Pacylex Pharmaceuticals has commenced a pioneering clinical trial investigating zelenirstat, an innovative oral therapy targeting N-myristoyltransferase (NMT) for the treatment of Acute Myeloid Leukemia (AML). The study, led by Dr. Naveen Pemmaraju at The University of Texas MD Anderson Cancer Center, will enroll patients whose disease has progressed or relapsed after standard treatments.
The clinical investigation follows promising preclinical research demonstrating zelenirstat’s ability to efficiently kill AML cancer cells, including cancer stem cells. Early research published in scientific journals suggests that AML cells are particularly sensitive to this targeted therapy, creating significant potential for treatment advancement.
Previous Phase 1 dose escalation studies in 29 heavily pre-treated solid tumor and lymphoma patients revealed encouraging results. The research showed self-limited adverse events and no dose-limiting toxicities at daily doses up to 280mg. Notably, patients receiving the recommended Phase 2 dose of 210mg experienced prolonged progression-free and overall survival.
The study is partially supported by a grant from the US Department of Defense, underscoring the potential clinical significance of zelenirstat. The US Food and Drug Administration has already granted zelenirstat both Orphan Drug Designation and Fast Track Designation for AML, highlighting the therapy’s innovative approach.
Zelenirstat represents a first-in-class, oral small-molecule NMT inhibitor with potential applications across hematologic cancers and solid tumors. Preclinical studies have demonstrated its ability to selectively target and kill cancer cells, with regression observed in hematologic malignancies and inhibition of lung and breast cancer tumor growth in animal models.
As a world leader in developing N-myristoyltransferase inhibitors, Pacylex Pharmaceuticals continues to advance targeted cancer therapies. The ongoing AML clinical trial represents a critical step in evaluating zelenirstat’s potential to provide new treatment options for patients with limited existing alternatives.
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