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 28 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.
Luc is a professor of cell biology at University of Alberta with 29 years of experience studying protein fatty acylation. He received his PhD from the Université de Sherbrooke and completed his Post-Doctoral Fellowship at the Memorial Sloan-Kettering Cancer Centre. He has more than 40 publications and 3 patents published or pending. Luc is also the founder and president of Eusera, an antibody manufacturing company in Edmonton, AB, which produces and markets many commercially successful antibodies.
John is a medical oncologist and Director, Clinical Trials Unit at the Cross Cancer Institute, Professor of Oncology, University of Alberta, and Chair of the Alberta Pre-Phase I Cancer Program (www.prephaseone.com). As former Executive Director of Translational Research In Oncology (TRIO) for fourteen years, a global clinical trial company with its head office in Edmonton, he has experience with clinical trial operations to expedite the clinical evaluation of Pacylex diagnostics and therapeutics.
Ryan has a background in research, technology commercialization, business development, and financing early stage companies. Ryan has provided business planning and company development for many early-stage, technology-based ventures in health-related fields in his previous positions as the Director of the Merck Invention Accelerator, with the University of Alberta affiliated TEC Edmonton, and as an independent consultant. He also has nearly a decade of experience in the angel investing space as a Co-Founder, Operational partner and investor in Valhalla Private Capital.
Dr. Jacobs is currently the President and Chief Medical Officer at Achieve Life Sciences, Inc, a public company focused on smoking cessation and public health. Dr. Jacobs is also the CEO/Founder of Eagles Ridge Executive Consulting L.L.C. which advises on C-suite leadership and corporate strategic product development for early stage companies. In addition, she is on the board of Renown Pharma Inc, a private UK company focused on Parkinson’s Disease. Prior to the merger with Achieve Life Sciences, Dr. Jacobs was the Executive Vice President and Chief Medical Officer at OncoGenex Pharmaceuticals, Inc. Prior to Oncogenex, she was the Senior Vice President and Chief Medical Officer at Corixa Corporation until acquired by GlaxoSmithKline. She has also held vice president positions and been a member of executive management teams for two other Seattle-based biotechnology companies. She has extensive product development expertise in preclinical development, clinical operations, biostatistics, regulatory and product approval strategies, and has achieved several FDA and EU market approvals. Based on her preclinical work at Immunex, she is one of two inventors on the initial patent for Enbrel, as a rheumatoid arthritis treatment.
Mark R. Huson is a Professor of Finance and the Dianne and Irving Kipnes Chair in Finance and Development University of Alberta. Although he has been at the University of Alberta since 1993, Mark has also taught at the McCombs School of Business (UT Austin), and at the Richard Ivey School of Business (Western University). He has published papers in the Journal of Financial Economics, Journal of Finance, Journal of Corporate Finance, The Accounting Review, and The Journal of Operations Management. His work has been featured in the Wall Street Journal and Chief Executive magazine. His current research interests relate to the impact of intangible assets on firm policies and the markets’ assessment of accounting information. Mark is a member of the Independent Review Committee for Canadian Western Bank’s mutual funds. He has experience in Angel investing through his previous membership in the Central Texas Angel Network (CTAN) in Austin. In addition, he is actively involved in educating future entrepreneurs and investors in his role as the academic director overseeing Alberta School of Business’ participation in Creative Destruction Lab-Rockies.
Erwan Beauchamp is the Director of Discovery Biology. He is an accomplished protein fatty acylation expert with over 15 years of study and experience in the field. Erwan made the seminal discovery of the sensitivity of hematologic cancers towards PCLX-001 and is an inventor on 2 Pacylex Pharmaceuticals patents.
Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX Dr. Borthakur’s clinical and research work is focused on improving therapies for acute myelogenous leukemia (AML) and myelodysplastic syndrome (MDS). His primary commitment is to bring new agents/combinations to the clinic for improving outcome in patients with these diseases. While doing this, he has taken to the clinic ideas that have emerged out of the works of translational science leaders in the department of Leukemia. He has opened more than thirty investigator-initiated or industry-sponsored clinical trials in his area of interest and has an active translational research program on autophagy and epigenetic targets, for example BRD4.
Professor of Haemato-oncology , Institute of Cancer and Genomic Sciences Charles Craddock is Academic Director of the Centre for Clinical Haematology, Queen Elizabeth Hospital, Birmingham, UK, and Professor of Haemato-oncology, at the University of Birmingham. He trained in haematology at the Hammersmith Hospital, London, the Institute of Molecular Medicine at the University of Oxford and the University of Washington, Seattle, USA. Professor Craddock is a recent President of the British Society of Blood and Marrow Transplantation and Chair of the UK Stem Cell Strategic Oversight Committee. He is Director of the Centre for Clinical Hematology at the Queen Elizabeth Hospital and was Transitional Director of the £24 million Birmingham Institute of Translational Medicine which opened in 2015. He was Medical Director of the Anthony Nolan from 2010-2014. His main research interests include the development of novel drug and transplant therapies in myeloid leukaemias and he leads the Bloodwise UK Trials Acceleration Programme.
Associate Professor, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX Dr. Pemmaraju completed his Internal Medicine training at Johns Hopkins in the Osler Medical Program and subsequently completed both hematology and oncology training at MD Anderson. He obtained ABIM board certification in Internal Medicine, Hematology, and Oncology. His clinical/translational research work has been focused on improving outcomes and developing novel therapies for adolescents, young adults, and older adult patients with acute myeloid leukemia (AML), blastic plasmacytoid dendritic cell neoplasm (BPDCN), and myeloproliferative neoplasms (MPN). He has authored/co-authored 129 publications in the medical literature; 4 book chapters; and has presented at many national/international meetings (ASCO, ASH, EHA, AACR).
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