X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today reported financial results for the second quarter ended June 30, 2020. The company also provided an update on its lead investigational candidate, mavorixafor, a novel small molecule in a Phase 3 clinical trial for patients with WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome and in two Phase 1b trials in patients with Waldenström’s macroglobulinemia (WM) and Severe Congenital Neutropenia (SCN).

“Despite the challenges posed by the ongoing COVID-19 pandemic, we achieved significant progress during the second quarter, benefiting from two key events related to the WHIM syndrome indication for mavorixafor, while continuing to advance our program in the Waldenström’s indication,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “At our Analyst Day in early April, we presented market research data that supported significantly increasing our disease prevalence estimate for WHIM in the U.S., and during our presentation at the European Hematology Association (EHA) Annual Congress in June, we highlighted Phase 2 mavorixafor data that strengthen our confidence in mavorixafor’s potential to be a disease-modifying therapy in patients with WHIM syndrome and in the Phase 3 trial design.”

Dr. Ragan continued, “While our public presentations during the first half of 2020 focused primarily on WHIM syndrome, we expect that the second half of 2020 will see us increase exposure to our program in patients with Waldenström’s macroglobulinemia, a rare form of lymphoma, where we look forward to announcing initial Phase 1b clinical results towards the end of the year.” The ongoing Phase 1b clinical trial is expected to enroll between 12 and 18 patients with WM and is a multi-center, open-label, dose-escalation clinical trial assessing the safety and tolerability of mavorixafor in combination with ibrutinib. The trial is being conducted as part of a collaboration with The Leukemia & Lymphoma Society to accelerate the development of mavorixafor for the treatment of WM.

“As we await these important results,” Dr. Ragan concluded, “we continue to expect top-line Phase 3 results of mavorixafor in WHIM syndrome in 2022 and initial data from our Phase 1b trial of mavorixafor in patients with SCN in 2021. In light of the continued uncertainties surrounding COVID-19, we intend to provide further clarity around these timelines as soon as is practicable.”

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