Molecure gets approval for Phase II trial of OATD-01 for pulmonary sarcoidosis in EU and Norway

Molecure S.A. has announced the receipt of national regulatory approvals from Denmark, France, Greece, Germany, and Norway to conduct a Phase II clinical trial for OATD-01, a pioneering chitotriosidase 1 (CHIT1) inhibitor. This approval marks a significant step in the development of OATD-01, which has the potential to modify the course of diseases such as pulmonary sarcoidosis and other interstitial lung diseases.

Molecure, a biotechnology company known for its expertise in medicinal chemistry and biology, focuses on developing first-in-class small molecule drugs that target protein activity and mRNA function to treat various incurable diseases. The company's CEO, Marcin Szumowski, highlighted the importance of this milestone, noting that the first patient administration of OATD-01 occurred in March at the Royal Infirmary in Edinburgh.

With these new regulatory approvals, the company plans to administer the first dose of OATD-01 to patients in Denmark, France, Greece, Germany, and Norway in the third quarter of this year. The Phase II clinical trials will be conducted in both the USA and Europe to gather crucial data. Positive results from these trials could confirm the clinical proof of concept for OATD-01 in treating pulmonary sarcoidosis, a condition that currently has limited treatment options with efficacy in disease modification.

OATD-01 has shown promising results in preclinical studies, suggesting its potential to redefine the standard of care for pulmonary sarcoidosis. The outcome of these studies will be pivotal for the further development and commercialization of OATD-01 for this and other potential indications, such as NASH/MASH, IPF, or inflammatory bowel disease (IBD). Molecure aims to present the unblinded results of the study by late 2025.

The Phase II clinical trial for OATD-01 is designed as a randomized, double-blinded, placebo-controlled, multicenter study. It will evaluate the safety and efficacy of OATD-01 in approximately 100 patients with active pulmonary sarcoidosis, including those who have not responded to previous therapies and those who have not been treated before. Due to the double-blind nature of the study, the final unblinded results will be published after the study's completion, scheduled for the end of 2025.

The study will measure the efficacy of OATD-01 through an innovative primary endpoint, assessing the response to a 12-week administration of OATD-01 by measuring the reduction in granulomatous inflammation in the lung parenchyma using PET/CT imaging. An interim analysis is planned after dosing approximately 50 patients, with an independent committee evaluating the results and deciding on the study's continuation in early Q1 of 2025.

Around 20-30 centers in the USA, European Union, Norway, and the United Kingdom will be involved in the study, with the renowned Contract Research Organization (CRO) Simbec Orion responsible for organizing and conducting the comprehensive study.

OATD-01 is an oral, once-daily, highly selective CHIT1 inhibitor for treating sarcoidosis. The enzyme CHIT1 is a promising molecular target due to its role in transforming anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. Blocking CHIT1 activity with OATD-01 has shown documented anti-inflammatory and anti-fibrotic effects in various disease models.

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 European Union countries, and Norway.

Sarcoidosis is a multi-organ disease of unknown origin, characterized by granulomatous structures in various organs, primarily the lungs and lymphatic system. It affects both men and women globally, with an estimated incidence of 5-50 cases per 100,000 population. Pulmonary fibrosis, a common and serious complication of sarcoidosis, significantly impairs lung function and is the leading cause of sarcoidosis-related deaths in Western countries.

Molecure S.A. is at the forefront of biotechnology innovation, leveraging its expertise to discover and develop drugs that target previously unexplored protein and RNA targets. The company's diversified portfolio includes seven distinct programs supported by leading academic institutions worldwide. Molecure's headquarters and laboratories are located in Warsaw and Łódź, and the company is listed on the Warsaw Stock Exchange under the ticker MOC.