Pacylex Pharmaceuticals has begun a critical clinical trial investigating zelenirstat, a novel targeted therapy for Acute Myeloid Leukemia (AML), marking a significant advancement in cancer treatment strategies. The Phase 1/2 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 conventional treatments.
The clinical investigation builds upon compelling preclinical research demonstrating zelenirstat's potential to efficiently eliminate AML cancer cells, including challenging cancer stem cells. Previous Phase 1 studies in solid tumor and lymphoma patients revealed promising safety profiles and early efficacy signals, providing optimism for this new AML-focused trial.
Zelenirstat represents a first-in-class oral small-molecule N-myristoyltransferase inhibitor (NMTi) designed to selectively target and destroy cancer cells. The drug has already received significant regulatory recognition, including Orphan Drug Designation and Fast Track Designation from the FDA for AML treatment.
The ongoing study is partially supported by a grant from the US Department of Defense, underscoring the potential clinical significance of this therapeutic approach. Dr. John Mackey, Pacylex's Chief Medical Officer, expressed enthusiasm about testing the drug in AML patients, noting that preclinical results suggest these cancer cells may be particularly sensitive to zelenirstat.
Prior Phase 1 research demonstrated that patients receiving the recommended 210mg dose experienced prolonged progression-free and overall survival compared to lower dose groups. The current AML trial will further explore the drug's therapeutic potential in a patient population with limited treatment options.
This research represents a critical step in developing targeted cancer therapies that could offer more precise and potentially less toxic treatment alternatives. By focusing on N-myristoyltransferase inhibition, Pacylex is exploring an innovative approach to interrupting cancer cell growth and survival mechanisms.
