Inhaled GB0139 for IPF Named Orphan Drug in US and EU

Galecto‘s investigational compound GB0139, now in an enrolling clinical trial, has been designated an orphan drug by the U.S. Food and Drug Administration and the European Medicines Agency (EMA) as a potential treatment of idiopathic pulmonary fibrosis (IPF).

GB0139 (formerly TD139) is an inhaled small molecule inhibitor of galectin-3, a protein that plays a central role in the scarring (or fibrosis) of several organs, including the lungs. IPF patients have particularly high levels of galectin-3.

“We are very pleased that our promising GB0139 product candidate has received orphan drug designation in these two major markets,” Hans Schambye, CEO of Galecto, said in a press release.

Orphan drug status gives financial and other incentives to companies developing therapies for rare diseases. In  the EU, these include access to scientific advice, fee reductions, and 10 years of marketing exclusivity if approved.

In the U.S., orphan status confers largely the same advantages, but marketing exclusivity upon approval is seven years and fees associated with the Prescription Drug User Fee Act are also waived.

The EMA cited clinically relevant biomarker data reported in an IPF trial when awarding its designation.

GB0139 reduced in a dose-dependent manner the levels of YKL-40, a blood biomarker that is associated with fibrosis, inflammation and tissue remodeling in a completed Phase 2a trial (NCT02257177) in 24 patients, according to a poster presented at a science conference this year.

According to Galecto in its release, the currently approved treatments Esbriet (pirfenidone) and Ofev (nintedanib), whether alone or in combination, did not report changes in patients’ YKL-40 levels.

GB0139 was also reported to reduce other biomarkers relevant to IPF after 14 days of treatment.

“We are very encouraged that the EMA has cited our data, showing a higher effect on IPF biomarkers than the currently approved treatments, as a clinically relevant advantage,” Schambye said.

GB0139 is currently under clinical testing in the Phase 2b GALACTIC-1 trial (NCT03832946), taking place at sites in the U.S., the EU, the U.K., Israel, and Canada.

The trial is evaluating the efficacy and safety of one year’s use of two GB0139 doses — 3 mg or 10 mg once daily — against a placebo in 450 adults with IPF, ages 40 and older. Its main goal is changes from study start to week 52 in forced vital capacity (FVC), a measure of the total amount of air expelled in one forced breath.

Recruitment remains active, and more information can be found here. This study is expected to finish in December 2021.

“Our Phase 2b study in IPF is progressing well, and we look forward to bringing GB0139 further through clinical trials and potentially to market to address a significant unmet medical need,” Schambye said.