Pacylex: Targeting myristoylation to selectively kill cancer.
Pacylex Pharmaceuticals, Inc. is developing a precision medicine solution for multiple common cancers. Our approach exploits a previously-unknown therapeutic target in cancer, a cellular process termed myristoylation, which is an essential process allowing many signaling proteins to propagate signals across cell membranes. Our initial indications of interest are blood cancers including leukemia and lymphoma to be followed by numerous solid tumour types. Our drug, PCLX-001, is a first-in-kind therapy.
Our diagnostic technology identifies those patients who will benefit from our drug, sparing many patients needless treatment. We are able to do this through our discovery that a subset of patients across many cancer types have lost one of the two proteins required for myristoylation, N-myristoyl transferase 2 (NMT2). This protein loss presents a therapeutic opportunity for our drug candidate, PCLX-001.
NMT2 expression is lost in numerous cancers. The prevalence of the loss reaches 70- 82% in several leukemias and lymphomas. This NMT2 loss appears to promote tumorigenesis but also exposes an Achilles heel that allows new treatment strategies.
Our drug is an orally bio-available small molecule that is optimized for NMT inhibition. PCLX-001 has promising attributes of a one pill per day dose strategy and has been shown to be active across many cancer types.
We find 19 types of cancer have our sensitivity biomarker,at a prevalence of 5% to 80% of individual cancers. Sensitivity biomarkers are more common in blood and immune system cancers.
Despite major advances in cancer care, many patients don’t survive long after their diagnosis. Fewer than one third of patients diagnosed with lung cancer or acute myeloid leukemia survive for 5 years. 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. We are working now to bring this new medicine to human clinical studies.
Dr. Weickert has been developing drugs and startup companies in biotech and pharmaceuticals for over 27 years. He has been CEO of illumiSonics, Sonescence, and SEA Medical Systems, CBO of Strategent Life Sciences, Corium, and Therashock, 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 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 the 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.
Board changes and development partnership with DavosPharma also announcedDavosPharma will oversee the preparation of clinical supplies and regulatory clearances to advance PCLX-001 through development for Phase I clinical trialsEdmonton, Alberta, Canada, March 4, 2020 – PACYLEX PHARMACEUTICALS INC...
Pacylex Pharmaceuticals Michael Weickert sat down with Proactive at the BIO Investor Forum in San Francisco. Have a look! Click here to see the interview....
Pacylex will present at two upcoming investment conferences and the American Society of Hematology (ASH) MeetingOctober 7, 2019Pacylex is pleased to announce that it has been selected to present at two upcoming investment conferences this fall. The first is the BIO Investor Forum, held on October 22...
Dr. Luc Berthiaume’s team at the University of Alberta (U of A) showed that many human cancers lose a protein needed to keep growth and proliferation in check. These cells then become very sensitive to low dose NMT inhibitor treatments while normal tissues are unaffected. This discovery triggered the creation of a U of A spinoff company, Pacylex Pharmaceuticals Inc., to commercialize a diagnostic test to identify patients with NMT inhibitor sensitive cancers.
Independently, the University of Dundee Drug Discovery program developed NMT inhibitors to treat parasitic infections. Pacylex then in-licensed the rights to Dundee's panel of drug candidates - our lead compound is called PCLX-001. By combining Pacylex’s diagnostic test with the Dundee drug, we have a precision medicine approach to selectively treat patients with cancers sensitive to PCLX-001.
PCLX-001 eradicates established tumours in three lymphoma or leukemia cell line xenograft studies
PCLX-001 achieves tumour regression, comparable to standard-of-care treatment, in solid tumour xenograft studies
Our new synthetic pathway reduces the 13 step synthesis of PCLX-001 to 3 steps with high yield; this new proprietary medicinal chemistry is of broad applicability
Multiple common cancer types have the biomarker for drug sensitivity based on:
Our sensitivity biomarker test performs well in formalin-fixed paraffin-embedded clinical tumour samples, standard methodology in clinical pathology
PCLX-001 eradicates established drug-refractory aggressive lymphoma in a patient derived xenograft study