Pacylex Reports Phase 1 Safety and Efficacy of Zelenirstat in Cancer Patients in Investigational New Drugs Journal

Edmonton, Alberta—Pacylex Pharmaceuticals, a clinical-stage pharmaceutical company, has announced promising results from its Phase 1 clinical trial of zelenirstat, an innovative N-myristoyltransferase (NMT) inhibitor developed for the treatment of hematologic cancers and solid tumors. These findings have been published in the journal Investigational New Drugs under the title: "A First-in-Human Phase I Trial of Daily Oral Zelenirstat, a N-myristoyltransferase Inhibitor, in Patients with Advanced Solid Tumors and Relapsed/Refractory B-cell Lymphomas."

Clinical Trial Findings

The Phase 1 trial primarily assessed the safety, tolerability, and pharmacokinetics of zelenirstat in patients with advanced solid tumors and relapsed or refractory B-cell lymphomas who had exhausted all other treatment options. Zelenirstat was found to be well tolerated, with most adverse events being gastrointestinal in nature and resolving without the need for dose adjustments. Moreover, patients with heavily pre-treated colorectal, appendiceal, and ovarian cancers showed stable disease for periods ranging from 6 to 16 months when treated with the recommended Phase 2 dose.

Mechanism and Efficacy

Zelenirstat inhibits the myristoylation of proteins that play critical roles in various cancer-driving processes, thereby offering a broad spectrum of activity against different types of cancer. This innovative mechanism of action enables zelenirstat to selectively target and kill cancer cells, as demonstrated in both in vitro studies and animal models. In acute myeloid leukemia (AML) models, zelenirstat notably killed leukemic stem cell populations and reduced the overall leukemic burden in bone marrow.

Ongoing and Future Studies

Following the successful completion of the Phase 1 study, which involved a multiple ascending dose safety and tolerability assessment (NCT04836195), the recommended Phase 2 dose for zelenirstat was established. In the Phase 1 study, zelenirstat exhibited a favorable safety profile, and its pharmacokinetics supported once-daily oral dosing. Furthermore, preliminary signs of efficacy were evident.

Currently, zelenirstat is being evaluated in a Phase 2a open-label study to further assess its safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity in patients with relapsed/refractory B-cell non-Hodgkin Lymphoma (NHL) and another cohort of patients with refractory metastatic colorectal cancer that has progressed despite standard therapies.

About Pacylex Pharmaceuticals

Pacylex Pharmaceuticals Inc., headquartered in Edmonton, Alberta, is at the forefront of developing oral NMT inhibitors to treat both hematologic and solid cancers. Zelenirstat is the lead drug in this new class of NMT inhibitors, positioning Pacylex to leverage NMTs as novel clinical targets for cancer therapy.

In addition to the ongoing Phase 2a studies in Canada for relapsed/refractory NHL and refractory metastatic colorectal cancer, Pacylex has also received clearance from the FDA for a Phase 1 multiple ascending dose study targeting AML. Significantly, zelenirstat has been granted both Orphan Drug Designation and Fast Track Designation by the FDA for the treatment of AML. The initial clinical investigation of zelenirstat in AML patients is supported by the US Department of Defense, with early clinical efforts also receiving backing from the Cure Cancer Foundation's World's Longest Games initiative.

As Pacylex continues to advance its clinical programs, the company remains committed to developing innovative treatments for patients with limited therapeutic options, holding promise for improved outcomes in various challenging cancer types.

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