Pacylex Pharmaceuticals has commenced a Phase 1/2 clinical trial investigating zelenirstat, a first-in-class oral therapy targeting N-myristoyltransferase inhibitors (NMTis), for patients with refractory or relapsed Acute Myeloid Leukemia (AML).
The clinical study, led by Dr. Naveen Pemmaraju at The University of Texas MD Anderson Cancer Center, will enroll AML patients whose disease has progressed after previous treatments. Partially supported by a US Department of Defense grant, the trial builds on preclinical research demonstrating zelenirstat's ability to efficiently kill AML cancer cells, including cancer stem cells.
Previous Phase 1 research in 29 heavily pre-treated solid tumor and lymphoma patients showed promising results. The study revealed self-limited adverse events and no dose-limiting toxicities at daily doses below 280mg. Patients receiving the recommended 210mg dose experienced prolonged progression-free and overall survival.
Dr. John Mackey, Pacylex's Chief Medical Officer, emphasized the potential significance of the trial, noting that preclinical results suggest AML cells are particularly sensitive to zelenirstat. The FDA has already granted the drug Orphan Drug Designation and Fast Track Designation for AML, underscoring its potential clinical importance.
The trial represents a critical step in developing targeted cancer therapies, potentially offering new hope for patients with treatment-resistant AML. Detailed study information is available on ClinicalTrials.gov under the identifier NCT06613217.
