Core Insights - X4 Pharmaceuticals announced the acceptance of two abstracts for presentation at the 30th Annual Congress of the European Hematology Association, highlighting positive results from a Phase 2 trial of mavorixafor for chronic neutropenia [1][2] - Mavorixafor is positioned as a potential first oral treatment for chronic neutropenia in over 30 years, with ongoing Phase 3 trials supporting its efficacy and safety [2][8] Company Overview - X4 Pharmaceuticals focuses on developing therapies for rare immune system diseases, with mavorixafor being a key product currently marketed in the U.S. as XOLREMDI® [9] - The company is headquartered in Boston, Massachusetts, and is actively pursuing additional indications for mavorixafor [9] Clinical Trial Details - The Phase 2 trial was a six-month open-label study involving 23 participants with various forms of chronic neutropenia, assessing the safety and efficacy of mavorixafor [3][7] - The ongoing pivotal Phase 3 trial, named 4WARD, aims to evaluate mavorixafor's efficacy and safety in a larger cohort of 150 participants with chronic neutropenia [8] Research Findings - The Phase 2 study demonstrated meaningful increases in functional neutrophils, with many participants able to reduce their G-CSF dosing when treated with mavorixafor [1][6] - Mavorixafor acts as a CXCR4 antagonist, mobilizing neutrophils from the bone marrow into the bloodstream, which is crucial for managing chronic neutropenia [5][6]
X4 Pharmaceuticals Announces Upcoming Presentation of Phase 2 Chronic Neutropenia Trial Data at the 30th Annual EHA Congress