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X4 Pharmaceuticals
X4 Pharmaceuticals is a Boston biotech whose oral CXCR4 blocker mavorixafor won approval as Xolremdi for WHIM syndrome and is being tested to free neutrophils in chronic neutropenia and to open tumours to immune attack.
Also called: X4 Pharmaceuticals, Inc.NASDAQ:XFOR
Overview
X4 Pharmaceuticals develops mavorixafor (X4P-001, formerly AMD-070), an oral antagonist of the chemokine receptor CXCR4. The FDA approved it in April 2024 as Xolremdi for patients aged 12 and older with WHIM syndrome, a rare immunodeficiency. The phase 3 4WARD trial tests it in chronic neutropenia. In oncology the drug has been studied with axitinib in renal cell carcinoma, with pembrolizumab in melanoma and in Waldenström macroglobulinaemia, where CXCR4 mutations confer resistance to BTK inhibitors.
Headquarters
Boston, Massachusetts, US
Scorecard
All companies#275of 1244 companies, score 17
- approved products +6
- targets +2
- modalities +2
- regions +1
- registry trials +6
1 approved, 0 in phase 3, 0 earlier, 1 targets, 8 registry studies. Formula on the scorecards page.
Type
Biotech · NASDAQ:XFOR
Stage
Public
Website
Founded
2014
ApprovedOral CXCR4 antagonist
Mavorixafor · Xolremdi
Mavorixafor is a once-daily tablet that blocks CXCR4, a receptor that traps white cells in the bone marrow. It is approved for the rare immunodeficiency WHIM syndrome and is being tested in Waldenström macroglobulinaemia, where CXCR4 mutations blunt the response to ibrutinib.