warts, antibody levels following re-vaccination and frequency of events requiring rescue therapy. Dose escalation is continuing in the Phase II portion of the trial...