Pacylex is developing PCLX-001, a first-in-class, once daily, oral cancer therapy with a novel, broad, mechanism of action. PCLX-001 attacks the membrane-associated proteins at the root of cancer signaling in leukemia and lymphoma. By interrupting cancer signaling before it spreads to redundant pathways, PCLX-001 offers a potent alternative to classic chemotherapies and immunotherapies to kill cancer cells. In an ongoing Phase 1 clinical trial, PCLX-001 has excellent bioavailability, half-life, safety profile, and drug exposure consistent with advancing the program to Phase 2 clinical studies. Pacylex is seeking Series B investors to support two Phase 2 programs in leukemia (AML) and lymphoma (DLBCL) that can lead to registration filings by 2025, and an expansion study in solid tumor patients.
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 at public and private companies for over 30 years. He has been CEO of Fe Pharmaceuticals, illumiSonics, Sonescence, and SEA Medical Systems, CBO of Strategent Life Sciences, Corium, and Therashock, COO of Greenfire Bio and 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 versions 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. He has a PhD in Genetics from the University of Wisconsin, Madison.
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 50 publications and 4 patent families 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.
Michele brings more than 35 years of experience in strategy, preclinical research, clinical development and commercialization of small molecules, large molecules, drug device combinations and gene therapies. Most recently she was SVP of Program Strategy Leadership at Gilead Sciences, where she built a new function of cross-functional leaders accountable for overall program strategy. Prior to Gilead, Michele was CEO of Proneurotech, Inc. (now Nura Bio) a biopharmaceutical start-up focused on discovering and developing neuroprotective drugs. Michele spent 12 years at Genentech/Roche leading neurology, ophthalmology and immunology product strategy and global commercialization. In these roles, she oversaw the successful development and commercialization of a number of innovative flagship products, including OCREVUS®, LUCENTIS® and Rituxan®. Prior to joining Genentech, she held global clinical development leadership positions with Elan Pharmaceuticals, Inc. where she developed TYSABRI® and spent several years working in start-up companies Somatix Therapy Corporation developing gene and cell therapy applications for the treatment of Parkinson’s disease and Alzheimer’s disease, and Transcept Pharmaceuticals, heading the clinical development of Intermezzo®. Michele started the first ten years of her career working in neurology and psychiatric small molecule preclinical research and clinical development at Schering-Plough Research Institute. Michele has a B.A. in Experimental Psychology (now known as Neuroscience) from Rutgers University and M.S. in Pharmacology from New York Medical College.
John is a medical oncologist and Professor of Oncology, University of Alberta, and Chair of the Alberta Pre-Phase I Cancer Program (www.prephaseone.com). As the former Director of Clinical Trials at the Cross Cancer Institute, and the former Executive Director of Translational Research In Oncology (TRIO), 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.
Annette joined Pacylex as Vice President, Clinical Operations after more than 30 years in senior clinical operations and development roles, leading large-scale clinical programs in complicated and novel therapeutic areas. Prior to Pacylex, she served as Vice-President of Clinical Affairs at Neurogastrx, where she successfully progressed the NG101 program in Gastroparesis. Prior to Neurogastrx, she served as Vice-President of Clinical Operations at Aimmune, where she was a key figure in designing the clinical development program and implemented and managed the studies leading to the approval of the first-in-class peanut allergy treatment Palforzia™. At prior companies, she has been a key participant in the filings of several US and EU marketing applications and has held key leadership positions in programs that have directly contributed to successful product approvals and label expansions for multiple drugs and biologics. Over the past two decades, Annette has also provided consulting support to numerous bio-pharmaceutical companies to support their development programs, and in one case served as a client's regulatory representative to the FDA. Annette received her Bachelor's degree from Loyola-Marymount University.
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, TEC Edmonton, and as an independent consultant. He also has nearly a decade of experience in the angel investing space as a Co-Founder and investor in Valhalla Private Capital. He holds a Masters in Experimental Oncology and an MBA from the University of Alberta.
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. She is currently also on the Board of Directors for Achieve Life Sciences Inc and HiberCell Inc, and was formerly on the board of Renown Pharma Inc, a private UK company focused on Parkinson’s Disease. Previously, 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 was one of two inventors on the initial patent for Enbrel, as a rheumatoid arthritis treatment.
Nola Masterson, MSc. has more than 45 years of business experience in the life sciences industry and in venture capital investment. Masterson was the first biotech Analyst on Wall Street working for Drexel Burnham Lambert and Merrill Lynch. She has served as a Consultant at Kleiner Perkins Caufield & Byers and Oak Investments and IVP to create companies such as IDEC, Resound, and InSite Vision. She served as Venture Partner at TVM Capital GmbH and opened its San Francisco office. Ms. Masterson serves as Managing Director of Science Futures Management Company, LLC and was co-founder and first CEO of Sequenom, Inc. (SQNM). She is Chair Emeritus of California Life Science Institute and served as founder and General Partner of Science Futures LLC I and Science Futures LLC II. She served on the Board of EpiCept Corporation, Nanostream, Inc., Omicia, Inc. (now called Fabric Genomics), Repros Therapeutics Inc. (RPRX, Chairman) until its sale to Allergan, EmbraceHer Innovations, Inc., Resonance-Med, and Lynx Bio. She is an Adjunct Professor at University of California in the School of Management, and co-chair of the Northern California chapter of the Women Corporate Directors. Her work with Boardwise includes coaching and consulting with boards and individual board members. She has been honored as a pioneer by Fordham University. She is a published author and dynamic public speaker.
Ajit Gill is the founder and CEO of Greenfire Bio, LLC a drug development company, and also the founder and CEO of MGFB, LLC. Previously he was CEO and President of Nektar Therapeutics, when the company grew from a private startup to a public company with 800 employees and a market cap of over $2 billion USD. Earlier, Mr. Gill was VP and General Manager of Kodak’s Interactive Systems, VP of Finance at TRW-Fujitsu and Director of Business Development at VisiCorp. He graduated from IIT Kanpur with a B. Tech degree in Electrical Engineering and earned an M.S from University of Nebraska and an MBA from University of Western Ontario, Canada.
Mark R. Huson is a Professor of Finance and the Dianne and Irving Kipnes Chair in Finance and Development University of Alberta. He is also an adjunct Professor of Entrepreneurship at the Haskayne School of Business (University of Calgary) and the Academic Director at Creative Destruction Labs – Rockies. 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 experience in Angel investing through his previous membership in the Central Texas Angel Network (CTAN) in Austin.
Erwan Beauchamp is the Director of Discovery Biology. He is an accomplished protein fatty acylation expert with 18 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 Charles Craddock is Professor of Haemato-oncology, University of Birmingham. Professor Craddock is Chair of the UK Stem Cell Strategic Oversight Committee and was Medical Director of Anthony Nolan from 2010–2014. He is a past President of the British Society of Haematology. Professor Craddock’s main research interests include the development of novel drug and transplant therapies in acute myeloid leukemia. He pioneered the development of the UK Haemato-oncology Trials Acceleration Programme and the UK stem cell transplant trials network, IMPACT. He has published more than 250 papers in peer reviewed journals. Professor Craddock was awarded the CBE for services to medicine and medical research in the 2016 New Year’s Honours list and elected a Fellow of the Academy of Medical Sciences in 2020.
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).
For all clinical trial inquiries:
Thank you for your interest in the PCLX-001 phase I clinical trial. All study details including clinical sites and contact information including enrollment information can be found at www.clinicaltrials.gov.
Enrollment is open at the Cross Cancer Institute in Edmonton, the Princess Margaret Hospital in Toronto, Le Centre Hospitalier de l'Université de Montréal (CHUM), and the BC Cancer Agency. To inquire about enrolling, please ask your oncologist.
Merci pour votre intérêt au sujet de nos études cliniques de phase 1 à venir pour le PCLX-001. Les détails sur nos études cliniques et les informations requises pour l'enrôlement des patients eligibles se trouvent sur le site www.clinicaltrials.gov.
L'inscription est ouverte au Cross Cancer Institute d'Edmonton, à l'Hôpital Princess Margaret de Toronto, Le Centre Hospitalier de l'Université de Montréal (CHUM), et à BC Cancer Agency. Pour vous renseigner sur l'inscription, veuillez demander à votre oncologue.
A myristoylation inhibition gene expression signature, MISS-91 (Myristoylation Inhibitors Sensitivity Signature), will be presented at the European Association for Cancer Research (EACR) 2023 Congress.Read More
The first patient has been dosed with PCLX-001 monotherapy, a first-in-class, oral small molecule N-myristoyltransferase (NMT) inhibitor, in a Phase 2a expansion study. The expansion study will enroll up to 20 patients.Read More
A myristoylation inhibition gene expression signature, MISS-91 (Myristoylation Inhibition Sensitivity Signature), will be unveiled at the American Association for Cancer Research (AACR) Annual Meeting.Read More
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