At FORMA, patients are our purpose. We are dedicated to delivering transformative medicines to patients with cancer and rare diseases. We are a fully-integrated, privately-held clinical stage biopharmaceutical company. A leader in metabolism, epigenetics and protein homeostasis focused on the discovery, development and commercialization of transformative medicines. Our proprietary R&D platform combines deep biology insight, chemistry expertise and clinical development capabilities to create differentiated drug candidates. We have delivered high-value clinical candidates to our partners and generated a broad proprietary portfolio of internal programs, ranging from pre-clinical to pivotal-stage, with the potential to provide profound patient benefit in hematologic, oncologic and metabolic indications.
X4 Pharmaceuticals (Nasdaq: XFOR) 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 antagonize 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 (SCN) and Waldenström`s macroglobulinemia (WM) in 2019. The company was founded and is led by a team with extensive biopharmaceutical product development and commercialization 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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