Pacylex Pharmaceuticals announces progress on early phase clinical trials of PCLX-001 in patients

Pacylex Pharmaceuticals has dosed 16 patients in the early phase clinical trials of the company’s new cancer drug, PCLX-001. PCLX-001 is an N-myristoyltransferase(NMT) inhibitor that targets the lipid modification of proteins that allows proteins to move to membranes to initiate the signaling pathways for the proliferation and survival of cancer cells, especially in leukemia and lymphoma. The drug is a once-per-day capsule that has been dosed in 16 solid tumor and non-Hodgkin lymphoma patients to date without any dose limiting toxicities.

In September 2021, Pacylex Pharmaceuticals began a Phase 1 open label, dose escalation clinical trial of PCLX-001 at the University of Alberta Cross Cancer Institute to examine the safety and tolerability of the drug, and, determine the dose to be used in initial efficacy studies. In December 2021, Pacylex announced the results of the first patients dosed with PCLX-001 at the American Society of Hematology (ASH) Annual meeting and Exposition. An abstract of the clinical study was also published online in the November supplemental issue of ‘Blood’. This preliminary experience was published in ‘Current Oncology’ in March of 2022.

Pacylex has now safely completed 4 dose cohorts and exceeded the predicted target drug exposure levels in patients based on in vitro testing of PCLX-001 with leukemia and lymphoma patient cells and cell lines. Our clinical study confirms once-per-day oral administration of PCLX-001is a schedule suitable to achieve the predicted target drug exposure required for efficacy without toxicity. The company is treating patients at the 5th dose level and will continue to escalate doses as long as they are well tolerated by patients. The study is continuing to enroll patients at four Canadian cancer centers now, the University of Alberta Cross Cancer Institute, Princess Margaret Hospital in Toronto, Center Hospitalier de l'Université de Montréal (CHUM) and the BC Cancer Agency in Vancouver. The plan is to enroll 20-30 patients in the initial phase.

Pacylex Pharmaceuticals has presented its progress in the development of this new drug at professional conferences like the American Society of Hematology (ASH) Annual Meeting in December 2021 and the American Association for Cancer Research Annual Meeting in April 2022. The results of its non-clinical studies have been published in industry journals like ‘Nature Communications’, and ‘Breast Cancer Research and Treatment’.

What makes PCLX-001 different from other drugs used for the treatment of cancer is that it targets the origin of the signaling process inside cells immediately downstream the activated receptor complexes that provide the signals used by cancer cells to rapidly proliferate and survive. By inhibiting and stopping signaling initiation right at the beginning, oral administration of the drug induces apoptosis, or cell death, preferentially in cancer cells in just a few days. Also noteworthy, PCLX-001 preferentially kills cancer cells thereby sparing normal cells and minimizing side-effects.

Commenting on the company’s ambitious plans for the future, CEO of Pacylex, Dr. Michael Weickert said “Cancer has many different origins and causes and no silver bullet has been developed to defeat them all. But as new cancer targets are discovered, new strategies are developed to exploit them. Pacylex has exploited a new cancer target, myristoylation, with PCLX-001, a drug designed to inhibit myristoylation in cancer cells, which rely heavily on this process for survival and proliferation signaling. PCLX-001 eliminates leukemia and lymphoma tumors in animal studies. It also inhibits the growth of lung and breast cancer tumors in animal models. Our focus is now on proving the efficacy and safety of the drug through human clinical studies. We have already achieved target drug exposure in phase 1 clinical trials predicted to be sufficient to kill cancer cells. We anticipate further dosing at this level to reveal signs of clinical efficacy in the near future. Therefore, we are now seeking Series B investors to support two Phase 2programs in leukemia (AML) and lymphoma (DLBCL), and an expansion study in solid tumor patients. The end goal is to file for initial regulatory approval in at least one indication by 2025 and make this drug widely available to cancer patients worldwide at an affordable cost. The convenience of an oral, once-per-day pill also stands out in the current environment of immunotherapies and cell-based treatment strategies.”

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