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.
Pacylex is developing zelenirstat, a first-in-class, once daily, oral cancer therapy with a novel, broad, mechanism of action. zelenirstat attacks the membrane-associated proteins at the root of cancer signaling in leukemia and lymphoma. By interrupting cancer signaling before it spreads to redundant pathways, zelenirstat offers a potent alternative to classic chemotherapies and immunotherapies to kill cancer cells. In an ongoing Phase 1 clinical trial, zelenirstat has excellent bioavailability, half-life, safety profile, and drug exposure consistent with advancing the program to Phase 2 clinical studies. Pacylex is seeking Series B investors to support two Phase 2 programs in leukemia (AML) and lymphoma (DLBCL) that can lead to registration filings by 2025, and an expansion study in solid tumor patients.
Our diagnostic technology identifies those patients who will benefit from our drug, sparing many patients needless treatment. We are able to do this through our discovery that a subset of patients across many cancer types have lost one of the two proteins required for myristoylation, N-myristoyl transferase 2 (NMT2). This protein loss presents a therapeutic opportunity for our drug candidate, zelenirstat.
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.
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.
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.
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.
Pacylex recently reported results at the Canadian Association of Radiation Oncology highlighting that zelenirstat was more effective than clinically used radiosensitizers
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.
The first patient has been dosed with PCLX-001 monotherapy, a first-in-class, oral small molecule N-myristoyltransferase (NMT) inhibitor, in a Phase 2a expansion study. The expansion study will enroll up to 20 patients.
A myristoylation inhibition gene expression signature, MISS-91 (Myristoylation Inhibition Sensitivity Signature), will be unveiled at the American Association for Cancer Research (AACR) Annual Meeting.