FDA Grants Orphan Drug Designation to ELC-100 for Pancreatic NETs

The FDA has granted orphan drug designation to ELC-100 for the treatment of patients with pancreatic neuroendocrine tumors (NETs).

In the fall of 2024, Elicera Therapeutics, the manufacturing company of ELC-100 (AdVince), announced that the final patient had been recruited for part 1 of the phase 1/2 RADNET trial (NCT02749331) evaluating the investigative agent. The trial will investigate the maximum tolerated dose and the safety of the treatment in this patient population. Specifically, the study will assess the safety of repeated infusions of ELC-100 into the hepatic artery in patients with metastatic NETs.2 ELC-100 is based on an oncolytic virus that works by destroying NETs.1 Final data from part 1 of the trial is expected to be reported in mid-2025.

“[NETs] represent a highly heterogeneous indication and in our ongoing clinical study with severely ill patients that can be divided into several subgroups, including based on treatment history,” Jamal El-Mosleh, chief executive officer of Elicera Therapeutics, stated in the news release. “This diversity highlights the need for new therapeutic solutions to be developed with a broad understanding of the specific needs of different patient groups.”

The open-label, uncontrolled, single-center phase 1/2 RADNET trial is estimated to include a minimum of 12 patients and a maximum of 35 patients with metastatic NETs.2 However, the number of patients in the study will be determined by the amount of toxicity observed. Eligible patients included those at least 18 years of age with histologically and radiologically confirmed progressive NETs of gastrointestinal, pancreatic, or bronchial origin with multiple liver metastases whose progression and tumor growth were determined via CT or MRI scans over the past 6 months. Patients also needed to have received a diagnosis of cancer that was deemed unresectable for potential cure or tumor reduction and have a life expectancy of at least 6 months.

The primary end point of the study is adverse effects, according to Common Terminology Criteria for Adverse Events (CTCAE) 4.03, which will be evaluated for an average of 18 months, from the screening visit until study completion. Secondary end points include changes in tumor size assessed by CT and/or PET scan with MRI based on RECIST or modified RECIST criteria measured within 4 weeks before the first treatment, after 80 days for evaluation visit 1, and after 214 days for evaluation visit 2; changes in tumor metabolic activity at baseline value within 24 hours before the first treatment and after 80 days for evaluation visit 1, and after 214 days for evaluation visit 2.

Other secondary end points from the study include progression-free survival at 12 weeks after 80 days from the first treatment or the corresponding time; and changes in the cytokine-mediated immune response measured by patients’ plasma at baseline and at 4 hours, 24 hours, and 72 hours after each treatment up to a period of 214 days.

“We are very pleased that ELC-100 has been granted orphan drug designation in the U.S. The decision by the FDA is a significant milestone in our efforts to develop a new form of treatment for patients with [NETs],” El-Mosleh concluded in the news release.

References

1. Elicera Therapeutics’ drug candidate ELC-100 received orphan drug designation in the U.S. for the treatment of pancreatic neuroendocrine tumors. News Release. Elicera Therapeutics. January 13, 2025. Accessed January 16, 2025. https://www.elicera.com/press-releases/elicera-therapeutics-drug-candidate-elc-100-receives-orphan-drug-designation-in-the-u-s-for-the-treatment-of-pancreatic-neuroendocrine-tumors
2. Study of recombinant adenovirus advince in patients with neuroendocrine tumors; safety and efficacy (RADNET). ClinicalTrials.gov. Updated December 3, 2024. Accessed January 16, 2025. https://clinicaltrials.gov/study/NCT02749331