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.

The trial also met a secondary endpoint of having superiority over the length of time that the patient’s absolute lymphocyte counts remained over 1,000 cells per microliter over 24 hours, which also earned a p-value of <0.0001. No adverse events related to treatment or discontinuations over safety were reported.

Murray Stewart

X4’s interim CMO Murray Stewart said in a statement that the company will meet with US regulatory authorities sometime in the first half of next year to discuss the next steps for mavorixafor, including submission for regulatory approval and commercialization.

X4’s stock price rose 5% since the opening bell Wednesday, but it is still down 58% to where the price was in November of last year.

“These data not only give us strong confidence in the potential of mavorixafor to make a difference in the lives of those with WHIM syndrome and their families but also strengthen our resolve to further evaluate mavorixafor in people living with chronic neutropenic disorders beyond those with WHIM,” X4 CEO Paula Ragan said in a statement.

The data release comes after X4 hit some turbulence in the summer. In July, to beat back a heavy bear market, the biotech had agreed to sell over 50 million shares of common stock to certain investors in a PIPE financing, which was expected to bring in around $55 million. However, just a few weeks later, X4 announced it would downsize its research to focus on mavorixafor. The biotech also let go of around 20% of its staff in the process.

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