On September 14, 2021 Pacylex Pharmaceuticals, Inc. reported that it initiated a Phase 1 clinical trial of PCLX-001, a first-in-class, orally bioavailable small molecule inhibitor of N-myristoyltransferase 1 and 2 (NMT1, NMT2), at the University of Alberta Cross Cancer Institute (Press release, Pacylex Pharmaceuticals, SEP 14, 2021, View Source [SID1234645062]). This open label, dose escalation study will examine the safety and tolerability of PCLX-001 and determine the dose to be used in initial efficacy studies. Pacylex received a No Objection Letter from Health Canada on March 8, 2021, authorizing the planned Phase 1 Trial of PCLX-001 in relapsed/refractory B-cell Non-Hodgkin’s Lymphoma and advanced solid malignancies. PCLX-001 is believed to be the first NMT inhibitor that will be clinically tested.

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PCLX-001 is the first clinical candidate sponsored by Pacylex Pharmaceuticals to reach the clinical stage. "We are excited to see PCLX-001 transition to helping serious refractory cancer patients," said Michael Weickert, CEO of Pacylex Pharmaceuticals. "Our mission is to bring this new potential cancer breakthrough to patients, and we are especially privileged to be working with the Cross Cancer Institute on this clinical program."

"We are genuinely excited to initiate a new therapeutic candidate for these very challenging patients," said Dr. John Mackey, the Chief Medical Officer of Pacylex Pharmaceuticals. "Based on its unique mechanism of action and preclinical results, we hope this therapy will provide at least some patients with relief from their disease."

Dr. Randeep Sangha is the principal investigator for the study of PCLX-001 at the Cross Cancer Institute in Edmonton. "We plan to test PCLX-001 on patients who have tried everything else. Testing something really new is the goal of every cancer researcher since it may open the door for new treatments for patients" said Dr. Sangha.

Patient enrollment and dosing will also begin soon at Princess Margaret Hospital in Toronto and the BC Cancer Agency in Vancouver. The study will enroll 20-30 patients in the initial phase. Three principal investigators will oversee the clinical study at the three clinical sites in Canada: Dr. John Kuruvilla at Princess Margaret Cancer Centre in Toronto, Dr. Randeep Sangha at the Cross Cancer Institute in Edmonton, and Dr. Laurie Sehn at the British Columbia Cancer Center in Vancouver.

This study is registered at ClinicalTrials.gov Identifier: NCT04836195.

About Non-Hodgkin’s Lymphoma

Non-Hodgkin’s Lymphoma (NHL) is the most common hematologic malignancy and the eleventh most common cancer worldwide, with nearly 510,000 new cases diagnosed in 2018. It accounted for nearly 249,000 deaths worldwide in 2018. The most prevalent form of NHL, accounting for about 40% of newly diagnosed NHL cases, is an aggressive form called diffuse large B-cell lymphoma (DLBCL), that comes with a life expectancy of weeks or months if left untreated.

PCLX-001

PCLX-001 is a small molecule, first-in-class N-myristoyltransferase (NMT) inhibitor, originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness funded by Wellcome Trust. Pacylex is developing PCLX-001, which has excellent oral bioavailability, to treat leukemia and lymphoma. PCLX-001 selectively kills cancer cells and completely regresses (eliminates) tumors in animal models of acute myeloid leukemia (AML), diffuse large B-cell lymphoma (DLBCL) and Burkitt lymphoma (BL). PCLX-001 has also been shown to strongly inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the level of NMT2 is correlated with survival, suggesting an important biological role in these cancers. In tests using cultured cancer cells in vitro, PCLX-001 is at least ten times as potent as ibrutinib (Imbruvica) and dasatinib (Sprycel), two clinically approved drugs currently used to treat hematologic malignancies.