A study by Ellipses Pharma, a global drug development company focused on accelerating the development of new oncology treatments, has presented preliminary data from the first in human Phase I/II trial of EP0042, a dual FLT-3 an Aurora kinase inhibitor, in patients with acute myeloid leukaemia (AML).
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.1
The preliminary data, from the ongoing dose ranging module of the trial, demonstrated that EP0042 had acceptable safety and tolerability with evidence of prolonged disease stabilisation in a number of heavily pre-treated patients.2
No dose-limiting toxicities were observed, and the emerging adverse event profile of EP0042 appears to be characterised by febrile neutropenia, fatigue diarrhoea, peripheral oedema, dizziness, and ataxia.
The preliminary data is based upon 25 patients across six dose cohorts including patients with FLT3 mutated and wild type AML at the point of enrolment. The median number of prior treatments was 2 (range 1-6), with a number of patients having received a prior FLT3 inhibitor.
Once a recommended Phase-II dose is confirmed, Ellipses intends to continue evaluating EP0042 as a monotherapy and explore EP0042 in combination with established standard treatments.
Dr David Taussig, Consultant Haematologist at The Royal Marsden NHS Foundation Trust and Chief Investigator, said: “I am excited to lead the first in-human clinical trial of EP0042, a drug which I hope will ultimately improve outcomes of patients with AML, for whom current treatment regimens are often ineffective. I look forward to building on this early clinical data alongside my colleagues, as we take this potentially important candidate further into the clinic.”
Dr Rajan Jethwa, CEO of Ellipses, said: “Ellipses’ unique approach to drug development allows us to accelerate drugs through the clinic and shorten the amount of time it takes for vital treatments to reach cancer patients. The preliminary data from EP0042 is a first step in potentially uncovering the promise that this compound holds and ultimately helping AML patients with limited treatment options.”