The presentation will detail final safety and efficacy results from the company's Phase 2a portion of an open-label Phase 1/2 clinical trial of mavorixafor in combination with axitinib in patients with advanced clear cell renal cell carcinoma (ccRCC).
X4 Pharmaceuticals' lead product candidate, mavorixafor (X4P-001), is a potential first-in-class, once-daily, oral inhibitor of CXCR4, currently in Phase 3 development for the treatment of WHIM syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.
Mavorixafor has demonstrated proof of concept in WHIM syndrome in a Phase 2 trial, including clinically meaningful increases in neutrophil and lymphocyte biomarker counts, as well as a trend of reduction in infection rates and wart burden, and a favorable safety profile.
Mavorixafor was designated orphan drug status by the US Food and Drug Administration in 2018 and by the European Commission in 2019 for the treatment of WHIM syndrome, and is also in development for the treatment of Severe Congenital Neutropenia, Waldenström's macroglobulinemia, and clear cell renal cell carcinoma (ccRCC).
X4 Pharmaceuticals is developing novel therapeutics designed to improve immune cell trafficking to treat rare diseases, including primary immunodeficiencies and certain cancers.
The company's oral small molecule drug candidates antagonise the CXCR4 pathway, which plays a central role in immune surveillance.
X4's most advanced product candidate, mavorixafor (X4P-001), is in a global Phase 3 pivotal trial in patients with WHIM syndrome, a rare, inherited, primary immunodeficiency disease, and is currently also under investigation in combination with axitinib in the Phase 2a portion of an open-label Phase 1/2 clinical trial in clear cell renal cell carcinoma (ccRCC).
X4 is also planning to commence clinical trials of mavorixafor in Severe Congenital Neutropenia and Waldenström's macroglobulinemia in 2019.
The company was founded and is led by a team with extensive biopharmaceutical product development and commercialisation expertise and is committed to advancing the development of innovative medicines on behalf of patients with limited treatment options.
X4 is a global company that is headquartered in Cambridge, Massachusetts with research offices based in Vienna, Austria.
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