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10 Best Healthcare Companies to Watch 2020

Pioneering a new approach for the treatment of cancer: Pacylex Pharmaceuticals, Inc.

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Precision medicine is an approach in patient care which will allow the doctors to select treatments that will most likely help patients based on the genetic profiles of their diseases. This is also known as personalized medicine. For decades, if you were diagnosed with cancer, you would usually receive the same treatment as anyone else who had the same type and stage of cancer. Scientists now understand that many tumors in patients have genetic changes that cause the cancer to grow and spread, and certain mutations in patients predispose them to certain cancer types and make them more or less susceptible to specific treatments.

Finding a new genetic marker is an important cancer advance but even more exciting is finding a new biological mechanism by which certain cancers can develop and grow in patients. Pacylex Pharmaceuticals, Inc. is a company based in Canada that is exploiting a discovery made in the lab of Dr. Luc Berthiaume at the University of Alberta. He connected certain cancers with abnormalities in a biological process called myristoylation. After the founding of Pacylex in 2012, the company executed royalty and relationship agreements with the University of Alberta in 2013 to obtain intellectual property developed by there Dr. Berthiaume, one of the Pacylex founders. He tested a class of drugs known as NMT inhibitors on various leukemia and lymphoma cell lines and animal cancer models and discovered one compound in particular was very potent in eliminating leukemia and lymphoma cells and tumors in animal models. Pacylex licensed the compound and related molecules from the University of Dundee in 2015. Since then, Pacylex developed and scaled up the manufacturing process and conducted toxicology studies to determine the safety and potential starting dose for studies in patients. The company had a pre-IND meeting with the US FDA in 2019 to confirm the path for clinical development. Regulatory authorization will be sought in 2020 to allow clinical trials of this new medicine, PCLX-001, to begin in cancer patients. Pacylex was one of the first 4 companies accepted in 2018 into the new Merck Invention Accelerator in Edmonton, Alberta.

In conversation with Michael Weickert, CEO of Pacylex Pharmaceuticals, Inc.

Q. Please talk about your precision medicine solution in brief.

The precision medicine solution refers to the observation that epigenetic suppression of one of the two enzymes responsible for myristoylation, the process mentioned earlier, seems correlated with the emergence of many different types of cancer. The hypothesis is that this enzyme, NMT2, acts as a cancer suppressor, so when its expression is suppressed, cancer emerges. We have two different tests for NMT2 levels and can use either of these to identify patients who may benefit most from PCLX-001 therapy. But in blood cancers, the prevalence of NMT2 suppression is so high that almost every patient may benefit from PCLX-001 therapy, so a test may not be necessary.

Q. Cancer is just after heart disease as the second highest cause of death in the US. What will you do to change this?

One of the most important things about PCLX-001 is it actually kills cancer cells and eliminates tumors in animal models. Lots of existing drugs prevent tumors from growing but often, after a period of stable disease, cancer cells become resistant to therapy and resume growth and eventually kill the patient. PCLX-001 causes apoptosis, or programmed cell death, in cancer cells and tumors. The result is that tumors disappear in animal models. In an acute myeloid leukemia model, tumors disappeared after only five once-per-day oral doses of PCLX-001, and did not come back throughout the rest of the experiment. We would rather have therapies that cure patients than therapies that extend life by a few months, and that’s what we hope to deliver.

Q. Cancer still has a lot of mortality and many patients don’t survive long after their diagnosis despite therapeutic advances. What is different about your drug?

Our lead cancer drug, PCLX-001, hits a new target in cancer, myristoylation, and is a first-in-class therapy. In animal cancer models, treatment with PCLX-001 rapidly eliminates leukemia and lymphoma tumors and inhibits lung and breast cancer tumors. In a mouse lymphoma cancer model using cancer tissue from a patient whose lymphoma was resistant to all current therapies, PCLX-001 completely “regressed”or shrunk the tumors until they were undetectable. This is expected when you have a genuinely new target and first-in-class therapy – tumors that are resistant to other drugs that work through other mechanisms are susceptible to our drug – it still kills the cancer. Not only does it work through a different mechanism than other cancer drugs, but in tests using cultured cancer cells in vitro, PCLX-001 is at least 10-times more potent than Ibrutinib (Imbruvica) and Dasatinib (Sprycel), two multi-billion-dollar drugs for blood cancers. And even better, PCLX-001 is an oral drug, meaning patients would be able to take it at home as a once-a-day pill.

Q. Apart from Cancer, is your company planning to expand its studies to any other field? 

The biology of myristoylation is important to normal control of cell growth and metabolism, its dysregulation is implicated in other diseases besides cancer. We have begun investigating other indications for NMT inhibitors but we are not talking about the results yet.

Meet the leader of Pacylex Pharmaceuticals, Inc.

In 2017, Pacylex recruited Dr. Michael Weickert to join the company as CEO. Dr. Weickert has been developing drugs and startup companies in biotech and pharmaceuticals for over 27 years. He has been CEO of 4 other startups, Chief Business Officer at 3 companies, COO of Ohm Oncology, VP of development at SciDose and Auspex, and Senior Program Executive or Manager at Nektar and Ligand. Michael has driven oncology product development including during the Phase III/NDA for Targretin for Cutaneous T-cell Lymphoma, and business planning for drug delivery of the cancer drugs Leuprolide, Paclitaxel and Irinotecan. He obtained Orphan Drug and Fast Track designations for oncology-related products in US and EU. Prior to joining the pharmaceutical industry, he was at the National Cancer Institute at NIH. At startups, he raised Seed through Series C capital and established partnerships worth more than $180 Million.

“Our lead cancer drug eliminates leukemia and lymphoma tumors and inhibits lung and breast cancer tumors in many animal models including with drug resistant patient tumor tissue.”