Ellipses Pharma announces FDA approval of IND application for EP0042 for patients with acute myeloid leukaemia

Ellipses Pharma announces FDA approval of IND application for EP0042 for patients with acute myeloid leukaemia

IND approval allows for US trial sites and recruitment of eligible US patients to be added to EP0042’s ongoing phase 1/2 trial

Ellipses Pharma (“Ellipses”), a global drug development company focused on accelerating the development of cancer medicines and treatments through an innovative drug development model, today announces that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for EP0042, a dual FLT-3 and Aurora kinase inhibitor.

EP0042 is being developed as a new potential treatment to combat acquired resistance to FLT3 inhibitors in patients with AML. Around one third of patients with AML are diagnosed with FLT3-mutations, which are associated with a higher risk of relapse and poor clinical outcome.^[i]

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.

In December 2022, Ellipses presented preliminary data from this ongoing study at the 64th American Society of Hematology (ASH) Annual Meeting, which demonstrated that EP0042 had acceptable safety and tolerability with evidence of prolonged disease control in a number of heavily pre-treated patients.^[ii]

Dr Rajan Jethwa, Chief Executive Officer & Founder of Ellipses, commented:

“This FDA approval of EP0042’s Investigational New Drug application allows us to open additional trial sites in the world’s foremost pharmaceutical market. This will help us in achieving our strategic goal of bringing potential new treatment options to patients in need at unprecedented speed, whilst also allowing us to engage with key industry and academic partners. The data we presented in December at ASH, one of the leading conferences in our sector, demonstrated the potential of EP0042, and as a team we are now focused on doing all we can to progress this potential towards 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.

[i] Lam, S. and Leung, A. 2020. Overcoming Resistance to FLT3 Inhibitors in the Treatment of FLT3-Mutated AML. International Journal of Molecular Science, 21(4): 1537.

Gebru, M. and Wang, H-G. 2020. Therapeutic targeting of FLT3 and associated drug resistance in acute myeloid leukemia. Journal of Hematology & Oncology, 13, 155.

[ii] Abstract 2768 EP0042, a dual FLT3 and aurora kinase inhibitor: preliminary results of an ongoing phase 1/2a First in Human (FIH) study in patients with relapsed/refractory acute myeloid leukemia (AML)