Molecure S.A. announced that it has received national regulatory approvals from Denmark, France, Greece, Germany and Norway to conduct a Phase II clinical trial for OATD-01, a first-in-class chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential in pulmonary sarcoidosis. The Phase II clinical trial for OATD-01 is a randomized, double-blinded, placebo-controlled, multicenter study to evaluate the safety and efficacy of OATD-01 in approximately 100 patients with active pulmonary sarcoidosis, including patients previously receiving other therapies with no clinical improvement and patients previously untreated. Due to the requirement for double blinding in the study, the final unblinded results publication will occur after its completion and is scheduled for the end of 2025.

To measure efficacy of OATD-01 in the study, an innovative primary endpoint has been agreed upon with the regulatory authorities, which is the response to 12-week administration of OATD-01, measured by the degree of granulomatous inflammation reduction in the lung parenchyma, assessed by PET/CT imaging. Following the completion of full dosing along with a monitoring period involving approximately 50 patients, an interim analysis (intermediate checkpoint) is planned to evaluate the statistical results by an independent committee and make decisions regarding the study's continuation in terms of patient numbers in early First Quarter of 2025. The study will involve approximately 20-30 centers in the USA, European Union, Norway, and the United Kingdom.

The renowned Contract Research Organization (CRO) responsible for organizing and conducting the comprehensive study is Simbec Orion. OATD-01 is an oral, once-daily, first-in-class, and highly selective CHIT1 inhibitor for potential use in the treatment of sarcoidosis. The CHIT1 enzyme is a promising molecular target due to its role in transforming local anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types.

Blocking CHIT1 activity by OATD-01 has resulted in documented anti-inflammatory and anti-fibrotic effects. The OATD-01 molecule has shown strong anti-inflammatory and anti-fibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with unmet medical needs, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH), recently relabeled as Metabolic Dysfunction-Associated Steatohepatitis (MASH). Molecure has obtained orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis and has received approval to initiate a Phase II clinical trial for the treatment of pulmonary sarcoidosis in the US, UK, selected countries of the European Union, and Norway.

Sarcoidosis is a multi-organ disease of unknown etiology characterized by the formation of granulomatous structures in various organs, primarily in the lungs and lymphatic system. It is a globally occurring disease affecting both men and women with an estimated incidence of 5-50 cases per 100,000 population, with 70% of patients being between 25-45 years old. The most serious and common complication of sarcoidosis is pulmonary fibrosis, usually associated with significant impairment of lung function.

Pulmonary fibrosis is the cause of most sarcoidosis-related deaths in Western countries.