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X4 Pharmaceuticals Inc
X4 Pharmaceuticals, Inc. operates as a biotechnology company, which engages in developing human antibodies for treating infectious diseases. The company is headquartered in Boston, Massachusetts and currently employs 83 full-time employees. The company went IPO on 2017-11-16. The firm is focused on the research, development and commercialization of therapeutics for the treatment of rare diseases with an initial focus on the treatment of people with immune system dysfunction. Its lead product candidate, mavorixafor, is a small molecule inhibitor of the chemokine receptor CXCR4 and is being developed as a once-daily oral therapy. Its mavorixafor demonstrated ability to antagonize CXCR4 and improve the healthy maturation and trafficking of white blood cells (WBCs), which provides therapeutic benefit across a variety of diseases, including primary immunodeficiencies (PIDs) and certain types of cancer. Its mavorixafor is in a global Phase III clinical trial for the treatment of warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome, a rare, inherited PID caused by genetic mutations in the CXCR4 receptor gene. Its product candidates include X4P-001, X4P-002 and X4P-003.
X4 Pharmaceuticals, Inc. operates as a biotechnology company, which engages in developing human antibodies for treating infectious diseases. The company is headquartered in Boston, Massachusetts and currently employs 83 full-time employees. The company went IPO on 2017-11-16. The firm is focused on the research, development and commercialization of therapeutics for the treatment of rare diseases with an initial focus on the treatment of people with immune system dysfunction. Its lead product candidate, mavorixafor, is a small molecule inhibitor of the chemokine receptor CXCR4 and is being developed as a once-daily oral therapy. Its mavorixafor demonstrated ability to antagonize CXCR4 and improve the healthy maturation and trafficking of white blood cells (WBCs), which provides therapeutic benefit across a variety of diseases, including primary immunodeficiencies (PIDs) and certain types of cancer. Its mavorixafor is in a global Phase III clinical trial for the treatment of warts, hypogammaglobulinemia, infections and myelokathexis (WHIM) syndrome, a rare, inherited PID caused by genetic mutations in the CXCR4 receptor gene. Its product candidates include X4P-001, X4P-002 and X4P-003.
CN Trial Progress: X4 is advancing its pivotal Phase III 4WARD trial for mavorixafor in chronic neutropenia (CN), with enrollment ongoing in over 20 countries and more than 90% of sites activated.
XOLREMDI Sales: Cumulative XOLREMDI sales reached $3.5 million since launch; Q1 sales were just under $1 million, slightly lower than Q4 due to inventory timing.
Financials: The company ended Q1 with just under $90 million in cash, expecting to fund operations into the first half of 2026.
Strategic Partnerships: Two international deals were completed for WHIM and CN: Norgine for Europe, Australia, New Zealand, and taiba rare for the Middle East and North Africa.
Patent & Market Opportunity: X4 received a notice of allowance for a U.S. patent for mavorixafor in CN, expiring 2041, and estimates a potential $1–2 billion US market opportunity.
Cost Reduction: Strategic restructuring is expected to cut annual spending by $30–35 million.
