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Lenumlostat (formerly PAT-1251 and GB2064) is a potent, selective, and orally bioavailable small-molecule inhibitor of lysyl oxidase-like 2 (LOXL2). LOXL2 is a copper-dependent amine oxidase that plays a critical role in the cross-linking of collagen and elastin fibers within the extracellular matrix, a process that is central to the progression of tissue fibrosis. By inhibiting LOXL2, lenumlostat aims to reduce the pathological deposition of fibrous tissue in the bone marrow, potentially improving hematologic function in patients with myelofibrosis. The drug was originally developed by PharmAkea and later licensed to Galecto. It has been evaluated in Phase 2 clinical trials, including studies at the M.D. Anderson Cancer Center, for the treatment of primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocytosis myelofibrosis.
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