US FDA approves Ellipses Pharma's IND application for EP0042 for treatment of acute myeloid leukaemia

Ellipses Pharma (Ellipses), a global drug development company focused on accelerating the development of cancer medicines and treatments through an innovative drug development model, announced that the US Food and Drug Administration (FDA) has approved EP0042, a dual FLT-3 and Aurora kinase inhibitor. EP0042 is being developed as a new potential treatment for AML patients who have developed resistance to FLT3 inhibitors. FLT3 mutations are found in about one-third of AML patients, and they are associated with a higher risk of relapse and a poor clinical outcome.

This approval will allow Ellipses to expand its ongoing first in human phase 1/2 clinical trial, which is currently undergoing its dose ranging phase. Once a recommended phase 2 dose is confirmed, Ellipses will continue to evaluate EP0042 as a monotherapy and explore EP0042 in combination with established standard treatments. Ellipses presented preliminary data from this ongoing study at the 64th Annual Meeting of the American Society of Hematology (ASH) in December 2022, demonstrating that EP0042 had acceptable safety and tolerability with evidence of prolonged disease control in a number of heavily pre-treated patients.

"This FDA approval of EP0042's Investigational New Drug application allows us to open additional trial sites in the world's foremost pharmaceutical market," said Dr Rajan Jethwa, chief executive officer and founder of Ellipses. This will assist us in meeting our strategic goal of bringing potentially new treatment options to patients in need at an unprecedented rate, while also allowing us to collaborate with key industry and academic partners. The data we presented in December at ASH, one of the leading conferences in our industry, demonstrated EP0042's potential, and as a team, we are now focused on doing everything we can to bring this potential to reality."

Professor Tobias Arkenau, global head of drug development and chief medical officer, Ellipses Pharma commented: “We are delighted to continue progressing through the regulatory pathway for EP0042, and this FDA IND approval is another successful milestone for our team. We will now work with our clinical partners to bring US sites online as quickly as possible as we look to continue this positive momentum in EP0042, and indeed across our broader portfolio.” EP0042's ongoing clinical-stage study follows earlier drug discovery and development work led by The Institute of Cancer Research, London, which was funded by organisations including The Institute of Cancer Research, Breast Cancer Now and Cancer Research UK.