X4 Pharmaceuticals pulls together some positive data results after a rocky year
X4 Pharmaceuticals said its rare disease drug cleared a Phase III on Tuesday afternoon, coming against a backdrop of layoffs and pipeline pruning earlier this year.
In the topline results, the Boston biotech said its lead therapy mavorixafor, a CXCR4 antagonist which the biotech picked up from Sanofi, met its primary endpoint in a trial of patients with WHIM syndrome, a rare immune deficiency.
The trial hit “clinical and statistical superiority” over the placebo when measuring the length of time that patients’ “absolute neutrophil counts (ANC)” had remained above the threshold of 500 cells per microliter over 24-hour periods throughout the 52-week trial. It also had 15 hours in the treatment group against the two hours in the placebo group, yielding a p-value of <0.0001.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.
