On May 18, 2023 Pacylex Pharmaceuticals, a clinical-stage company developing PCLX-001, a first-in-class oral investigational small molecule for leukemia, lymphoma, and solid tumor cancers, reported the first patient has been dosed in a Phase 2a expansion study in patients with relapsed/refractory B-cell Non-Hodgkin Lymphoma (R/R NHL) (Press release, Pacylex Pharmaceuticals, MAY 18, 2023, View Source [SID1234645057]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The Company’s Phase 1 multiple ascending dose (MAD) clinical trial evaluating PCLX-001 monotherapy in patients with R/R NHL or advanced solid tumors successfully completed six dose escalations without any dose-limiting toxicities (DLTs) and reached a drug exposure level expected to show clinical activity. Three patients with advanced solid tumors in the highest dose level are continuing to receive PCLX-001.

The Company received a No Objection Letter from Health Canada earlier this year to initiate the expansion cohort, designed to evaluate a potential Phase 2 dose in patients with R/R NHL. Four Canadian clinical sites have been activated and the first patient has been dosed. The expansion study will enroll up to 20 patients to assess the preliminary clinical activity of PCLX-001.

"The first stage of this first-in-human study assessed the safety of PCLX-001 with repeated dosing. We now transition our clinical investigations from primarily establishing safety to both safety and efficacy at a potential Phase 2 dose," said Dr. John Mackey, CMO of Pacylex.

"This milestone represents a major step in the development of PCLX-001 as a first-in-class oral cancer therapy" said Pacylex CEO Dr. Michael Weickert. "Our pre-clinical work showed that PCLX-001 at an equivalent dose in animals regressed R/R NHL tumors so it is exciting to see these patients finally receiving this as an investigational therapy" added Dr. Luc Berthiaume, Pacylex CSO and Professor at the University of Alberta.

Twenty-one patients have received PCLX-001 through 6 dose level escalations with no dose limiting toxicities, therefore dose escalation will continue in patients with advanced solid tumors in parallel with the Phase 2a study in patients with R/R NHL. Pacylex anticipates that it will present an update from the Phase 1 dose escalation portion of the study at a scientific conference this year.

About PCLX-001
PCLX-001 is a first-in-class, oral, small molecule N-myristoyltransferase (NMT) inhibitor being developed to treat patients with leukemia and lymphoma. PCLX-001 selectively kills cancer cells in vitro and have been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors in animal models. PCLX-001 appears to be particularly effective against Acute Myeloid Leukemia (AML) stem cells in animal disease models.
The Phase 1/2a study (NCT04836195) is an open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of PCLX-001 in patients with R/R NHL and advanced solid malignancies who have progressed on all available standard therapies.