- X4 Pharmaceuticals Inc 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.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Comments
Loading...
Posted In: BiotechNewsPenny StocksHealth CarePre-Market OutlookMoversTrading IdeasGeneralBriefswhy it's moving
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in