Pacylex Announces FDA Clearance of IND Application for PCLX-001 to Treat Acute Myeloid Leukemia

Pacylex, a clinical-stage NMT inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for PCLX-001 for the treatment of acute myeloid leukemia (AML). The open IND enables a Phase 1/2 clinical study to start in the coming months at The University of Texas MD Anderson Cancer Center, which was recently awarded a US Department of Defense (DOD) grant for this purpose.

Last month the U.S. FDA also granted PCLX-001 Orphan Drug Designation for “treatment of patients with acute myeloid leukemia.” PCLX-001 is a first-in-class N-myristoylation inhibitor in clinical development by Pacylex. PCLX-001 is currently being studied in non-Hodgkin lymphoma (NHL) and solid tumor cancer patients at 4 sites in Canada. Pacylex has the first and only NMT inhibitor in clinical studies.

“With this IND clearance, Pacylex opens up a second clinical indication for the investigation of PCLX-001 in patients,” said Michael Weickert, PhD, CEO of Pacylex. In the ongoing studies of PCLX-001, seventeen patients have been accrued through 5 dose levels of oral, once-per-day PCLX-001 with no dose limiting toxicities observed.

“Non clinical data suggests that AML may be the cancer type most sensitive to NMT inhibitors, so we are excited to move PCLX-001 into clinical studies in AML patients in the near future,” said John Mackey, MD, CMO of Pacylex. Luc Berthiaume, PhD, CSO of Pacylex commented, “It is gratifying to see the pre-clinical work pioneered in my lab, translating to clinical investigations and the potential to help patients in the near future.” Data on the scientific rationale for PCLX-001 in AML patients, will be presented at the American Society of Hematology (ASH) Annual Meeting and Exposition from 10-13 December 2022.

The ongoing clinical PCLX-001 trial in NHL and solid tumor patients is registered at ClinicalTrials.gov Identifier: NCT04836195.

Read the full press release here.

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