Edmonton, Alberta, Canada April 24, 2025. Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company and world leader in developing N-myristoyltransferase inhibitors (NMTis) for cancer. Oral zelenirstat is a proprietary, potent, NMTi under clinical investigation as a first-in-class targeted therapy for the treatment of hematologic cancers. Pacylex has 27 additional potent NMTis as potential payloads for antibody drug conjugates (ADCs) for the treatment of solid tumor cancers. Today, Pacylex announced that new research conducted at the University of Alberta, demonstrating that zelenirstat reduces complex I formation and oxidative phosphorylation in the mitochondria of triple-negative breast cancer (TNBC) cells, will be presented at the American Association for Cancer Research (AACR) Annual Meeting from April 25-30, 2025, in Chicago, Illinois, USA. Zelenirstat also reduced the growth of triple-negative breast cancer (TNBC) stem cells, with implications for reducing metastasis.
A poster will be presented describing evidence for profound disruption of mitochondrial complex I formation in TNBC cancer cells with concomitant loss of oxidative phosphorylation. The company’s CEO, Dr. Michael Weickert, will be available during the poster session and for one-on-one meetings. The details of the poster are below:
“We recently published evidence that zelenirstat disrupts growth signaling and energy production in the mitochondria of acute myeloid leukemia cells”, said Dr. Luc Berthiaume, CSO of Pacylex. “These new studies show zelenirstat similarly impacts aggressive breast cancer cells. Since mitochondrial oxidative phosphorylation is required for metastasis and cancer stem cell survival, the two major causes of death from cancer and cancer relapses, it has major implications for its potential use as a therapy in solid tumors.”
For more information:
Pacylex Pharmaceuticals Contact: Michael J. Weickert
CEO, Pacylex Pharmaceuticals, Inc.
P: 650-218-1840
About zelenirstat (aka PCLX-001 or DDD86481)
Zelenirstat (formerly identified as PCLX-001 or DDD86481) is a first-in-class, oral, small-molecule NMTi being developed to treat patients with leukemia, lymphoma, and for the treatment of solid tumors when used as a payload for ADCs. Zelenirstat selectively kills cancer cells in vitro and has been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors in animal models.
Pacylex completed and published a
Phase 1 multiple ascending dose safety, tolerability, and pharmacokinetics study on zelenirstat in patients with relapsed/refractory lymphoma and refractory solid tumors (
NCT04836195). Zelenirstat demonstrated an acceptable safety and tolerability profile, pharmacokinetics consistent with once-daily oral dosing, and early signs of efficacy. A clinical study in relapsed/refractory AML patients is now underway (ClinicalTrials.gov ID
NCT06613217).
About Pacylex Pharmaceuticals
Pacylex is the world leader in developing N-myristoyltransferase inhibitors (NMTis) as targeted therapies for the treatment of hematologic cancers and as payloads for antibody drug conjugates (ADCs) for solid tumors. Pacylex is conducting clinical studies of its lead drug, oral Zelenirstat, the first and only clinically validated NMTi. 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 AML patients. Pacylex is headquartered in Edmonton, Alberta, Canada, but also has an office in the San Francisco Bay area. The Cure Cancer Foundation, Alberta Cancer Foundation, and Alberta Innovates supported the initial clinical studies. For additional information, please visit
www.pacylex.com.
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