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X4 Pharmaceuticals Shares Tumble After Phase 3 Data From Rare Immune Deficiency Candidate

  • X4 Pharmaceuticals Inc (NASDAQ: XFOR) announced topline results from the pivotal Phase 3 (4WHIM) trial of its lead investigational therapy, mavorixafor, a novel CXCR4 antagonist, in people with WHIM syndrome.

  •  Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) is a rare congenital immune deficiency in which individuals are more susceptible to potentially life-threatening bacterial infections.

  • 4WHIM met its primary endpoint, with mavorixafor achieving clinical and statistical superiority over placebo when measuring TATANC, or the length of time that participants' absolute neutrophil counts (ANC) remained above a clinically meaningful threshold of 500 cells per microliter (severe neutropenia), over 24-hour periods at 4 time points throughout the 52-week trial.

  • Related: X4 Pharma Highlights New Data Supporting Mavorixafor's Broad Potential In Chronic Neutropenia.

  • Mean TATANC was 15.04 hours in the treatment group versus 2.75 hours in the placebo group.

  • 4WHIM also met a key secondary endpoint, with mavorixafor achieving clinical and statistical superiority over placebo when measuring TATALC, or the length of time that participants' absolute lymphocyte counts (ALC) remained above a clinically meaningful threshold of 1,000 cells per microliter (lymphopenia), over 24-hour periods at 4 time points throughout the 52-week trial. Mean TATALC was 15.80 hours in the treatment group versus 4.55 hours in the placebo group.

  • Mavorixafor was generally well tolerated in the trial, with no treatment-related serious adverse events reported and no discontinuations for safety events.

  • Price Action: XFOR shares are 17.30% lower at $1.53 during premarket trading on Wednesday.

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