Elicera Therapeutics AB (publ) has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its drug candidate ELC-100, an oncolytic virus, for the treatment of pancreatic neuroendocrine tumors (NETs). The designation aims to facilitate the development of treatments for rare diseases, offering several benefits to Elicera during the drug's development and potential marketing approval.
Benefits of Orphan Drug Designation
Orphan Drug Designation is granted to drugs or biological products intended to treat diseases affecting fewer than 200,000 people in the United States. The FDA provides incentives to encourage the development of such treatments, including tax credits for clinical trials conducted in the U.S., exemption from certain application fees, and up to seven years of market exclusivity upon approval.
ELC-100: A Novel Oncolytic Virus Therapy
ELC-100 is based on a genetically modified adenovirus, Ad5PTD, optimized to selectively infect and replicate within NET cancer cells. The accumulation of virus particles leads to tumor cell lysis through immunogenic oncolysis, potentially activating the immune system. This process involves the release of neoantigens, which are then captured by dendritic cells, triggering a T-cell-mediated immune response against cancer cells.
Clinical Development and Trial Design
ELC-100 is currently being evaluated in an open-label, single-center Phase I/II clinical trial (NCT02749331) to assess its safety and determine the maximum tolerated dose (MTD) in patients with metastatic NETs. The trial involves repeated infusions of ELC-100 via the hepatic artery, with doses ranging from 10,000,000,000 to 1,000,000,000,000 virus particles.
The primary endpoint of the study is the incidence of adverse events (AEs) based on CTCAE v4.03 criteria. Secondary endpoints include changes in tumor size, changes in tumor metabolic activity, progression-free survival, changes in the replication profile of ELC-100, changes in the humoral response to ELC-100, and changes in the cytokine-mediated immune response.
Patient Eligibility and Enrollment
Eligible patients for the trial include individuals aged 18 to 100 years with histologically and radiologically confirmed progressive neuroendocrine carcinoma of gastrointestinal, pancreatic, or bronchial origin, harboring multiple liver metastases. Additional criteria include patent portal vein and adequate liver perfusion, a Karnofsky performance status of at least 70%, a life expectancy of at least 6 months, and functioning NET with somatostatin analog cover.
Management Perspective
"Neuroendocrine tumors represent a highly heterogeneous indication, and in our ongoing clinical study with severely ill patients, there are several subgroups, including those based on treatment history. This diversity highlights the need for new therapeutic solutions developed with a broad understanding of the specific needs of different patient groups," said Jamal El-Mosleh, CEO of Elicera Therapeutics. "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 neuroendocrine tumors."
The company expects to report final data from the first part of the study around mid-2025.
