Ellipses Pharma has received the Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its compound, EP0031/A400, to possibly treat tissue-agnostic RET fusion-positive solid tumours.

EP0031 is a potential next-generation selective RET inhibitor (SRI). It is said to have wide activity against general RET fusions and mutations, including recognised resistance mutations.

The US Food and Drug Administration (FDA) approved the Investigational New Drug (IND) for EP0031/A400 in June 2022.

Currently, a global, modular Phase 1/2 trial is being conducted on EP0031 to assess its safety, tolerability, and efficacy in patients with advanced RET-altered tumours, including those who have progressed on or are naïve to first-generation SRIs.

Ellipses CEO and founder Rajan Jethwa said: “Orphan Drug Designation from the FDA is an important moment in the ongoing development of EP0031 and we are currently accelerating its development based on the very encouraging clinical data we have shared at recent major conferences.

“This FDA decision further focuses our vision as we continue our drive towards bringing EP0031 to patients.”

It is estimated that approximately 2% of all solid tumours could be caused by RET mutations and rearrangements.

In preclinical tests, EP0031/A400 showed favourable inhibitory efficacy against important RET kinases both in vitro and in vivo.

It also demonstrated better blood-brain barrier penetration than first-generation SRIs.

EP0031 is being developed in collaboration with Kelun-Biotech Pharmaceutical.

The compound is also known as A400 when identified in connection with Kelun-Biotech’s ongoing regional development.

Ellipses Pharma chairman and founder Christopher Evans said: “Achievements such as this designation underline the vision and model we have developed at Ellipses – rapidly identifying and developing assets which we believe can have the most impact on patients around the world.”