Zelenirstat

Edmonton, Alberta--(News - 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. Click image above to view full announcement. 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 This press release contains forward-looking statements and forward-looking information within the meaning of applicable securities laws, such as statements relating to future events or the Company's future financial and operating performance, as well as the Company's business plans, growth initiatives, and objectives and prospects. Generally, forward-looking statements and forward-looking information may be identified by the use of forward-looking terminology, including the words "believe," "may," "will," "estimate," "continue," "anticipate," "intend," "should," "plan," "goal," "expect," "strategy," "future," "likely," "proposed," "scheduled," "forecast," "budget," "could," "would," variations of such words of phrases and other similar expressions, or by the use of words or phrases which state that certain actions, events or results may, could, would, or might occur or be achieved. However, the absence of these words does not mean that a statement is not forward looking. Forward-looking statements and forward-looking information are subject to numerous factors, risks and uncertainties that could cause actual results to differ materially, including, but not limited to, the Company's ability to successfully execute on its business plans and strategies, avoid delays in planned clinical trials, hire and retain key personnel, obtain appropriate or necessary governmental approvals to market potential products and obtain future funding for product development and working capital on commercially reasonable terms, changes in laws, and general macroeconomic conditions, including economic slowdowns, recessionary risks, rising inflation and interest rates, and supply chain disruptions. Forward-looking statements and forward-looking information are based on the beliefs of management as well as assumptions made by and information currently available to management as of the date hereof, and none of the Company or its affiliates undertakes any obligation to update or issue revisions to any forward-looking statements or forward-looking information contained herein to reflect any future events or circumstances, except as required by law. The foregoing does not constitute an offer or solicitation to acquire any securities in the Company or any related or associated entity or affiliate. The information contained herein is not intended as legal, tax, financial or investment advice. Furthermore, the information contained herein may not be applicable to or suitable for an individual's specific circumstances or needs. Contacts: Michael Weickert Ph.D 650-218-1840 michael.weickert@pacylex.com Source: Pacylex To view the source version of this press release, please visit

The presentation will be delivered by CEO Michael Weickert at the World Orphan Drug Congress USA 2024 which starts tomorrow (April 23). “The phase 1 results mark a significant milestone in our journey towards addressing critical unmet needs in cancer treatment,” says Dr. Michael Weickert, CEO of Pacylex Pharmaceuticals. “The data underscores the potential of zelenirstat as a promising therapeutic option for patients battling hematologic cancers and solid tumors.” Solid tumors and lymphomas The phase 1 study, focused on dose escalation safety and tolerability, involved 29 heavily pre-treated patients with solid tumors and lymphomas, each having a median history of four prior lines of drug therapy. According to the findings, treatment-related adverse events were observed in a minority of patients, primarily consisting of mild to moderate gastrointestinal side effects. The establishment of a recommended phase 2 dose (RP2D) for expansion studies was a significant outcome of the trial. “Zelenirstat demonstrated promising efficacy outcomes, particularly in solid tumor patients receiving the RP2D,” says Weickert. “This includes prolonged progression-free and overall survival rates, along with instances of prolonged Stable Disease, further highlighting its potential as a therapeutic breakthrough.” Acute Myeloid Leukemia (AML) cells Furthermore, preclinical studies, encompassing in vitro, ex vivo, and in vivo animal trials, have shown that NMT inhibition disrupts prosurvival signal initiation and oxidative phosphorylation in Acute Myeloid Leukemia (AML) cells, leading to complete regression of AML xenografts. These findings suggest a potential application of zelenirstat in AML treatment. googletag.cmd.push(function () { googletag.display('text-ad1'); }); “AML patients, especially those who have exhausted other treatment options, represent a population with significant unmet needs,” Weickert adds. “The promising preclinical evidence, coupled with the Orphan and Fast Track designations granted by the US FDA, positions zelenirstat as a compelling candidate for further clinical investigation in AML.” Investigational New Drug clearance In light of these findings, Pacylex Pharmaceuticals is advancing zelenirstat into a Phase 1/2 clinical study for AML patients, following the clearance of a US Investigational New Drug (IND) application by the US Food and Drink Administration (FDA). “Bringing zelenirstat to AML patients is a top priority for Pacylex Pharmaceuticals,” concludes Weickert. “The urgency stems from the high mortality rates associated with AML, particularly among patients who have exhausted other available therapies. We aim to provide a much-needed therapeutic option to this patient population.” The company says the presentation of phase 1 results for zelenirstat at the World Orphan Drug Congress USA 2024 represents a significant step forward in Pacylex Pharmaceuticals' mission to innovate cancer treatment and improve patient outcomes.