Next-Gen RET Inhibitor EP0031/A400 Advances to Phase 2

London-based Ellipses Pharma Limited has announced that their next-generation selective RET inhibitor, known as EP0031/A400, has received the green light from the U.S. Food and Drug Administration (FDA) to advance to Phase 2 clinical trials. This drug is being developed as a potential therapy for tumors with RET alterations and has shown significant efficacy in patients with non-small cell lung cancer (NSCLC), thyroid cancer, and other solid tumors. Notably, sustained responses have been observed in both treatment-naive patients and those previously treated with first-generation selective RET inhibitors, including those with brain metastases.

The clinical data from Ellipses' Phase 1 study is set to be unveiled at the American Society of Clinical Oncology (ASCO) meeting in Chicago on June 3, 2024. The development of EP0031 is a collaborative effort between Ellipses and Sichuan-Kelun Biotech, with trials being conducted globally under different names but sharing the same clinical trial identification numbers.

In November 2023, the FDA designated EP0031/A400 as an Orphan Drug, and in March of the following year, it was granted Fast Track Designation. This designation is significant as it aids in the rapid development and review of drugs intended for serious or life-threatening conditions, particularly when there is a potential to fulfill unmet medical needs.

Ellipses' Chairman, Professor Sir Christopher Evans, expressed his optimism about the progress of EP0031/A400 and the anticipation of quickly generating Phase 2 data, which is a pivotal step in bringing this treatment to patients. Dr. Rajan Jethwa, CEO of Ellipses, highlighted the importance of this clinical milestone in the development of EP0031 as a next-generation treatment for RET-altered NSCLC and other cancers.

Professor Tobias Arkenau, the Global Head of Drug Development and Chief Medical Officer at Ellipses, emphasized the transformative potential of EP0031/A400 for patients with RET fusion-positive NSCLC, a condition that continues to be progressive and fatal for many despite existing treatment options.

In March 2021, Sichuan-Kelun Biotech granted Ellipses an exclusive license for EP0031 (A400) in the U.S., Europe, and other territories, while retaining certain rights in Greater China. The drug's Investigational New Drug Application was approved by China's National Medicinal Products Administration in June 2021, and a Phase 2 trial is currently underway in China. Additionally, the FDA approved the IND application for the EP0031 Phase 1/2 study in the U.S. in June 2022.

RET alterations, including mutations and rearrangements, have been identified as key drivers of tumor growth in various cancer types, particularly in NSCLC and thyroid cancer. It is estimated that approximately 2% of all solid tumors are associated with RET alterations. The development of next-generation selective RET inhibitors like EP0031/A400 is crucial due to the need to address resistance mechanisms that have emerged with the use of first-generation drugs.

Ellipses Pharma Limited is a global drug development company that employs an innovative model to expedite cancer treatment development. This model combines thorough initial asset selection with a continuous funding approach to reduce the time required to move lead products through clinical trials and make them available to patients.

Sichuan-Kelun Biotech, a subsidiary of Kelun Pharmaceutical, focuses on the research, development, manufacturing, and global collaboration of innovative drugs. The company is currently working on 33 innovative projects targeting major diseases such as solid tumors, autoimmune, inflammatory, and metabolic diseases, with 14 projects in clinical stages and ongoing trials in regions including China, Europe, and the United States.