EP0042 receives Orphan Drug Designation from the US Food and Drug Administration
pharmafile | March 14, 2023 | News story | Business Services |
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.
