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Orphan drug designation granted to PCLX-001 by the FDA
On October 14, 2022, PCLX-001, a first-in-class, oral, small-molecule N-myristoylation inhibitor currently in clinical development, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with acute myeloid leukemia (AML).1
PCLX-001 has shown positive results in animal models of AML and in AML patient-derived xenografts, where PCLX-001 treatment reduced human peripheral blood and bone marrow CD45+ cells by up to 95%.1
PCLX-001 is currently being evaluated in a phase I trial in patients with non-Hodgkin lymphoma and solid tumors in Canada (NCT04836195). An investigational new drug application has been filed to study PCLX-001 in patients with AML in the United States.1
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Approximately what proportion of your patients with FLT3-mutations also have NPM1 and DNMT3A co-mutations?