Arcus Biosciences, Inc. has announced that quemliclustat, an investigational small molecule CD73 inhibitor, has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of pancreatic cancer.
“The orphan drug designation indicates the importance of developing new treatment options for rare diseases like pancreatic cancer, which has the highest mortality rate of all major cancers, and which has seen few treatment advancements over the past 30 years,” said Richard Markus, chief medical officer at Arcus Biosciences.
“We expect the phase 3 PRISM-1 study to be fully enrolled this year and, if positive, intend to quickly bring this new first-line treatment option to patients, with the goal of prolonging survival for those with metastatic pancreatic cancer.”
Orphan drug designation is intended to support the development and evaluation of new treatments for rare diseases affecting fewer than 200,000 people in the US. Orphan drug designation qualifies sponsors for incentives including tax credits for qualified clinical trials, exemption from user fees including New Drug Application (NDA), and a potential seven years of market exclusivity after approval.
In January 2024, Arcus presented results from the phase 1 ARC-8 study, which showed no new safety signals and median overall survival (OS) of 15.7 months in a pooled analysis of all patients treated with the 100mg quemliclustat-based regimens. The median OS exceeded the historical benchmark data for chemotherapy alone, including for patients with liver metastasis, which account for more than half of all pancreatic cancers.
Based on the ARC-8 results, the company initiated PRISM-1, a registrational phase 3 study to evaluate quemliclustat plus gemcitabine/nab-paclitaxel chemotherapy versus gemcitabine/nab-paclitaxel chemotherapy alone in approximately 610 patients with pancreatic ductal adenocarcinoma (PDAC) that have not been previously treated in the metastatic setting. The study is expected to be fully enrolled this year.
Quemliclustat is an investigational molecule. Approval from any regulatory authority for its use has not been received, and its safety and efficacy have not been established.
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