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X4 Pharmaceuticals (NASDAQ:XFOR) traded sharply lower on Thursday after the biotech updated data from an ongoing Phase 2 trial for its lead candidate mavorixafor in chronic neutropenia (CN), a rare blood disorder.
Citing a May 24 data cut, the Boston, Massachusetts-based biotech said mavorixafor, which X4 (XFOR) markets as Xolremdi for an ultra-rare disorder called WHIM syndrome, demonstrated a safety profile consistent with its previous clinical studies.
However, three out of 23 subjects who received the drug discontinued the therapy due to non-serious adverse events.
CN is characterized by a low number of white blood cells known as neutrophils, which leads to a higher incidence of infections.
As for efficacy, the company said that once-daily oral mavorixafor as a single agent with or without granulocyte colony-stimulating factor (G-CSF), the current FDA-approved CN therapy, caused an increase in patients’ neutrophil counts at months three and six.
The conclusion was based on six subjects who had completed the six-month open-label clinical trial. More data from the study is expected later this year.