The US Food and Drug Administration (FDA) has given Molecure SA the green light to begin its phase 2 proof-of-concept study of OATD-01 to treat patients with pulmonary sarcoidosis.

Molecure will also seek approval from the European Medicine Agency to conduct phase 2 clinical testing of OATD-01 in the European Union. OATD-01 is the first-ever chitotriosidase 1 inhibitor with disease-modifying potential in sarcoidosis and other interstitial lung diseases, according to a release from Molecure.

The first pulmonary sarcoidosis patients in this phase 2 study are scheduled to begin receiving treatment in the fourth quarter of the year.

“We are extremely excited to share this great news, not only for us but for every patient looking for a better and more effective way of treating sarcoidosis,” says Marcin Szumowski, CEO of Molecure, in a release. “This marks the beginning of a new chapter in the development of our flagship project, as we enter human proof-of-concept studies and begin treating pulmonary sarcoidosis patients with OATD-01.”

OATD-01 has demonstrated potent anti-inflammatory and antifibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with high unmet medical needs such as sarcoidosis, as well as idiopathic pulmonary fibrosis and NASH.

The phase 2 clinical trial of OATD-01 is expected to be a multi-center, randomized, double-blind, placebo-controlled study assessing the drug’s safety and effectiveness in treating approximately 90 pulmonary sarcoidosis patients. As a result of the double-blind requirement, the study’s final unblinded results will be published after its conclusion, which is scheduled for the first half of 2025. 

The study has an innovative primary efficacy endpoint—the level to which OATD-01 is able to reduce granulomatous inflammation in the pulmonary parenchyma over a 12-week period based on PET/CT scan results. This endpoint was agreed with the FDA following a pre-Investigative New Drug meeting.

According to a release from Molecure, the company has received orphan drug designation from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis.