Galera Therapeutics’s pancreatic cancer program has gained an important boost after the US Food and Drug Administration (FDA) granted rucosopasem orphan drug designation.

Galera is testing rucosopasem in an ongoing, placebo-controlled Phase IIb trial (NCT04698915) in 220 patients with locally advanced pancreatic cancer. The study tests rucosopasem alongside stereotactic body radiation therapy (SBRT), a type of highly focused radiation beam treatment.

The Phase IIb trial of rucosopasem, dubbed GRECO-2, has a primary endpoint of median overall survival for up to three years. As a secondary endpoint, the study assesses median progression free survival over the same timeframe.

Rucosopasem is a small molecule selective dismutase mimetic in development for both pancreatic cancer and lung cancer. Galera’s lead candidate avasopasem is in late-stage development for severe oral mucositis and has a target FDA PDUFA date of August 9.

Galera and pancreatic cancer

The orphan drug designation will give Galera a much needed push as it targets a notoriously tough-to-treat cancer. According to GlobalData, the pancreatic cancer market in the US is expected to reach $1.66 billion in 2029.

Pancreatic cancer typically comes with a bleak prognosis and few treatments available, and many drug companies are hesitant to enter the space. In addition, the steep patient burden can stymie clinical trial enrolment.

The FDA grants Orphan Drug Designation to experimental therapies targeting diseases affecting fewer than 200,000 US patients, offering potential tax credits and up to seven years of market exclusivity. During the past decade, the FDA has doubled the number of new orphan drug designations, the majority of which have come in oncology.