Pacylex Reveals Zelenirstat Phase 1 Data at ASCO 2024

Edmonton, Alberta – Pacylex Pharmaceuticals, a clinical-stage pharmaceutical company headquartered in Edmonton, Alberta, has announced that the complete Phase 1 clinical results for zelenirstat, an N-myristoyltransferase (NMT) inhibitor, will be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting. The event is scheduled from May 31 to June 4, 2024, at McCormick Place in Chicago, Illinois.

Key Findings on Zelenirstat

Zelenirstat, a potent oral NMT inhibitor, has shown promising results in Phase 1 studies. The drug, known scientifically as PCLX-001, targets various hematologic cancers and solid tumors. Key findings from the study revealed that zelenirstat was generally well tolerated among patients, with the majority of adverse events being gastrointestinal side effects that resolved spontaneously without requiring dose adjustment.

Patients administered the recommended Phase 2 dose exhibited significantly improved progression-free and overall survival rates compared to those on lower doses. Notably, 57% of these patients maintained stable disease or better for over six months, despite having five different types of cancer. The mechanism of action for zelenirstat involves inhibiting the myristoylation of several oncogenic targets, leading to the suppression of validated cancer targets like EGFR and VEGFR.

About Zelenirstat (PCLX-001)

Zelenirstat is a pioneering oral small molecule drug aimed at treating leukemia, lymphoma, and various solid tumors. Preclinical studies demonstrated that zelenirstat effectively kills cancer cells in vitro and in animal models, leading to the regression of hematologic malignancies and suppression of lung and breast cancer tumors. In models of acute myeloid leukemia (AML), zelenirstat preferentially targeted leukemic stem cells, significantly reducing the leukemic burden in bone marrow.

Phase 1 and Phase 2 Study Overview

Pacylex completed the dose escalation portion of the Phase 1 trial, focusing on multiple ascending doses to evaluate safety, tolerability, and pharmacokinetics in patients with relapsed or refractory lymphoma and solid tumors. The findings supported the recommendation of a Phase 2 dose, showing a favorable safety profile and pharmacokinetics conducive to once-daily oral dosing, along with early signs of efficacy.

Currently, zelenirstat is being evaluated in two Phase 2a open-label studies. One study focuses on patients with relapsed or refractory B-cell non-Hodgkin Lymphoma (NHL), while the other targets patients with metastatic colorectal cancer that has not responded to standard therapies.

About Pacylex Pharmaceuticals

Pacylex Pharmaceuticals is a clinical-stage biotech firm based in Edmonton, Alberta, dedicated to developing NMT inhibitors as treatments for both hematologic and solid tumors. Zelenirstat, the company's lead drug, represents a new class of NMT inhibitors designed to exploit NMTs as therapeutic targets in cancer treatment. Pacylex is actively conducting two multicenter Phase 2a studies in Canada involving patients with relapsed/refractory NHL and metastatic colorectal cancer. Additionally, the FDA has cleared the investigational new drug (IND) application for a Phase 1 study in AML, granting zelenirstat both Orphan Drug Designation and Fast Track Designation for this indication. The initial clinical investigation of zelenirstat in AML patients is being supported by the U.S. Department of Defense. Furthermore, the Cure Cancer Foundation has backed the early clinical studies through its World's Longest Games initiative.

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