Zelenirstat Phase 1 Results: Safety and Efficacy in Cancers

Pacylex Pharmaceuticals, a clinical-stage company based in Edmonton, Canada, is making strides in cancer treatment with its lead product candidate, zelenirstat. This innovative drug is an N-myristoyltransferase inhibitor (NMTi), a new class of targeted therapies designed to combat both hematologic and solid tumor cancers. The company recently announced that it will present the results of its Phase 1 study on the safety and tolerability of zelenirstat at the ASCO Gastrointestinal Cancers Symposium, taking place from January 18-20, 2024, in San Francisco.

The study's findings are promising, indicating that zelenirstat has the potential to extend progression-free survival in patients with gastrointestinal and other types of cancer. Notably, a patient with advanced colorectal cancer, who had previously undergone seven lines of therapy, experienced a significant reduction in both CEA levels and tumor volumes after treatment with zelenirstat.

Zelenirstat is a first-of-its-kind oral, small molecule NMT inhibitor. It has shown remarkable efficacy in preclinical studies, selectively targeting and killing cancer cells while promoting the growth of healthy bone marrow cells. The drug's development has been supported by various organizations, including the US Department of Defense and the Cure Cancer Foundation.

Currently, Pacylex is conducting a Phase 2a study to further evaluate the drug's safety, tolerability, and clinical activity in patients with relapsed or refractory B-cell non-Hodgkin Lymphoma (NHL). Additionally, plans are underway to include a separate cohort of patients with advanced solid malignancies who have exhausted all standard treatment options.

Pacylex Pharmaceuticals has received both Orphan Drug Designation and Fast Track Designation from the FDA for zelenirstat's application in treating acute myeloid leukemia (AML). These designations underscore the drug's potential to address unmet medical needs and expedite its development and review process.

The company's commitment to advancing cancer treatment is evident through its ongoing clinical trials and the support it has garnered from various institutions. As Pacylex continues to explore the therapeutic potential of NMT inhibitors, the future looks promising for patients seeking novel and effective cancer therapies.