Pacylex Pharmaceuticals Is Poised to Begin a Second Independent Clinical Trial for Its innovative Anti-Cancer Drug

Pacylex Pharmaceuticals plans to begin a second independent clinical trial of its anti-cancer drug, PCLX-001. The firm has recently been in the news for receiving FDA fast-track designation for PCLX-001 for treating relapsed or refractory acute myeloid leukemia (AML). Three weeks ago, the company announced that it had received Orphan Drug designation from the FDA and clearance of an IND (Investigational New Drug) application to start dosing AML patients.

Cancer is the leading cause of death worldwide, accounting for nearly 10 million deaths in 2020 alone. Every year, the United States spends more than USD 200 billion on cancer treatment. Treatment for cancer is not only time-consuming and expensive but also has a limited effect on mortality rates. Pacylex Pharmaceuticals seeks to change this reality through clinical trials and the commercialization of its pathbreaking drug, PCLX-001.

Cancer drugs are often very toxic. A key concern when it comes to the development of any new cancer drug is safety. The first stage of any clinical testing in human beings is the phase I dose escalation stage. Clinical development starts with low doses of the drug and ramps up the dosage gradually to find a level of the drug that is tolerated with few or manageable side effects while offering therapeutic benefits.

"We have gone through five of these stages for PCLX-001 after starting with very low doses. There have been no dose-limiting side effects. We have now achieved blood levels of drugs in patients that are well above the levels that kill cancer cells in culture and in animal cancer models. At least 40% of new drugs for new targets fail before this stage, and we are very excited that the trials have been successful so far." said John Mackey, Pacylex’s Chief Medical Officer.

PCLX-001 is the first of a new class of drugs called myristoylation inhibitors. This drug targets leukemia and lymphoma by inhibiting signaling processes that lead to the growth and spread of cancer cells. What makes this drug particularly promising is that it can completely eradicate these cells in animal models, whereas many other medications only slow down the growth of cancer. The drug can be taken orally once a day, while most other cancer drugs or cell-based therapies are administered intravenously and require hospital stays or infusion center appointments. 

Since PCLX-001 is a new drug, the primary concern was safety. It was first extensively tested on animals. The next step was to conduct clinical trials for safety after receiving clearance from regulatory authorities; Health Canada approved the first safety study. The company conducted a clinical trial that demonstrated the drug's safety in patients with a wide variety of cancer types, safely achieving doses predicted to have therapeutic benefits in blood cancers. The next step, clinical trials testing the impact of the drug in patients with AML and lymphomas, are scheduled to start at the beginning of next year. 

"Our next goal is to demonstrate that taking PCLX-001 causes leukemia and lymphoma to stop growing and even shrink. Our trials indicate that, from a pharmacokinetic perspective, the drug concentrations for each dose are much higher than we expected from animal models. Our first clinical targets are acute myeloid leukemia and diffuse large B-cell lymphoma. If first-line treatment fails, the next treatment has historically been very risky: stem cell or bone marrow transplantation.

While cell-based therapies like CAR-T are becoming more common, many leukemias and lymphomas do not respond to or return after treatment. This is why it is vital for us to complete clinical trials and commercialize PCLX-001, a simple, well-tolerated oral alternative. PCLX-001 is set to transform cancer treatment by targeting the root of cancer signaling and will hopefully save many lives in the future," said Pacylex CEO Michael Weickert.

About Pacylex Pharmaceuticals

Pacylex Pharmaceuticals is a pharmaceutical firm based in Edmonton, Canada, that has developed a new first-in-class, oral, once-per-day drug for the treatment of cancer. Pacylex began clinical studies of the drug in Canada in September 2021 and based on the success of those initial studies, is currently seeking funds for the expansion of clinical trials and the commercialization of the drug.

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