Molecure, a clinical stage biotechnology company, has filed an Investigational New Drug (IND) application with the U.S. FDA for OATD-01, a first-in-class Chitotriosidase (CHIT1) inhibitor that could become the new standard of care for sarcoidosis. The IND will allow Molecure to initiate a global Phase 2 study to evaluate OATD-01's safety and efficacy in approximately 90 patients with active pulmonary sarcoidosis. The primary endpoint is the response to a 12-week treatment with OATD-01 measured by the reduction of granulomatous inflammation in pulmonary parenchyma evaluated by PET/CT imaging. OATD-01 has received orphan drug designation from the FDA for sarcoidosis and idiopathic pulmonary fibrosis.
OATD-01 is an oral, once-daily CHIT1 inhibitor that has demonstrated potent anti-inflammatory and antifibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases such as sarcoidosis, idiopathic pulmonary fibrosis (IPF), and NASH. Molecure aims