X4 Pharmaceuticals presents positive data from neutropenia trial

BOSTON - X4 Pharmaceuticals (NASDAQ:XFOR), a clinical-stage biotech company currently trading near its 52-week low of $2.82 (down from $31.20), presented positive results from its completed Phase 2 trial of mavorixafor in the treatment of chronic neutropenic conditions at the European Hematology Association Congress in Milan. According to InvestingPro data, the company maintains strong liquidity with a current ratio of 3.7, though it faces challenges in cash burn rate.

The six-month, open-label clinical trial enrolled 23 participants diagnosed with idiopathic, congenital, or cyclic chronic neutropenia, divided into two groups: those receiving mavorixafor monotherapy and those receiving mavorixafor with injectable granulocyte colony-stimulating factor (G-CSF). With a market capitalization of just $16.32 million, X4 Pharmaceuticals is positioning itself in a potentially lucrative market segment.

Data showed that participants receiving once-daily oral mavorixafor monotherapy experienced durable increases in mean absolute neutrophil count (ANC) over the six-month period. Those with severe chronic neutropenia achieved nearly three-fold increases in mean ANC levels, reaching levels typically targeted by physicians.

The majority of participants (89%) reduced or discontinued injectable G-CSF use while on mavorixafor treatment. All participants with congenital neutropenia concurrently treated with G-CSF were able to decrease G-CSF while maintaining mean ANC at normal levels.

A sub-study indicated that the percentage of functional circulating neutrophils in participants was comparable to that of healthy donors after six months of mavorixafor dosing.

The treatment was generally well tolerated as both monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported.

X4 is currently conducting a global Phase 3 trial to assess mavorixafor’s ability to increase ANC levels while reducing infection rates over 52 weeks. The company expects the trial to complete enrollment in the third or fourth quarter of 2025. Analysts maintain a strong buy consensus on the stock, with revenue growth forecast at 12.6% for the current fiscal year. For detailed analysis and 15 additional key insights, visit InvestingPro, where you can access the comprehensive Pro Research Report covering X4 Pharmaceuticals’ financial health and growth prospects.

Chronic neutropenia is characterized by persistently low levels of circulating neutrophils, increased infection risk, and reduced quality of life. Currently, only one approved therapy exists for this condition.

This information is based on a press release statement from X4 Pharmaceuticals.

In other recent news, X4 Pharmaceuticals has reported impressive financial results for the first quarter of 2025, with earnings per share reaching $0.04, significantly surpassing the forecasted loss of $0.12. The company’s revenue also exceeded expectations, coming in at $28.81 million compared to the anticipated $12.03 million. This performance was bolstered by strong license revenue from a partnership with Norgene. Additionally, X4 Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to its drug mavorixafor for treating chronic neutropenia. This designation is intended to expedite the review of drugs that address serious conditions with unmet medical needs. Meanwhile, H.C. Wainwright has raised its price target for X4 Pharmaceuticals to $7.00, up from the previous $1.50, while maintaining a Buy rating on the stock. The firm cited the company’s ongoing progress with its Phase 3 4WARD study as a key factor in this decision. X4 Pharmaceuticals is also advancing its clinical trials, with the 4WARD trial expected to complete enrollment by the second half of 2025.

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