Business
X4 Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full Year 2019 Financial Results
– Pivotal Phase 3 clinical trial of lead candidate mavorixafor in WHIM syndrome ongoing with top-line results expected in the second half of 2021 – – Initial
About this update from X4 Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Pivotal Phase 3 clinical trial of lead candidate mavorixafor in WHIM syndrome ongoing with top-line results expected in the second half of 2021 –\n\n\n– Initial Phase 1b results in ongoing Waldenström’s macroglobulinemia (WM) and Severe Congenital Neutropenia (SCN) trials expected in the second half of 2020 – \n\n\n– Conference call today at 8:30 a.m. ET – \n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nX4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today provided a corporate update and reported financial results for the fourth quarter and full year ended December 31, 2019.\n\n\n“2019 was a remarkable year for X4, with significant achievements across the entire organization,” said Paula Ragan, Ph.D., President and Chief Executive Officer of the Company. “We made important progress advancing our lead therapeutic candidate mavorixafor into pivotal Phase 3 development for patients with WHIM syndrome, while also initiating two proof-of-concept clinical trials in SCN and Waldenström’s, and strengthening both our leadership team and Board of Directors. We’re now well positioned for the years ahead as we focus on near-term clinical trial execution and prepare for key value creation events in our quest to bring new transformative therapies to patients with rare diseases.”\n\n\nKey 2019 Program Achievements and Upcoming Milestones\n\n\n\nInitiated Pivotal Phase 3 Clinical Trial of Mavorixafor for the Treatment of WHIM Syndrome – June 2019: The 4WHIM trial is a pivotal Phase 3 global clinical trial of mavorixafor for the treatment of WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease.\n\n\nTop-line data from the trial are expected in the second half of 2021.\n\n\nCompany to hold Analyst Day, which will also be webcast, on April 7, 2020 to discuss strategic focus on WHIM.\n\n\nPhase 2 open-label extension study data update expected in mid-2020.\n\n\n\n\nReceived Orphan Drug Designation from the European Commission for Mavorixafor for the Treatment of WHIM Syndrome – July 2019\n\n\nReceived Scientific Advice from the European Medicines Agency to align the Phase 3 registration trial globally.\n\n\n\n\nAnnounced Positive Data from Phase 2a Trial o...