Ellipses' new RET inhibitor EP0031/A400 approved for Phase 2

Ellipses Pharma Limited, a global company dedicated to accelerating cancer treatment development, has announced that its advanced selective RET inhibitor (SRI), EP0031/A400, has been approved by the U.S. Food and Drug Administration (FDA) to proceed to Phase 2 clinical trials. This significant milestone came after a successful Phase 1 study, establishing the efficacy of EP0031/A400 in treating RET-altered tumors, including non-small cell lung cancer (NSCLC) and thyroid cancer. The drug has demonstrated durable responses, even in patients who were either naive to or pre-treated with first-generation SRIs and those with brain metastases.

EP0031/A400 is being developed through a collaborative effort between Ellipses and Sichuan-Kelun Biotech. The drug is undergoing a global Phase 1/2 trial under the name EP0031 (NCT05443126) and is being tested in China as KL590586/A400 (NCT05265091). Phase 1 trials have already been conducted across the US, Europe, and China, and a registrational Phase 2 trial is currently ongoing in China.

In addition to this progress, the FDA has granted EP0031/A400 Orphan Drug Designation in November 2023 and Fast Track Designation in March 2024. These designations are intended to expedite the development and review of drugs that address serious or life-threatening conditions and have the potential to meet unmet medical needs.

Ellipses Pharma's Chairman, Professor Sir Christopher Evans, expressed optimism about the progress, emphasizing the rapid generation of Phase 2 data as an essential step toward making this treatment available to patients. CEO Dr. Rajan Jethwa highlighted this milestone as crucial in advancing EP0031 towards a New Drug Application (NDA) for treating RET-altered NSCLC and other cancers. Furthermore, Professor Tobias Arkenau, Ellipses' Global Head of Drug Development and Chief Medical Officer, underscored the transformative potential of EP0031/A400 for patients with RET fusion-positive NSCLC, a condition with limited treatment options.

In March 2021, Ellipses obtained an exclusive license from Kelun-Biotech for EP0031 (A400) in specified territories, including the US and Europe, while Kelun-Biotech retained rights in Greater China. The Investigational New Drug Application (IND) for EP0031 (A400) was approved by China's National Medical Products Administration in June 2021, with Phase 2 trials underway in China. Additionally, in June 2022, the FDA approved the IND application for the EP0031 Phase 1/2 study in the US.

RET alterations, including mutations and rearrangements, are recognized as actionable drivers of oncogenesis in various tumor types, particularly prevalent in NSCLC and thyroid cancer. RET mutations and rearrangements are estimated to account for approximately 2% of all solid tumors. With the development of first-generation SRIs and a growing understanding of resistance mechanisms, there is a crucial need for next-generation SRIs to address acquired resistance in cancer treatment.

Ellipses Pharma Limited, headquartered in London, aims to accelerate cancer treatment development through a pioneering model that emphasizes unbiased vetting, minimal risk in asset selection, and continuous funding to expedite clinical trials. Kelun-Biotech, a subsidiary of Kelun Pharmaceutical, focuses on the research, development, manufacturing, and global collaboration of innovative drugs targeting major disease areas, including solid tumors and autoimmune diseases. The company currently has 33 ongoing innovative projects, including 14 in the clinical stage, with multiple global trials conducted in regions such as China, Europe, and the United States.