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Pacylex Presents Evidence Supporting Advancement of PCLX-001 for Treatment of AML at AACR 2022

PCLX-001 reduced leukemic stem cell burden in xenograft models
Key Takeaways
  • New data on PCLX-001 being presented at American Association for Cancer Research (AACR) Annual Meeting.
  • In vitro and animal model data shows the potential for N-myristoylation inhibition to treat for Acute Myeloid Leukemia (AML).
  • PCLX-001 dramatically reduces AML stem cells in the bone marrow.

Edmonton, Alberta, Canada, April 8, 2022 Pacylex today announced that data from an investigation of the potential for N-myristoylation inhibition to treat Acute Myeloid Leukemia (AML) will be presented at the American Association for Cancer Research (AACR) Annual Meeting from April 8-13, 2022. This investigation included the association of N-myristoyl transferase (NMT) expression levels with AML patient survival and the in vitro and in vivo demonstration of AML cell killing and tumor reduction with PCLX-001, a proprietary, potent, NMT inhibitor in clinical investigation in Non-Hodgkin’s Lymphoma (NHL) and solid tumor cancer patients. The poster details are below:

AACR Presentation Details: 

Title of poster: “Targeting N-myristoylation for Therapy of Adult Acute Myeloid Leukemia"

Session Category: Molecular/Cellular Biology and Genetics

Session Title: Cell Growth Signaling Pathways

Poster Number: 6391

Permanent abstract Number: 5662

Poster will be available in the e-poster website starting 1pm Friday April 8th, and will remain available through Wednesday July 13th.

PCLX-001 induced apoptosis in AML cell lines and patient blasts at concentrations that spared a large proportion of peripheral blood lymphocytes and monocytes from healthy individuals. PCLX-001 monotherapy had dose-dependent anticancer activity in an AML MV-4-11 cell line derived xenograft (CDX) and two AML patient derived xenografts (PDXs) and produced complete remissions in subcutaneous AML CDX. In tail-vein injected PDX models, PCLX-001 treatment resulted in up to 95% reduction of human CD45+ cells in peripheral blood and bone marrow.  

“We are building on our previous success demonstrating the efficacy of PCLX-001 in lymphoma, which has now advanced to clinical studies, and showing the same potential exists in leukemia,” said Luc Berthiaume, CSO of Pacylex. Pacylex plans to initiate a clinical investigation in AML patients later in 2022.

In the ongoing clinical study in NHL and solid tumor patients in Canada, patients have been enrolled at the University of Alberta Cross Cancer Institute, Princess Margaret Hospital in Toronto, Centre Hospitalier de l'Université de Montréal (CHUM) and are expected to be dosed at the BC Cancer Agency in Vancouver. The study will enroll 20-30 patients in the initial phase. Four principal investigators will oversee the clinical study at the four clinical sites in Canada: Dr. John Kuruvilla at Princess Margaret Cancer Centre in Toronto, Dr. Randeep Sangha at the Cross Cancer Institute in Edmonton, Dr. Laurie Sehn at the British Columbia Cancer Center in Vancouver, and Dr. Rahima Jamal at CHUM in Montreal. 

This study is registered at ClinicalTrials.gov Identifier: NCT04836195.


PCLX-001 (aka DDD86481) is a first-in-class, small molecule NMT inhibitor originally developed by the University of Dundee Drug Discovery Unit as part of a program to treat African sleeping sickness, funded by Wellcome Trust. Pacylex is developing PCLX-001 in the form of a once-a-day pill initially to treat leukemia and lymphoma. PCLX-001 has also been shown to inhibit the growth of lung and breast cancer tumors in animal models. In leukemia, lymphoma and breast cancer patients, the levels of NMT2 is correlated with survival, suggesting an important biological role in these cancers. In tests using cultured cancer cells in vitro, PCLX-001 is at least ten times as potent as ibrutinib (Imbruvica) and dasatinib (Sprycel), two clinically approved drugs currently used to treat hematologic malignancies. 

Pacylex is a pharmaceutical company targeting hematologic and solid cancers with a new first-in-class therapeutic, headquartered in Edmonton, Alberta, Canada. Pacylex’s technology combines new insights from Dr. Luc Berthiaume of the University of Alberta connecting myristoylation to cancer with a family of high quality myristoylation inhibitors Pacylex licensed from the University of Dundee. 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 initiated clinical studies in Canada in the fall of 2021 in diffuse large B-cell lymphoma and solid tumors. Pacylex is also receiving 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 more information:
Pacylex Pharmaceuticals Contact: Michael J. Weickert
CEO, Pacylex Pharmaceuticals, Inc.
E: michael.weickert@pacylex.com
P: 650-218-1840
Twitter @Pacylex (https://twitter.com/pacylex)
LinkedIn (www.linkedin.com/company/pacylex-pharma)
Facebook (https://www.facebook.com/pacylex)
#cancer, #lymphoma, #leukemia, #albertacancer, #Pacylex, #PCLX001, #UAlberta; #UAlberta_FoMD; #Worldslongestgame

Key Takeaways
  • New data on PCLX-001 being presented at American Association for Cancer Research (AACR) Annual Meeting.
  • In vitro and animal model data shows the potential for N-myristoylation inhibition to treat for Acute Myeloid Leukemia (AML).
  • PCLX-001 dramatically reduces AML stem cells in the bone marrow.
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“We are building on our previous success demonstrating the efficacy of PCLX-001 in lymphoma, which has now advanced to clinical studies, and showin...
Luc BerthiaumeCSO of Pacylex
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Michael J. Weickert
CEO
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Luc G. Berthiaume
Chief Scientific Officer
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Contacts
Michael Weickert Ph.D
michael.weickert@pacylex.com
650-218-1840
Chief Executive Officer