Pharma Focus Europe

Molecure Granted Approval to Conduct Phase II Clinical Trial for OATD-01 Targeting Pulmonary Sarcoidosis in Specific European Union and Norwegian Regions

Wednesday, May 22, 2024

Molecure S.A. has recently obtained regulatory approvals from several European countries, including Denmark, France, Greece, Germany, and Norway, to proceed with a Phase II clinical trial for OATD-01. This drug, designed to inhibit chitotriosidase 1 (CHIT1), shows promise in treating pulmonary sarcoidosis.

CEO Marcin Szumowski expressed enthusiasm for this development, noting that patient administration began in March at the Royal Infirmary in Edinburgh. With approvals secured in key European markets, the trial aims to administer initial doses to patients in the third quarter of this year. Szumowski emphasized the importance of conducting trials in both the USA and Europe to gather crucial data, expected to confirm the clinical efficacy of OATD-01 in treating pulmonary sarcoidosis, a condition with limited treatment options.

The Phase II trial, a randomized, double-blinded, placebo-controlled study, seeks to assess OATD-01's safety and effectiveness in approximately 100 patients with active pulmonary sarcoidosis. Notably, the trial incorporates innovative endpoints, including PET/CT imaging to measure granulomatous inflammation reduction in lung tissue.

Following completion of dosing and monitoring, an interim analysis is scheduled for early 2025 to guide further study. Simbec Orion, a prominent Contract Research Organization, will oversee the comprehensive trial, which will span multiple centers across the USA, EU, Norway, and the UK.

OATD-01, an oral, once-daily CHIT1 inhibitor, holds promise for treating sarcoidosis by targeting inflammatory processes. Its documented anti-inflammatory and anti-fibrotic effects suggest broader applicability in fibrotic diseases like idiopathic pulmonary fibrosis (IPF) and metabolic dysfunction-associated steatohepatitis (MASH).

Molecure has secured orphan drug designation from the FDA for OATD-01, reflecting its potential in sarcoidosis and pulmonary fibrosis. Sarcoidosis, a multi-organ disease of unknown origin, affects individuals globally and poses significant health risks, particularly pulmonary fibrosis, which is a leading cause of mortality associated with the condition.



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