NuCana (NASDAQ:NCNA) received the FDA’s orphan drug designation for acelarin, a nucleoside analog of gemcitabine, for the treatment of biliary tract cancer.
“There is a high unmet need for patients suffering from this cancer type,” Hugh Griffith, NuCana’s founder and CEO, said in a statement.
“Our Phase Ib study of acelarin combined with cisplatin showed an approximate doubling of the response rate expected with the standard of care, gemcitabine plus cisplatin, with several patients achieving significant reductions in their tumor volume as well as further tumor shrinkage over time,” he added.
Acelarin is also being evaluated in a Phase 2 and Phase 3 study for patients with ovarian and pancreatic cancer, respectively.
NuCana plans to initiate a Phase 3 study of acelarin in combination with cisplatin as a front-line treatment for patients with biliary tract cancer later this year.