DENVER, CO — Numiera Therapeutics Inc., a pharmaceutical company focusing on targeted cancer metabolism therapies, has been awarded a $2.3 million Direct-to-Phase II Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI). The funding will directly support clinical development and early-phase evaluation of the first-in-class CPT1 (Carnitine Palmitoyl Transferase) inhibitor, Etomoxir, for glioblastoma (GBM).
GBM remains one of the most aggressive and fatal cancers, with no new treatments improving survival since the leading chemotherapy, temozolomide. Affecting over 250,000 newly diagnosed patients worldwide each year, GBM patients have very limited options. Despite a significant amount of research dedicated to examining DNA damaging agents, immunotherapies, growth factor receptor inhibitors, and kinase inhibitors, no progress has been made over the past two decades to improve patient survival.
To address this urgent unmet need, Numiera Therapeutics is introducing its lead candidate drug, Etomoxir. Etomoxir is a brain-penetrant CPT1 inhibitor that blocks a key rate-limiting step in the fatty acid oxidation pathway and shows potential to combat GBM tumor growth with a novel mechanism of action. This drug was originally developed for the treatment of diabetes and congestive heart disease and is now being strategically repositioned under orphan drug designation to treat malignant brain tumors including GBM.
Building on prior Phase I data establishing preliminary safety parameters, the upcoming clinical study will run a dose-escalation and window-of-opportunity trial in patients with recurrent GBM. The trial is designed with three core objectives: identifying the maximum tolerated dose within a neuro-oncology population, evaluating the drug’s pharmacology within resected brain tumor tissue, and capturing early signals of clinical efficacy.
The project received recognition during the NIH peer review. The NIH Study Section Summary Statement noted: “The notable strengths of this project include the high significance of developing new targeted approaches to tackle deadly glioblastoma, the innovation of targeting fatty acid oxidation in this cancer, the outstanding team with strong record of accomplishments in drug development and glioblastoma, and the clinical trial outlined in excellence with appropriate statistical design.”
Dr. Izi Stoll, Co-Founder and CEO of Numiera Therapeutics, stated: “The recent FDA approvals and billion-dollar transactions involving Vorasidenib and Dordaviprone in neuro oncology indications have shown that mitochondrial-targeting small-molecule drugs are a complete game-changer in the field. With an even broader mitochondrial target, orphan designation for all malignant gliomas, independent validation in five different preclinical models from five different laboratories, and promising Phase I safety data for our lead drug candidate, we believe our approach will have a significant impact in this space.”
About The Company:
Numiera Therapeutics is dedicated to pioneering the development of new oncology drugs by targeting cancer cell metabolism. With an orphan-designated lead asset and a growing pipeline, its mission is to transform cancer patient care, by developing safer and more effective therapies especially designed to treat chemotherapy-resistant solid tumors. The Direct-To-Phase II SBIR Grant Award Number, issued to Numiera Therapeutics, Inc. by the National Cancer Institute, is 1R44CA310352-01.
