MicroStockHub 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. More on X4 Pharmaceuticals X4 Pharmaceuticals Inc (XFOR) Q1 2024 Earnings Call Transcript X4 Pharmaceuticals: FDA Approval For WHIM Warrants Further Upside X4 Pharmaceuticals: PDUFA Excitement Ahead X4 Pharma gains on new $125 financing X4 Pharmaceuticals GAAP EPS of -$0.26.
