Study of Oral PCLX-001 in R/R Acute Myeloid Leukemia

Summary

This is a dose-finding study of oral zelenirstat (PCLX-001) in patients with R/R AML. There are two parts to the study: Dose Escalation and Dose Expansion.

Timeline

Start

2025-03-03

Primary completion

2026-03-01

Completion

2026-09-01

Publications

• Background Beauchamp E, Yap MC, Iyer A, Perinpanayagam MA, Gamma JM, Vincent KM, Lakshmanan M, Raju A, Tergaonkar V, Tan SY, Lim ST, Dong WF, Postovit LM, Read KD, Gray DW, Wyatt PG, Mackey JR, Berthiaume LG. Targeting N-myristoylation for therapy of B-cell lymphomas. Nat Commun. 2020 Oct 22;11(1):5348. doi: 10.1038/s41467-020-18998-1.
• Background Sangha R, Davies NM, Namdar A, Chu M, Spratlin J, Beauchamp E, Berthiaume LG, Mackey JR. Novel, First-in-Human, Oral PCLX-001 Treatment in a Patient with Relapsed Diffuse Large B-Cell Lymphoma. Curr Oncol. 2022 Mar 13;29(3):1939-1946. doi: 10.3390/curroncol29030158.
• Results Beauchamp E, Gamma JM, Cromwell CR, Moussa EW, Pain R, Kostiuk MA, Acevedo-Morantes C, Iyer A, Yap M, Vincent KM, Postovit LM, Julien O, Hubbard BP, Mackey JR, Berthiaume LG. Multiomics analysis identifies oxidative phosphorylation as a cancer vulnerability arising from myristoylation inhibition. J Transl Med. 2024 May 7;22(1):431. doi: 10.1186/s12967-024-05150-6.

Drugs

Indications