X4 Pharmaceuticals

X4 Pharmaceuticals specializes in developing therapies for rare immune system diseases. The company focuses on the chemokine receptor CXCR4, a cell receptor that regulates immune cell movement. This stems from its crucial role in the maturation and mobilization of white blood cells in the bloodstream.

As of August 2023, the company had three drug candidates in the pipeline, including its lead clinical candidate, mavorixafor, a small-molecule antagonist targeting CXCR4. The Phase III completed drug candidate is an oral, once-daily therapy to treat chronic neutropenia disorders, including the rare primary immunodeficiency, known as the WHIM (warts, hypogammaglobulinemia, infections, and myelokathexis) syndrome, and certain cancers. The company aimed to provide a new treatment option for those who have to rely on injectable therapies with significant side effects.

The company's core research activities and expertise in CXCR4 and immune system biology are conducted at its corporate headquarters in Boston, Massachusetts, with a research center in Austria.

Key customers and partnerships

The company has several key partners including Abbisko Therapeutics (July 2019) to develop and commercialize mavorixafor in China, the Leukemia & Lymphoma Society (May 2019) to develop mavorixafor for the treatment of Waldenström’s macroglobulinemia (WM), a rare form of non-Hodgkin lymphoma, and Invitae (June 2019) to provide genetic testing at no cost to patients through its collaborative PATH4WARD program.  

Funding and financials

The company began trading on the Nasdaq in November 2017 under the ticker symbol “XFOR.” In August 2023, the company secured a USD 115 million loan facility from Hercules Capital. The new funds were channelled to prepare for the potential commercial launch of mavorixafor, continue to develop the drug for certain chronic neutropenic disorders, and create expanded future options beyond equity capital markets, including potentially monetizing a priority review voucher.

For the three months ended March 2023, the company did not generate any revenue. However, it reported a decline in net loss by 9% YoY to USD 24 million. Its cash and cash equivalent as of March 2023 was USD 93.4 million compared with USD 121.7 million in December 2022.