CAMBRIDGE, Mass. — The U.S. Food and Drug Administration has granted Orphan Drug Designation to Zai Lab’s zocilurtatug pelitecan, an investigational antibody-drug conjugate being developed for neuroendocrine carcinomas.
The therapy, known as zoci and formerly called ZL-1310, targets Delta-like ligand 3, or DLL3, a protein frequently expressed in neuroendocrine cancers.
Neuroendocrine carcinomas are aggressive malignancies associated with poor outcomes and limited treatment options. Zai Lab said there are currently no approved targeted therapies or established standard of care for previously treated patients with these cancers.
“Zoci has now received important regulatory designations around the world, signifying its potential to become an important new therapeutic option for patients with multiple types of cancer with DLL3 expressions,” said Rafael G. Amado, M.D., President and Head of Global Research and Development at Zai Lab.
Amado said the company is focused on advancing its clinical programs for the investigational DLL3-targeting antibody-drug conjugate.
The FDA previously granted Fast Track designation to zoci for extrapulmonary neuroendocrine carcinomas. The therapy has also received Fast Track and Orphan Drug designations for small cell lung cancer, a pulmonary neuroendocrine carcinoma and one of the most aggressive solid tumors.
The European Medicines Agency has granted Orphan Drug Designation to zoci for pulmonary neuroendocrine carcinomas.
Orphan Drug Designation provides potential development incentives, including tax credits for certain clinical trials, a waiver of the Prescription Drug User Fee Act application fee and the possibility of seven years of U.S. market exclusivity following approval.
Zoci is designed to target DLL3, which is overexpressed in several neuroendocrine cancers, including small cell lung cancer and extrapulmonary neuroendocrine carcinomas. High DLL3 expression is generally associated with poor clinical outcomes.
Zai Lab plans to begin three registration-enabling studies by the end of 2026. The studies will evaluate zoci in second- and third-line small cell lung cancer, first-line small cell lung cancer and extrapulmonary neuroendocrine carcinomas.
The company said zoci has shown systemic and intracranial activity and could potentially become a treatment option for previously treated extensive-stage small cell lung cancer and extrapulmonary neuroendocrine carcinomas.
Zai Lab is also evaluating zoci as a potential component of first-line combination regimens, including combinations with checkpoint inhibitors and T-cell engagers.
