Pharma Focus Europe

Molecure's Flagship Program, OATD-01, Granted FDA Approval for Phase Ii Clinical Testing Launch

Tuesday, July 25, 2023

Molecure S.A., a biotechnology company listed on the WSE, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) Application. This approval allows them to proceed with Phase II clinical testing of OATD-01, an innovative small molecule drug candidate. OATD-01 is the first chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential, aiming to treat several currently incurable diseases.

The Phase II study is scheduled to commence in the fourth quarter of 2023 and will focus on treating patients with pulmonary sarcoidosis. The CEO of Molecure, Marcin Szumowski, expressed great excitement about this development, as it represents a significant milestone in the advancement of their flagship project. The drug has shown promising results in preclinical trials, demonstrating its ability to modulate macrophage activity, which may lead to disease modification and treatment of various inflammatory and fibrotic diseases with similar molecular mechanisms.

In addition to the FDA clearance, Molecure is also planning to seek approval for Phase II clinical trials with the European Medicines Agency (EMA), intending to conduct studies in the European Union, including Poland. The company expects to conclude these Phase II clinical trials by mid-2025, with the publication of a comprehensive report analyzing the headline data.

OATD-01 has exhibited disease-modifying potential in preclinical trials and holds promise to become the new standard of care for pulmonary sarcoidosis treatment. It is an oral, once-daily, and highly selective CHIT1 inhibitor that has demonstrated significant anti-inflammatory and antifibrotic effects in various disease models. Beyond sarcoidosis, the drug shows potential for treating other inflammatory and fibrotic diseases with high unmet medical needs, such as idiopathic pulmonary fibrosis (IPF) and NASH.

The FDA has granted OATD-01 orphan drug designation (ODD) for its use in sarcoidosis and idiopathic pulmonary fibrosis, recognizing its potential to address critical medical needs in these areas.

Sarcoidosis is a systemic disease of unknown cause, characterized by the formation of immune granulomas in organs, particularly the lungs and lymphatic system. It affects individuals globally, with a higher prevalence among those aged between 25 and 45 years. One of the most severe complications of sarcoidosis is the development of pulmonary fibrosis, which significantly impairs pulmonary function and is a leading cause of death in sarcoidosis patients in western countries.

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