Molecure S.A. is a biotechnology company specializing in the development of innovative drugs for treating various incurable diseases. The company focuses on leveraging its expertise in medicinal chemistry and biology to create unique small-molecule drugs targeting protein activity and mRNA translation. Recently, Molecure initiated a Phase II clinical trial for OATD-01, an oral CHIT1 inhibitor, for patients with active pulmonary sarcoidosis.

This significant trial began with the first administration of OATD-01 or a placebo to patients at the Royal Infirmary in Edinburgh. Patients will receive a daily dose of 25 mg OATD-01 or placebo tablets for 12 weeks, with regular safety monitoring through laboratory tests, neurological examinations, and ECG and spirometry.

The Phase II trial, designed as a randomised, double-blind, placebo-controlled, multi-center study, aims to assess the safety and efficacy of OATD-01 in approximately 100 patients with active pulmonary sarcoidosis. It involves 20-30 centers across the US, European Union, Norway, and the UK, with CRO Simbec Orion managing the study.

Dr. Marcin Szumowski, CEO and Chairman of Molecure S.A., highlighted the trial's significance in offering new treatment options for patients with incurable diseases. He emphasized Molecure's leadership in developing therapies based on chitinase activity inhibition, with this trial serving as a critical validation of the approach.

The primary endpoint of the study, agreed with the FDA, is the response to 12-week administration of OATD-01 measured by the reduction in granulomatous inflammation in the lung parenchyma, assessed through PET/CT imaging. An intermediate analysis will occur after approximately 50 patients have completed the trial, guiding further study protocols.

Dr. Samson Fung, Chief Medical Officer and Board Member of Molecure S.A., underscored the promising potential of OATD-01 shown in preclinical and phase 1 studies. The therapy aims to arrest or reverse disease progression, improve lung function, and enhance patients' quality of life.

OATD-01, an orally administered, first-in-class CHIT1 inhibitor, holds promise for treating sarcoidosis and other inflammatory and fibrotic diseases such as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH). Molecure has secured orphan drug designation (ODD) from the FDA for OATD-01 in sarcoidosis and IPF indications.

Sarcoidosis, characterized by granulomatous structures primarily in the lungs and lymphatic system, affects individuals globally, with pulmonary fibrosis being its most severe complication. The Phase II trial for OATD-01 represents a significant advancement in addressing this unmet medical need, with results expected to shape future treatment strategies and improve patient outcomes.

 

Source: globenewswire.com

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