Making A Real Difference In Cancer Care
Pacylex Pharmaceuticals is a clinical stage company developing a first-in-class, daily, oral cancer therapy with a novel, broad, mechanism of action, as a refractory cancer monotherapy and potential future combination therapy.
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Pacylex: Targeting myristoylation to selectively kill cancer.
Pacylex Pharmaceuticals is the world leader in the clinical development of N-myristoyltransferase (NMT; myristoylation) inhibition therapy for cancer with multiple Phase 2a programs underway. Myristoylation is a post-translational modification facilitating membrane association of proteins essential for many cancer processes such as pro-survival and proliferation signaling, energy production, receptor translocation and angiogenesis. By interrupting myristoylation, NMT inhibitors offer a potent alternative to classic chemotherapies and immunotherapies to kill cancer cells.
NMT inhibitors are also being developed as a new class of ADC payloads and Pacylex has exclusive license to a family of 503 small molecule NMTis, 27 of which have single nM IC50s against the critical enzyme, human NMT1. Pacylex is developing zelenirstat, a first-in-class myristoylation inhibitor, as an oral daily therapy for cancer. Zelenirstat has excellent bioavailability, half-life, safety profile including long-term, and drug exposure, and signs of activity in multiple types of solid tumor cancers and lymphoma. Zelenirstat is now in Phase 2a studies in heme cancers.
Pacylex is seeking ADC development partners and partners and investors for oral zelenirstat development which could be approved for AML for which Pacylex already has Orphan and Fast Track designations, as early as 2027.
NMT2 Loss
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.
zelenirstat
Our drug is an orally bio-available small molecule that is optimized for NMT inhibition. zelenirstat has promising attributes of a one pill per day dose strategy and has been shown to be active across many cancer types.
Sensitivity Biomarker
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.
Targeting Urgent Needs
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.
Progress for zelenirstat
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