Pacylex Pharmaceuticals Reports Safety and Efficacy Results from Its Phase 1 First-In-Human Study of Zelenirstat (PCLX-001)

On November 30, 2023 Pacylex Pharmaceuticals Inc. (Pacylex), a clinical-stage pharmaceutical company focused on the development of a new class of targeted therapies for the treatment of hematologic and solid tumor cancers, reported that the results of its Phase 1 dose escalation safety and tolerability study for zelenirstat, an investigational NMT inhibitor and Pacylex’s lead product candidate, will be included in investor updates presented at upcoming conferences in December and January (Press release, Pacylex Pharmaceuticals, NOV 30, 2023, View Source [SID1234645054]).

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The Phase 1 dose escalation safety and tolerability study was conducted in 29 lymphoma and solid tumor patients, who averaged 4 prior lines of therapy to which they were relapsed or refractor (R/R). The most common treatment related adverse events identified in the trial were mild to moderate gastrointestinal side effects which were self-limiting and occurred in a minority of patients. A recommended Phase 2 dose for expansion studies was established. Zelenirstat unexpectedly prolonged progression free survival in some Phase 1 solid tumor patients receiving the recommended Phase 2 dose; several patients at this dose continue zelenirstat treatment for up to ten 28-day cycles and counting. The Company has commenced a Phase 2a expansion study in patients with B-cell non-Hodgkin lymphoma.

Dr. Michael Weickert, Ph.D., Pacylex’s Chief Executive Officer, will present a corporate overview and discuss the Company’s Phase 1 study safety results and efficacy signals as well as development plans for this potential first-in-class oral therapy, at two upcoming investment conferences:

MedInvest Oncology Investor Conference on December 5-6, 2023 in Palo Alto, CA and present Phase 1 safety, tolerability and efficacy results for zelenirstat on Tuesday December 5, 2023 at 11:10 AM PT.
Pacylex will also participate in Biotech Showcase a premier investor and networking conference on January 8-10, 2024, alongside the JP Morgan Healthcare Conference. Dr. Weickert will present an update on the clinical progress of zelenirstat at the meeting in San Francisco, CA.
Dr. Weickert will also be available for one-on-one meetings at both conferences.

"The safety profile for zelenirstat is consistent with advancement to Phase 2 but the surprise in this study were the benefits observed in patients with heavily pre-treated solid tumors," said Dr. John Mackey, Chief Medical Officer for Pacylex and a practicing oncologist. "The fact that several patients receiving the Phase 2 dose have been treated with zelenirstat longer than any prior therapy, with reductions in disease burden, is very encouraging."

"We are delighted to attend these two premier investor conferences to showcase our Phase 1 study results, where zelenirstat demonstrated an acceptable safety and tolerability profile, favorable pharmacokinetics, and early efficacy signals," said Dr. Michael Weickert, CEO of Pacylex. "These better-than-expected Phase 1 results give us a sense of urgency to evaluate zelenirstat in a broader group of patients than initially planned."

Those interested in requesting a one-on-one meeting at the MedInvest Conference can submit a registration request using the conference website HERE. Presentation materials will be available under the News section of Pacylex’s website HERE.

Registered attendees of Biotech Showcase can request a one-on-one meeting with Dr. Weickert using the partneringONE platform HERE. If there are no compatible time slots available, please contact Dr. Weickert directly to schedule a meeting.

About zelenirstat (PCLX-001)

Zelenirstat (formerly identified as PCLX-001) is a first-in-class, oral, small molecule N-myristoyltransferase (NMT) inhibitor being developed to treat patients with leukemia, lymphoma, and solid tumors. In animal models, zelenirstat selectively killed cancer cells in vitro and has been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors. In AML models, zelenirstat 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 R/R lymphoma and solid tumor patients (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 potential 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 R/R B-cell non-Hodgkin Lymphoma (NHL). A separate Phase 2a cohort is planned in advanced solid malignancies who have progressed on all available standard therapies.