X4 Pharmaceuticals Announces Data from Phase 2 Open-Label Extension Trial of Mavorixafor in WHIM Syndrome as Published in EHA Abstract
Data reveal reductions in annual infections and warts, and sustained dose-dependent increases in neutrophil counts
These and additional Phase 2 data to be presented in an e-Poster on
The original Phase 2 clinical trial was a two-part, open-label, dose-escalation study of mavorixafor in adult patients followed by an open-label extension study to determine the safety, tolerability, and dose selection of mavorixafor in participants with genetically confirmed WHIM syndrome. The extension phase was open to patients who completed at least 24 weeks of the initial dose-escalation study and explored additional endpoints related to infection rates and wart burden, along with long-term safety. The trial results informed the design of the company’s ongoing pivotal Phase 3 clinical trial (4WHIM). Data from the Phase 2 trial also resulted in the receipt of Breakthrough Therapy Designation by the
Data from patients treated with 300 mg or 400 mg once-daily doses of mavorixafor for up to 28.6 months will be presented at this year’s
- Observed sustained, dose-dependent increases in absolute neutrophil count (ANC) and absolute lymphocyte count (ALC).
Long-term hematological improvements correlated with fewer infections and improved cutaneous warts.
- Clinical yearly infection rates decreased from 4.63 in the 12 months prior to the trial, to 2.41 (a 48% reduction) at 300 mg and 2.14 (a 54% reduction) at 400 mg of mavorixafor.
- Patients with cutaneous warts on hands and/or feet at baseline demonstrated a greater than 50% reduction in the number of warts at their last dermatological evaluation.
- Mavorixafor was well tolerated for the extended duration of more than two years without any attributable serious adverse effects.
These and additional data from the Phase 2 open-label extension study will be presented during a virtual e-Poster presentation:
Abstract #EP852: Oral CXCR4 Antagonist Mavorixafor Treatment in Patients with WHIM Syndrome: Results of an Open-label Phase 2 Study with Long-term Extension
Date and Time:
Session Title: Bone marrow failure syndromes incl. PNH - Clinical
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” or other similar terms or expressions that concern X4's expectations, strategy, plans or intentions. Forward-looking statements include, without limitation, statements regarding X4’s plans for clinical development of mavorixafor, including the timing of completion and results of its global Phase 3 clinical trial in patients with WHIM syndrome, estimates regarding the WHIM patient population and potential market opportunity, and the potential therapeutic benefit of mavorixafor. Any forward-looking statements in this press release are based on management's current expectations and beliefs. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the
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