Privately-held Pacylex Pharmaceuticals is developing PCLX-001, a first-in-class, small molecule with a novel mechanism of action targeting a pathway believed to play a significant role in developing certain types of blood cancers and solid tumors. Data from preclinical models suggest that PCLX-001 has potential efficacy in leukemia and lymphoma, as well as in solid tumors such as breast cancer. A Phase 1 dose escalation safety and tolerability clinical trial is currently underway in patients with Non-Hodgkin’s Lymphoma (NHL) and solid tumors, with an additional study in patients with Acute Myeloid Leukemia (AML) planned for 2023. PCLX-001 has been granted FDA Fast Track designation and Orphan Drug designation in the U.S. for AML.
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Key Takeaways:
- Pacylex lead drug candidate, PCLX-001, is currently being evaluated in a Phase 1 clinical study in patients with an aggressive form of refractory lymphoma.
- By targeting cancers with increased dysregulated myristoylation, PCLX-001 can selectively kill cancer cells while sparing healthy cells.
- A second Phase 1 study in patients with relapsed or refractory acute myeloid leukemia will begin Q2 2023.
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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.
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.
Contacts:
Michael Weickert Ph.D
650-218-1840
michael.weickert@pacylex.com
Source: Pacylex
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