X4 Pharmaceuticals

X4 Pharmaceuticals, Inc

18 Investors

Biopharmaceutical

BOSTON, MA

X4 Pharmaceuticals, Inc. is a commercial-stage biopharmaceutical company focused on developing and commercializing therapies for rare diseases of the immune system. The company has successfully transitioned from clinical to commercial operations following the U.S. FDA approval of its lead product, XOLREMDI®. Its core scientific focus is on the CXCR4 pathway, a crucial regulator of immune cell trafficking.

Products & Team

XOLREMDI® (mavorixafor)

Rare Disease TherapySeed

XOLREMDI® is an oral, once-daily, small molecule antagonist of the CXCR4 receptor. It functions by blocking the binding of the CXCL12 ligand to the receptor, which increases the mobilization and circulation of mature white blood cells from the bone marrow into the bloodstream. It is the first therapy specifically approved for patients aged 12 and older with WHIM syndrome, a rare primary immunodeficiency.

Value Proposition

XOLREMDI® addresses a significant unmet medical need by providing a targeted therapy for patients with WHIM syndrome, a condition that previously had no approved treatments and leads to severe, recurrent infections and other complications.

Pain Points

Patients with these rare disorders face chronic and severe health challenges, including recurrent infections, low white blood cell counts, and a lack of effective, targeted treatment options, leading to a significant impact on their quality of life.

Oral, once-daily administrationSmall molecule antagonist of the CXCR4 receptorFirst and only FDA-approved therapy for WHIM syndromeMechanism of action increases circulating neutrophils and lymphocytes