X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 allosteric antagonist drugs to improve immune cell trafficking to treat rare diseases and cancer, today announced the presentation of additional Phase 2 clinical data demonstrating a positive safety profile and clinical activity of X4P‑001 in patients with WHIM syndrome, a rare primary immunodeficiency disease. The clinical results are being presented today in a poster session at the 60th American Society of Hematology (ASH) Annual Meeting taking place in San Diego.
“We continue to see encouraging results from this Phase 2 trial, as X4P-001 drug exposure appears to correlate with increases in absolute neutrophil count and absolute lymphocyte count levels. Increases in these biomarkers are an indication of potential improvement in the pathophysiology underlying WHIM syndrome. An example of clinical improvement and impact on symptoms was the continued reduction in a WHIM patient’s human papillomavirus-related warts following X4P‑001 therapy,” said David C. Dale, MD, Professor of Medicine at the University of Washington School of Medicine, Seattle, WA, and lead investigator of the trial.
The poster presentation at ASH describes results from the first 8 patients with genetically confirmed WHIM syndrome and demonstrates the dose-dependent biomarker activity used to support dose selection for the Phase 3 trial. Additional updates on safety and clinical activity across all patients who were enrolled as of the August 17, 2018 data-cut off are also presented in the poster.
Information about the clinical trial of X4P-001 in patients with WHIM syndrome can be found on clinicaltrials.gov: https://clinicaltrials.gov/ct2/show/NCT03005327.
“X4P-001 in WHIM syndrome is X4’s lead product candidate and our team is rapidly advancing toward the Phase 3 trial, with the goal of delivering X4P-001 to the global community of WHIM patients who currently lack any approved treatment specifically for this disease,” said Paula Ragan, PhD, president and chief executive officer of X4 Pharmaceuticals. “We are confident in our path forward based on these favorable Phase 2 results, and we look forward to starting the pivotal Phase 3 trial in the first half of 2019.”