Edmonton, Alberta, Canada April 22, 2024. Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company focused on the development of a new class of targeted therapies, N-myristoyltransferase inhibitors (NMTi) for the treatment of hematologic cancers and solid tumors. Today Pacylex announced that CEO Michael Weickert will present Phase 1 safety and efficacy results for zelenirstat and evidence supporting advancing it into clinical development in the Orphan Drug indication of Acute Myeloid Leukemia (AML) at the World Orphan Drug Congress USA 2024, Apr 23-25, 2024, at the Boston Convention and Exhibition Center, Boston, MA, United States.
Details for the presentation are below. The Company’s CEO, Dr. Michael Weickert, will also be available during the conference for one-on-one meetings.
The Phase 1 dose escalation safety and tolerability study was conducted in 29 heavily pre-treated (median of 4 prior lines of drug therapy) solid tumor and lymphoma patients. Treatment related adverse events were observed in a minority of patients, and were self-limited mild to moderate gastrointestinal side effects. A recommended Phase 2 dose (RP2D) for expansion studies was established. Zelenirstat prolonged progression free and overall survival in Phase 1 solid tumor patients receiving the RP2D, compared to those receiving lower doses. Prolonged Stable Disease of 6 months or longer was observed in 57% (4/7) of the solid tumor patients receiving RP2D, including a patient with metastatic colorectal cancer who continues on treatment for more than 14 months with ongoing reductions of approximately 50% in CEA (carcinoembryonic antigen) and tumor volumes.
Preclinical studies including in vitro, ex vivo, and in vivo animal studies indicate NMT inhibition disrupts prosurvival signal initiation and oxidative phosphorylation in AML cells and completely regresses AML xenografts. Additional in vivo evidence indicates AML stem cells are even more sensitive to zelenirstat than blasts. This strongly suggests that AML patients are excellent candidates for zelenirstat, a once per day oral investigational therapy. Orphan and Fast Track designations have been granted for AML by the US FDA, and a US IND has cleared the FDA for an AML Phase 1/2 clinical study.
"Advancing zelenirstat into a second hematologic indication, AML, is a top priority for the company”, said Dr. Michael Weickert, CEO of Pacylex. “AML is among the cancers most likely to respond to NMT inhibition. The high mortality of patients who have failed other available therapies makes it urgent for us to bring zelenirstat to people with AML."
For additional information, please visit www.pacylex.com.
Pacylex Pharmaceuticals Contact: Michael J. Weickert
CEO, Pacylex Pharmaceuticals, Inc.
E: michael.weickert@pacylex.com
P: 650-218-1840
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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 allowed the regeneration and growth of normal bone marrow cells.
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.
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