Potential treatment for patients with acute myeloid leukaemia 


Ellipses Pharma, a global drug development company focused on accelerating the development of cancer medicines and treatments through an innovative drug development model, has been granted Orphan Drug Designation (ODD) by the FDA for EP0042, a dual FLT-3 and Aurora kinase inhibitor, for the treatment of acute myeloid leukaemia (AML).  

In February 2023, the FDA approved EP0042’s Investigational New Drug Application, which allowed for the opening of additional trial sites in the US for this compound.  

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

Trial design

EP0042 is currently being investigated in an adaptive Phase I/II dose ranging and optimisation study in patients with relapsed or refractory acute myeloid leukaemia, and Ellipses plans to further evaluate it as both a potential monotherapy and in combination with standard treatments once a Phase II dose is confirmed.  

Preliminary data from this study was presented at the 64th American Society of Hematology (ASH) Annual Meeting in December 2022, which demonstrated acceptable safety and tolerability for EP0042, and evidence of prolonged disease control in a number of heavily pre-treated AML patients.2

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. 

Official comments

Dr Rajan Jethwa, Chief Executive Officer & co-Founder of Ellipses, commented: “Receiving an FDA Orphan Drug Designation for EP0042 validates this compound’s potential in a currently underserved area of medicine. The designation is an important milestone in the development of EP0042, and underscores the work we are already undertaking towards accelerating its potential access to patients. We believe its early clinical data merits its continued study, and this FDA decision further focuses our vision as we continue our drive towards bringing EP0042 to more patients.”  

Professor Sir Christopher Evans, Chairman and Founder of Ellipses Pharma, said: “Achievements like this FDA Orphan Drug Designation for EP0042 are why Rajan and I made the decision to found Ellipses Pharma, and underline the validity of our model of rapidly identifying and developing assets which we believe can have the most impact on patients around the world. I look forward to supporting the team as we continue to drive this compound through the clinic and as we continue our search for assets of promise.”  


  1. 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.  
  2. 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) 

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