Wed, 29 Nov 2023

Edmonton, Alberta, Canada, November 22, 2022 Pacylex, a clinical-stage N-myristoyltransferase (NMT) inhibitor company developing first-in-class therapies for leukemia, lymphoma, and solid tumor cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted PCLX-001 Fast Track Designation for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML). Fast Track is a process to facilitate drug development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address unmet medical needs.

To view the full announcement, including downloadable images, bios, and more, click here.

Key Takeaways:

  • PCLX-001 is a first-in-class N-myristoyltransferase inhibitor which inhibits signaling necessary for cancer cell proliferation and survival in leukemia and lymphoma.
  • PCLX-001 is currently in clinical studies at 4 hospitals in Canada in non-Hodgkin Lymphoma and solid tumor patients.
  • The company also recently received FDA Orphan Drug Designation for PCLX-001 in AML and IND clearance for a Phase 1/2 study to start in the next quarter.

Click image above to view full announcement.

About Acute Myeloid Leukemia

In 2020, more than 474,500 new cases of leukemia were reported globally with more than 311,500 deaths (Source: 1). In 2017, AML accounted for 23.1% of total leukemia cases worldwide, and it is one of the most common types of leukemia in adults (Source: 2,3). In the U.S., an estimated 20,050 new cases of AML will be diagnosed in 2022 with the five-year survival rate reported at 30.5% (Source: 2,4).

The conventional treatment for newly diagnosed AML is intensive induction and consolidation chemotherapy with HSCT for eligible patients (Source: 5). The introduction of new targeted therapies in recent years has added to the standard of care and improved outcomes for some patients with molecularly defined AML subtypes (Source: 6). However, there remains a need to improve survival for the majority of patients with AML (Source: 5).


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 Welcome 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 are correlated with survival, suggesting an important biological role in these cancers.

About Pacylex

Pacylex is a pharmaceutical company headquartered in Edmonton, Alberta, Canada, targeting hematologic and solid cancers with a new first-in-class therapeutic, PCLX-001. 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 non-Hodgkin 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. The US Department of Defense is supporting the initial clinical investigation of PCLX-001 in AML patients.

For more information:Pacylex Pharmaceuticals Contact: Michael J. WeickertCEO, Pacylex Pharmaceuticals, Inc.E: michael.weickert@pacylex.comP: 650-218-1840Twitter @Pacylex ( LinkedIn ( ( page: ( #cancer, #lymphoma, #leukemia, #albertacancer, #Pacylex, #PCLX001, #UAlberta; #UAlberta_FoMD; #Worldslongestgame, #ASH22, #MDACC, #AML

References 1 Global Cancer Observatory. Population Fact Sheet: World. Updated March 2021. 2 American Cancer Society: Key Statistics for Acute Myeloid Leukemia. Updated January 2022. 3 Dong Y et al. Exp Hematol Oncol. 2020;9:14.

4 National Cancer Institute SEER Program. Cancer Stat Facts: Acute Myeloid Leukemia 5 Daver et al. Blood Cancer J. 2020;10(10):107.

6 Short et al. Cancer Discov. 2020;10:506-25.


Michael Weickert Ph.D


Source: Pacylex

Distributed by: Reportable, Inc.

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