FDA Grants Fast Track Designation For EP0031/A400 to Treat RET-Altered NSCLC


The next-generation SRI was previously granted an Orphan Drug Designation in November 2023 and its safety and efficacy are currently being evaluated in clinical trials.

About the Trials

1. Trial Name: A Study of EP0031 in Patients With Advanced RET-altered Malignancies

  • ClinicalTrials.gov ID: NCT05443126
  • Sponsor: Ellipses Pharma
  • Completion Date (Estimated): June 2027.

2. Trial Name: A Study of KL590586in Patients With Advanced Solid Tumors

  • ClinicalTrials.gov ID: NCT05265091
  • Sponsor: Sichuan Kelun Pharmaceutical Research Institute Co., Ltd.
  • Completion Date (Estimated): May 30, 2025.
X-ray of lungs -- Image credit: utah51 | stock.adobe.com

Image credit: utah51 | stock.adobe.com

The FDA has granted a Fast Track Designation for a next generation selective RET inhibitor, EP0031/A400, for the potential treatment of RET-fusion positive non–small cell lung cancer (NSCLC). Fast Track Designations help enable the development of the treatment while accelerating the review of medicines to treat serious conditions to meet a previously unmet medical need.1

EP0031/A400 is a potent next-generation selective RET inhibitor (SRI) that demonstrated favorable inhibitory activity against key RET kinases both in vitro and in vivo during preclinical trials. Compared with first generation SRIs, EP0031/A400 improved the penetration of the blood-brain barrier. Clinical data indicated positive response rates in RET-altered NSCLC, as well as other solid tumors in individuals who received prior therapies or are treatment naïve. Additionally, EP0031/A400 was granted an Orphan Drug Designation by the FDA in November 2023.1

"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," said Christopher Evans, chairman of Ellipses Pharma, in a press release.1

The Fast Track Designation application was based on data observed during the ongoing modular interventional phases 1 and 2 trial (NCT05443126), which evaluated the safety, adverse effects, and efficacy of EP0031. The trial includes dose escalation and dose expansion segments to investigate the optimal dose in adult patients who have advanced RET-altered malignancies. During the trial, the drug demonstrated tumor responses in patients with NSCLC who were previously treated with first-generation SRIs.1,2

Another trial (NCT05265091) in China is evaluating EP0031/A400 under the names KL590586/A400. Similarly, this trial is evaluating the safety, tolerability, efficacy, and pharmacokinetic properties of KL590586 in patients with advanced NSCLC and other solid tumors that have an RET fusion or mutation gene. The phase 1 section of the trial includes a dose escalation phase and a dose expansion phase, as necessary; phase 2 will assess the safety and efficacy of the drug in other cohorts at the recommended phase 2 dose. The primary outcome measures for this study are dose limiting toxicities in phase 1 (from date of initial dose until up of 28 days for treatment), and objective response rate in phase 2 (approximately 12 months).1-3

"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,” said Tobias Arkenau, MD, PhD, global head of drug development and chief medical officer, in the press release. “This milestone has been reached rapidly and we look forward to working with the FDA to finalize the requirements for our submission for an NDA in due course."

  1. Ellipses Pharma. Ellipses' next generation selective RET inhibitor EP0031/A400 granted Fast Track designation by US Food and Drug Administration. News release. March 5, 2024. Accessed March 5, 2024.
  2. A Study of EP0031 in Patients With Advanced RET-altered Malignancies.ClinicalTrials.gov identifier: NCT05443126. Updated February 15, 2024. Accessed March 5, 2024. https://clinicaltrials.gov/study/NCT05443126
  3. A Study of KL590586 in Patients With Advanced Solid Tumors.ClinicalTrials.gov identifier: NCT05265091. Last Updated October 13, 2022. Accessed March 5, 2024.
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