Molecure S.A. announced that it has received clearance from the U.S. Food and Drug Administration (FDA) on its Investigational New Drug (IND) Application to conduct Phase II clinical testing of OATD-01, the first-ever chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential. The first pulmonary sarcoidosis patients in this Phase II study are scheduled to begin receiving treatment in Fourth Quarter 2O23. OATD-01 has displayed disease modifying abilities in preclinical trials and has the potential to become the new standard of care for treating pulmonary sarcoidosis.

The phase II 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 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 end point ?

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-IND meeting.