Pacylex Pharmaceuticals Presents New Data on the Mechanism of Action of Myristoylation Inhibition

Pacylex Presents Recent Research Results at Two European Cancer Congresses This Month

Key Takeaways

  • 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
  • Effects of N-myristoyltransferase (NMT) inhibition in Acute Myeloid Leukemia stem cells will be presented at the European Hematology Association’s EHA2023 Hybrid Congress
  • Patients with lower overall survival in AML have a higher MISS-91 score, which is expected to predict patients most likely to respond to NMT inhibition

Edmonton, Alberta, Canada/June 13, 2023Pacylex Pharmaceuticals today announced recent research on N-myristoyltransferase inhibition (NMTi) will be presented at two upcoming European conferences, the European Hematology Association’s EHA2023 Hybrid Congress June 8-11 and the European Association for Cancer Research (EACR) June 12-15. These results expand the understanding of how NMT inhibition selectively kills cancer cells.

Further data on identifying cancer cells likely to respond to NMT inhibition therapy will also be included in the EACR presentation June 12-15 where details of a gene expression signature set, MISS-91 (Myristoylation Inhibitors Sensitivity Signature), will be described. MISS-91 predicts multiple cancers that are sensitive to PCLX-001, a first-in-class small molecule NMT inhibitor, including both hematologic and solid tumors, at concentrations at which normal tissues would be insensitive.

“Understanding the mechanism of action by which NMT inhibition selectively kills cancer cells is critically important,” said Luc Berthiaume, CSO of Pacylex. “Predicting the cancer types with MISS-91 that may respond best to PCLX-001 would enable it to be used where it can do the most for patients.” 

The European Hematology Association’s EHA2023 Hybrid Congress focuses on clinical research and practice and basic and translational research in the field of hematology and will take place in Frankfurt, Germany from June 8-11, 2023, continuing virtually through June 15, 2023. 

Details are as follows:

  • Title: N-Myristoylation Inhibition Abrogates Oxidative Phosphorylation to Target Acute Myeloid Leukemia Stem Cells 
  • Abstract number: P465
  • Presenting Author: Erwan Beauchamp, Ph.D., Pacylex Pharmaceuticals, Inc.
  • Session Data and Time: Friday, June 9 from 18:00 to 19:00 CEST
  • Full abstract information can be found here.

The 2023 Annual Congress of the European Association for Cancer Research (EACR 2023) covers basic, preclinical and translational cancer research across a wide breadth of topics, and takes place 12-15 June 2023, in Torino, Italy.

Details are as follows:

  • Title: Identification of a gene signature for cancers sensitive to N-Myristoylation inhibition
  • Abstract number: 1451
  • Presenting Author: Erwan Beauchamp, Ph.D., Pacylex Pharmaceuticals, Inc. 
  • Session: Posters: Experimental / Molecular Therapeutics, Pharmacogenomics
  • Session Data and Time: Wednesday, 14 June 2023 from 11:00 to 18:00 CEST
  • EACR 2023 abstracts will be published in an online supplement to the journal Molecular Oncology.

About PCLX-001
PCLX-001 is a first-in-class, oral, small molecule N-myristoyltransferase (NMT) inhibitor being developed to treat patients with leukemia and lymphoma. PCLX-001 selectively kills cancer cells in vitro and has been shown to regress hematologic malignancies and inhibit the growth of lung and breast cancer tumors in animal models. PCLX-001 appears to be particularly effective against Acute Myeloid Leukemia (AML) stem cells in animal disease models. 

The Phase 1/2a study (NCT04836195) is an open-label study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical activity of PCLX-001 in patients with R/R NHL and advanced solid malignancies who have progressed on all available standard therapies. 

About Pacylex Pharmaceuticals
Pacylex is a clinical-stage pharmaceutical company headquartered in Edmonton, Alberta, Canada, targeting hematologic and solid cancers with a first-in-class, oral, once daily investigational therapeutic, PCLX-001. Pacylex has a US Subsidiary in the San Francisco Bay area. PCLX-001 is the lead drug in a new class of NMT inhibitors, enabling Pacylex to exploit NMTs as new clinical targets for cancer treatment. Pacylex is conducting a multi-center Phase 1 multiple ascending dose study in Canada in patients with R/R NHL and advanced solid tumors, and a Phase 2a dose expansion in patients with R/R NHL.  The US Department of Defense is supporting the initial clinical investigation of PCLX-001 in patients with AML. The FDA has granted PCLX-001 both Orphan Drug Designation and Fast Track Designation for AML. Pacylex received support from an Alberta Innovates AICE grant in 2020, and the research leading to this breakthrough was supported in part by the Alberta Cancer Foundation and the Cure Cancer Foundation. 

For additional information, please visit www.pacylex.com.

Contacts
Pacylex Pharmaceuticals: Michael J. Weickert
CEO, Pacylex Pharmaceuticals, Inc.
P: 650-218-1840
Twitter @Pacylex (https://twitter.com/pacylex
#cancer, #lymphoma, #leukemia, #albertacancer, #Pacylex, #PCLX001, #UAlberta, #EACR2023,  #EHA2023, #MDACC,  #BIO2023


Key Takeaways

  • 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
  • Effects of N-myristoyltransferase (NMT) inhibition in Acute Myeloid Leukemia stem cells will be presented at the European Hematology Association’s EHA2023 Hybrid Congress
  • Patients with lower overall survival in AML have a higher MISS-91 score, which is expected to predict patients most likely to respond to NMT inhibition

Quotes

Predicting the cancer types with MISS-91 that may respond best to PCLX-001 would enable it to be used where it can do the most for patients.
Luc G. Berthiaume, PhDChief Scientific Officer

Related Bios

Michael J. Weickert, PhD
CEO
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.
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Luc G. Berthiaume, PhD
Chief Scientific Officer
Luc is a professor of cell biology at University of Alberta with 28 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.
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John Mackey, MD
Chief Medical Officer
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.
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Michele Libonati, MSc
Chief Operating Officer
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.
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Erwan Beauchamp, PhD
Director of Discovery Biology
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.
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Contacts

Michael Weickert Ph.D
michael.weickert@pacylex.com
650-218-1840
Chief Executive Officer