X4 Pharmaceuticals to Host Virtual Event on June 27, 2024, Reviewing New Mavorixafor Phase 2 Data in Chronic Neutropenia

BOSTON, May 29, 2024 – X4 Pharmaceuticals (Nasdaq: XFOR), a company dedicated to enhancing the lives of individuals with rare immune system diseases, has announced a virtual investor event scheduled for June 27, 2024, at 8:00 am ET. The event will highlight interim findings from an ongoing Phase 2 clinical trial that evaluates the efficacy of mavorixafor as a once-daily oral treatment for patients suffering from chronic neutropenia (CN).

The virtual event will present hematological data from a minimum of 15 participants who have been treated with either mavorixafor alone or in combination with stable doses of injectable granulocyte colony-stimulating factor (G-CSF). Additionally, expert physicians in the field will provide commentary on these clinical results and discuss the significant unmet needs in the treatment of CN.

X4 Pharmaceuticals also reiterated its plans to commence a global, pivotal Phase 3 clinical trial within this quarter. This trial aims to assess the effectiveness, safety, and tolerability of oral mavorixafor, with or without G-CSF, in patients with congenital or acquired primary autoimmune and idiopathic chronic neutropenia who are experiencing recurrent and/or severe infections.

A live Q&A session will follow the formal presentation, allowing participants to engage directly with the experts and company representatives.

Understanding Chronic Neutropenia and Mavorixafor

Chronic neutropenia is characterized by persistently or intermittently low levels of neutrophils, a type of white blood cell crucial for fighting infections. This condition lasts over three months and is associated with a heightened risk of infections and reduced quality of life. Neutrophils are typically retained in the bone marrow due to the CXCR4/CXCL12 axis. The administration of G-CSF or the inhibition of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, helps mobilize neutrophils from the bone marrow into the bloodstream.

About X4 Pharmaceuticals

X4 Pharmaceuticals is committed to advancing innovative therapies for rare immune system diseases with significant unmet medical needs. Utilizing their expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, which has been approved in the U.S. under the brand name XOLREMDI™ for its first indication. The company is also investigating additional potential indications for mavorixafor. Headquartered in Boston, Massachusetts, X4 Pharmaceuticals operates a research center of excellence in Vienna, Austria.

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