Ellipses EP0031 granted Orphan Drug Designation from the US Food and Drug Administration

EP0031/A400, a next generation selective RET inhibitor (SRI), is currently being investigated as a treatment option for patients with RET altered tumours

Ellipses Pharma (“Ellipses”), a global drug development company focused on accelerating the development of cancer treatments through an innovative drug development model, announces today that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to EP0031/A400 for the treatment of tissue-agnostic RET fusion-positive solid tumours.

The FDA grants ODD for investigational treatments for rare diseases, such as RET fusion-positive solid tumours, defined as affecting fewer than 200,000 people in the United States. 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 June 2022, the FDA approved EP0031/A400’s Investigational New Drug Application (IND), and EP0031/A400 is currently being investigated in a global, modular Phase 1/2 trial to evaluate the safety, tolerability and efficacy in patients with advanced RET-altered tumours including patients who are naïve to, or have progressed on, first generation SRIs.¹

EP0031 is being developed in partnership with Kelun-Biotech Pharmaceutical Co. Ltd. (Kelun-Biotech) and is also known as A400 when identified in conjunction with Kelun-Biotech’s ongoing regional development.

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

“Receiving an FDA Orphan Drug Designation for EP0031/A400 validates this compound’s potential in a currently underserved area of medicine. The designation is an important milestone in the development of EP0031/A400 and underscores the work we are already doing to accelerate its clinical development based on the encouraging clinical data we have shared at recent major congresses. We believe This FDA decision further focuses our vision as we continue our drive towards bringing EP0031/A400 to patients.”

Professor Sir Christopher Evans, Chairman and Founder of Ellipses Pharma, said:

“Achievements like this FDA Orphan Drug Designation for EP0031/A400 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:

“The FDA’s ODD approval is a significant milestone for EP0031/A400 and we are excited by the considerable momentum we have in the clinical development of this drug, including rapid progress with the ongoing phase 1/2 trial following the encouraging clinical data  presented at ASCO2 and the AACR-NCI-EORTC3 meeting.”

About EP0031

EP0031/A400 is a potent next generation SRI with broad activity against common RET fusions and mutations, including known resistance mutations. In preclinical studies, EP0031/A400 demonstrated favourable inhibitory activity against key RET kinases in-vitro and in-vivo with improved penetration of the blood brain barrier compared to first generation SRIs. An Investigational New Drug Application (IND) application for EP0031/A400 was approved by China’s National Medicinal Products Administration in June 2021 and a registrational Phase 2 trial is ongoing in China. In March 2021, Kelun-Biotech granted Ellipses an exclusive license for EP0031/A400 in certain territories including the US and Europe, with Kelun-Biotech retaining certain rights in Greater China.

About RET altered malignancies

Activating RET mutations and rearrangements have been identified as actionable drivers of oncogenesis in numerous tumour types and are most prevalent in non-small cell lung and thyroid cancer. It is estimated that RET mutations and rearrangements may be responsible for ~2% of all solid tumours. After the successful development of first generation SRIs and an increasing understanding of escape mechanisms to these agents, there is an unmet need to develop new treatments that can address acquired resistance, including the development of next-generation SRIs.

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.

About Kelun-Biotech

Kelun-Biotech6990.HKis a holding subsidiary of Kelun Pharmaceutical (002422.SZ), which focuses on the R&D, manufacturing, commercialization and global collaboration of innovative drugs. The company focuses on major disease areas such as solid tumors, autoimmune, inflammatory, and metabolic diseases,

At present, the company has 33 on-going innovative projects for medical needs in selected major disease areas such as solid tumors, autoimmune, inflammatory, and metabolic diseases, including 14 projects in the clinical stage and multiple global trials conducted in several regions such as China, Europe, and the United States. For more information, please visit https://kelun-biotech.com/.

For more information, please email media@ellipses.life 


1 https://clinicaltrials.gov/study/NCT05443126
2 J Clin Oncol 2023, abstract 3007
3 EORTC-NCI-AACR2023, abstract B043