Pacylex Pharmaceuticals Reports Zelenirstat Phase 1 Safety and Efficacy Data at ASCO 2024

May 24, 2024 9:08 AM EDT | Source: Reportable, Inc.

Edmonton, Alberta--(Newsfile Corp. - May 24, 2024) - Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company developing N-myristoyltransferase (NMT) inhibitors as targeted therapies for the treatment of hematologic cancers and solid tumors. Today Pacylex announced that complete Phase 1 clinical study results for zelenirstat, a proprietary, potent, NMTi, will be presented at the American Society of Clinical Oncology (ASCO) Annual Meeting from May 31 to June 4, 2024, at McCormick Place in Chicago, Illinois.

To view the full announcement, including downloadable images, bios, and more, click here.

Key Takeaways:

  • Zelenirstat was well tolerated, and adverse events were primarily gastrointestinal side effects, most of which resolved spontaneously without dose reduction.
  • Patients receiving the recommended Phase 2 dose of zelenirstat had significantly better progression free and overall survival than those treated at lower doses; 57% had stable disease or better for six months or longer, despite having 5 different types of cancer.
  • Zelenirstat inhibits myristoylation of multiple oncogenic targets leading to the inhibition of the signaling of validated colon cancer targets EGFR and VEGFR.

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About zelenirstat (PCLX-001)

Zelenirstat (formerly identified as PCLX-001) is a first-in-class, oral, small molecule NMTi being developed to treat patients with leukemia, lymphoma, and solid tumors. Zelenirstat selectively killed cancer cells in vitro and in animal models has been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors. In AML models, zelenirstat preferentially killed leukemic stem enriched cell populations and reduction of the leukemic burden directly in the bone marrow.

About zelenirstat Phase 1 and 2 studies 

Pacylex completed the dose escalation phase of a Phase 1 multiple ascending dose safety, tolerability, and pharmacokinetics study on zelenirstat in people with relapsed/refractory lymphoma and refractory solid tumors (NCT04836195). A recommended Phase 2 dose was determined. Zelenirstat demonstrated an acceptable safety and tolerability profile, pharmacokinetics consistent with once daily oral dosing, and early signs of efficacy.

Zelenirstat is currently being studied in a Phase 2a open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of zelenirstat in patients with relapsed/refractory B-cell non-Hodgkin Lymphoma (NHL) and a separate Phase 2a cohort in patients with refractory metastatic colorectal cancer that has progressed despite all available standard therapies. 

About Pacylex Pharmaceuticals

Pacylex is a clinical-stage pharmaceutical company headquartered in Edmonton, Alberta, Canada, targeting hematologic and solid cancers with orally bioavailable NMT inhibitors. Zelenirstat is the lead drug in a new class of NMT inhibitors, enabling Pacylex to exploit NMTs as new clinical targets for cancer treatment. Pacylex is conducting two multi-center Phase 2a studies in Canada in patients with relapsed/refractory NHL and refractory metastatic colorectal cancer. The IND for a Phase 1 multiple ascending dose study in acute myeloid leukemia (AML) has been cleared and the FDA has granted zelenirstat both Orphan Drug Designation and Fast Track Designation for AML. The US Department of Defense is supporting the initial clinical investigation of zelenirstat in patients with AML. The Cure Cancer Foundation supported initial clinical studies through its World's Longest Games. 

Forward-Looking Statements

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Michael Weickert Ph.D

Source: Pacylex

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