Ellipses’ next generation selective RET inhibitor EP0031/A400 granted Fast Track designation by US Food and Drug Administration

Ellipses Pharma (“Ellipses”), a global drug development company focused on accelerating the development of cancer treatments through an innovative drug development model, announced today that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its next generation selective RET inhibitor EP0031/A400 for the potential treatment of RET-fusion positive Non-Small Cell Lung Cancer (NSCLC).

The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need The purpose is to get important new drugs to the patient earlier[1].

Data from the ongoing modular interventional phase 1/2 trial, which aims to assess the safety, side effects and effectiveness of EP0031/A400 in patients with advanced RET-altered malignancies, were submitted in support of the application, where for the first time EP0031/A400 showed tumour responses in NSCLC patients who were previously treated with first-generation SRIs.  Responses appeared to be durable regardless of tumour type, RET fusion type, previous treatment or presence of brain metastases.

This announcement follows the FDA’s decision in November 2023 to grant Orphan Drug Designation to EP0031/A400.

Ellipses will now enter more frequent discussion with the FDA to advance rapidly towards an NDA for EP0031/A400 and earlier access by patients.

EP0031 is being developed jointly by Ellipses Pharma in a global clinical trial under the name EP0031 (NCT05443126) and Sichuan-Kelun Biotech in China under the names KL590586/A400 (NCT05265091).

Dr Rajan Jethwa, Chief Executive Officer, Ellipses, commented:

“We are committed to accelerating innovation and addressing the spectrum of unmet patient needs in cancer. With our world class drug development expertise, we are building on our heritage in oncology to bring a potential best-in-class treatment to people living with RET fusion-positive NSCLC.”

Professor Sir Christopher Evans, Chairman,  Ellipses Pharma, said:

“We are pleased and encouraged that the FDA has approved our company’s application for Fast Track Designation for EP0031/A400. This is an important step towards bringing this treatment to patients that confirms the potential for EP0031/A400 to address real unmet need in patients with NSCLC. We remain grateful to our partners at Sichuan-Kelun for their continued support.”

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

“Receiving Fast Track designation from the FDA reinforces our belief that EP0031/A400 has the potential to be a transformational treatment for patients with RET fusion-positive NSCLC, which remains a progressive and fatal condition for hundreds of thousands of people worldwide, despite available treatment options. This milestone has been reached rapidly and we look forward to working with the FDA to finalise the requirements for our submission for an NDA in due course.”

ENDS

About EP0031

In March 2021, Kelun-Biotech granted Ellipses an exclusive licence for EP0031 (A400) in certain territories including the US and Europe, with Kelun-Biotech retaining certain rights in Greater China. 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.

EP0031/A400 is a potent next generation SRI and in preclinical studies, 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.

Clinical data from both studies have shown promising response rates in RET-altered NSCLC, Thyroid and other solid tumours, who were either treatment naïve, or who had received prior therapies. Responses appeared to be durable regardless of tumour type, previous treatment or presence of brain metastases[2]. For the first time EP0031 showed tumour responses in NSCLC patients who were previously treated with first-generation SRIs.

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-Biotech（6990.HK）is a holding subsidiary of Kelun Pharmaceutical (002422.SZ), which focuses on the R&D, manufacturing, commercialisation and global collaboration of innovative drugs. The company focuses on major disease areas such as solid tumours, 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 tumours, 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/.

[1] U.S. Food and Drug Administration Fast Track. Available at https://www.fda.gov/ForPatients/Approvals/Fast/ucm405399.htm

[2] Zhou Q, ASCO 2023; Garralda E, ENA 2023