Inhaled GB0139 for IPF Named Orphan Drug in US and EU

Inhaled GB0139 for IPF Named Orphan Drug in US and EU
4.8
(24)

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. 

Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
Total Posts: 110
Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
×
Forest Ray received his PhD in systems biology from Columbia University, where he developed tools to match drug side effects to other diseases. He has since worked as a journalist and science writer, covering topics from rare diseases to the intersection between environmental science and social justice. He currently lives in Long Beach, California.
Latest Posts
  • training module
  • bilirubin levels, IPF
  • PF awareness month
  • GB0139 named orphan drug

How useful was this post?

Click on a star to rate it!

Average rating 4.8 / 5. Vote count: 24

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?