Zelenirstat

Edmonton, Alberta, Canada October 14, 2024. Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company and 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 treatment of solid tumor cancers. Today, Pacylex announced that CEO, Michael Weickert will present progress in clinical development of oral zelenirstat, the first in class NMTi for heme cancers, and initial analysis of zelenirstat and other NMTis as ADC payloads, at the BIO Investor Forum, October 15-16, 2024, at the Fairmont Hotel, in San Francisco, CA, United States. Details for the presentation are below. Dr. Weickert will also be available during the conference for one-on-one meetings. Pacylex Pharmaceuticals has the only clinically validated NMTi, zelenirstat. A Phase 1 dose escalation safety and tolerability study in 29 heavily pre-treated (median of 4 prior lines of drug therapy) solid tumor and lymphoma patients showed treatment related, self-limited, mild to moderate gastrointestinal adverse events in a minority of patients with no does limiting toxicity observed below a daily dose of 280mg. Zelenirstat prolonged progression free and overall survival in 57% of Phase 1 solid tumor patients receiving the recommended Phase 2 dose (210mg), compared to those receiving lower doses. One colorectal patient and one DLBCL (Diffuse large B-cell lymphoma) patient have each received zelenirstat 210mg/day for a year or more with benefit, the DLBCL patient having achieved objective partial tumor volume and metabolic responses. ADCs are a major category of cancer therapeutics but in recent years, the need for new payloads has increased. Five attributes make zelenirstat an attractive payload candidate: 1) clinical validation of safety, 2) clinical validation of anti-cancer activity, 3) multiple mechanisms of anti-cancer activity that disrupt novel cancer signaling and survival pathways as well as metabolism, 4) activity in models of drug-resistant cancer, 5) limited acquired resistance despite long-term drug exposure. Pacylex has exclusive license to 503 NMTis, 27 of which have single-digit nM IC50s against human NMT1. “We look forward to accelerating the development of our extensive NMTi portfolio as ADC payloads with the participation of partners and investors” said Dr. Michael Weickert, CEO of Pacylex. “We will continue to develop oral zelenirstat in parallel for heme cancers and several other cancer and non-cancer indications.”

Edmonton, Alberta--(News - June 10, 2024) - Pacylex Pharmaceuticals Inc. (Pacylex) is a clinical-stage pharmaceutical company developing N-myristoyltransferase (NMT) inhibitors to treat hematologic cancers and solid tumors. Today Pacylex announced the publication of Phase 1 clinical trial results in the journal Investigational New Drugs. The report, titled: "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," describes effects of zelenirstat at various doses on cancer patients who exhausted all other therapeutic options. 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 with heavily pre-treated colorectal, appendiceal, and ovarian cancers had 6 to 16 months of stable disease when treated with the recommended Phase 2 dose. Zelenirstat inhibits myristoylation of proteins involved in several key cancer-driving processes explaining its broad spectrum of activity. 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 fully 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 reduced the bone marrow leukemic burden. 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. 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