EP0042 receives Orphan Drug Designation from the US Food and Drug Administration

EP0042 is currently being investigated as a potential treatment option for patients with 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, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to EP0042, a dual FLT-3 and Aurora kinase inhibitor, for the treatment of acute myeloid leukaemia (AML).

The FDA grants ODD based on review of promising early clinical data from investigational treatments for rare diseases, such as AML, defined as affecting fewer than 200,000 people in the US. ODD qualifies the developer for certain incentives with the goal of accelerating drug development for patients, including tax credits and seven years of market exclusivity in the US upon approval by the FDA. 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

EP0042 is currently being investigated in an adaptive phase 1/2 dose ranging and optimisation study in patients with relapsed / 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 2 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

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.”

Professor Tobias Arkenau, Global Head of Drug Development and Chief Medical Officer, Ellipses Pharma commented:

“We believe that this FDA decision is a reflection of the strength of the early clinical data for EP0042, which was demonstrated in a presentation at ASH in December 2022. We are excited to continue the positive momentum in the clinical development of this drug, including progressing the ongoing phase 1/2 trial following the IND received earlier this year.”

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.

About EP0042

EP0042 is a dual FLT3 and Aurora kinase inhibitor under development as a new treatment for AML patients who have developed FLT3 inhibitor resistance. Dual inhibition of FLT3 and Aurora kinase has been shown to overcome acquired resistance to selective FLT3 inhibition both in vitro and in vivo

About acute myeloid leukaemia

Acute myeloid leukaemia (AML) is a cancer of the bone marrow, which begins to produce excess volumes of monocytes and granulocytes. It is one of the most common types of leukaemia in adults. Approximately 20,000 patients are diagnosed with AML in the US each year,⁴ and a further 18,000 patients in Europe, with around 40% of cases being diagnosed in people over the age of 75.⁵ The 5-year survival rate following an initial diagnosis is currently 15%.⁶

About Ellipses Pharma Limited

Ellipses Pharma is a global drug development company based in London, focused on accelerating the development of cancer treatments through an innovative drug development model that combines unbiased vetting to de-risk initial asset selection with an uninterrupted funding flow to minimise the time it takes to advance lead products through clinical trials and reach patients.

For more information, please visit ellipses.life

For more information contact us: media@ellipses.life


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)

3 Moore A et al. Leukemia 2012;26:1462–70; Tariq M et al. Br J Cancer 2021;125:966–74).

4 American Cancer Society. Key Statistics for Acute Myeloid Leukemia (AML). Accessed 24th October 2022. https://www.cancer.org/cancer/acute-myeloid-leukemia/about/key-statistics.html

5 Cancer Research UK. What is acute myeloid leukaemia (AML)? Accessed 24th October 2022. https://www.cancerresearchuk.org/about-cancer/acute-myeloid-leukaemia-aml/about-acute-myeloid-leukaemia

6 Cancer Research UK. Survival for acute myeloid leukaemia (AML). Accessed 24th October 2022. https://www.cancerresearchuk.org/about-cancer/acute-myeloid-leukaemia-aml/survival