Pacylex Pharmaceuticals has launched a new clinical trial investigating zelenirstat, a first-in-class oral therapy targeting N-myristoyltransferase inhibitors (NMTis), for the treatment of refractory and relapsed 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 after previous treatments. Partially supported by a US Department of Defense grant, the trial represents a significant step in developing potential new treatment options for AML patients.
Preclinical research suggests AML cells are particularly sensitive to zelenirstat. Previous Phase 1 studies in solid tumor and lymphoma patients demonstrated promising results, including an acceptable safety profile and signs of efficacy. In the earlier trial of 29 heavily pre-treated patients, zelenirstat showed self-limited adverse events and prolonged progression-free survival at recommended dosage levels.
Dr. John Mackey, Pacylex's Chief Medical Officer, emphasized the potential of the drug, noting that preliminary data indicates AML cells may be more responsive to zelenirstat compared to other cancer types. The FDA has already granted zelenirstat both Orphan Drug and Fast Track Designations for AML, underscoring the potential significance of this research.
The ongoing clinical trial represents a critical opportunity to assess zelenirstat's effectiveness in a challenging cancer subtype with limited treatment options. If successful, the research could provide a new targeted therapeutic approach for AML patients who have exhausted existing treatment protocols.
