X4 Pharmaceuticals ( NASDAQ: XFOR ) said its drug mavorixafor helped in increasing a type of white blood cell called neutrophil in people with chronic neutropenia (CN) in a phase 1b trial.
Neutropenia occurs when the body has too few neutrophils.
The study is evaluating mavorixafor's ability to increase the absolute neutrophil count (ANC) in people with idiopathic, cyclic or congenital CN, as monotherapy or concurrently with injectable granulocyte colony-stimulating factor (G-CSF).
The company said all participants (n=25) in the trial responded to treatment with a single 400-mg dose of mavorixafor alone or dosed concurrently with G-CSF.
Participants achieved a mean ANC increase at peak of >3,000 cells/microliter, the company added in a Sept. 27 press release.
X4 noted that consistent responses were seen across all the CN disorders studied – idiopathic, cyclic, and congenital.
In addition, the company said that all neutropenic participants (n=14) reached normalized ANC levels (>1,500 cells/microliter).
"We are very pleased that this Phase 1b trial achieved all of its key objectives, and, most importantly, that all patients dosed with mavorixafor responded with meaningful increases in neutrophil counts," said X4 Chief Medical Officer Diego Cadavid.
When assessed in participants with moderate or severe neutropenia despite being treated with G-CSF (n=8), 100% reached normalized ANC levels, the company noted.
X4 added that assessed in participants with CN with normalized ANC counts on chronic G-CSF (n=11), all these people experienced a consistent and sustained increase in ANC.
Mavorixafor was well tolerated and all treatment-related adverse events were deemed to be low grade, consistent with previous clinical studies in WHIM syndrome, the company added.
XFOR +9.90% to $0.98 premarket Sept. 27
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X4 stock rises 10% as mavorixafor shows promise in trial for white blood cell disorder