Ellipses Pharma has obtained orphan drug designation from the US FDA for EP0031/A400 to treat tissue-agnostic RET fusion-positive solid tumours. Credit: Peddalanka Ramesh Babu / shutterstock.com.

Ellipses Pharma has secured orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for its next-generation selective RET inhibitor (SRI), EP0031/A400, to treat solid tumours.

The compound is currently being assessed as a treatment option for tissue-agnostic RET fusion-positive solid tumours.

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Approximately 2% of solid tumours are believed to be attributable to RET mutations and re-arrangements.

In March 2021, Kelun-Biotech Pharmaceutical granted an exclusive licence to Ellipses for EP0031/A400 in territories including Europe and the US, while retaining specific rights in Greater China.

EP0031, being developed collaboratively with Kelun-Biotech, is known as A400 when identified with the latter’s ongoing regional development.

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Ellipses Pharma chairman and founder Professor Christopher Evans stated: “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.”

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In preclinical trials, the compound showed favourable inhibitory activity against key RET kinases in in-vitro and in-vivo settings.

It also exhibited an enhanced ability to cross the blood-brain barrier compared to earlier-generation SRIs.

In June 2022, the FDA granted approval for the investigational new drug application (IND) of EP0031/A400.

Ellipses CEO and founder Dr Rajan Jethwa stated: “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.”

In June 2021, China’s National Medicinal Products Administration granted approval for the IND of EP0031/A400. A registrational Phase II trial is ongoing for this therapy in China.