Molecure receives approval to initiate a Phase II clinical trial (KITE) for OATD-01 for the treatment of pulmonary sarcoidosis in selected countries of the European Union and Norway

· OATD-01 is a first-in-class chitotriosidase 1 (CHIT1) inhibitor with the potential to modify the course of disease in sarcoidosis and other interstitial lung diseases.
· Obtaining regulatory approvals in Denmark, France, Greece, Germany, and Norway enables the conduct of the Phase II clinical trial of OATD-01 in these countries.
· First administration of OATD-01 in the European Union and Norway is planned for the third quarter of this year.
· Previously, Molecure received clinical trial approvals from the US FDA and the UK MHRA, with first patients dosed in March 2024 in the UK.
· First-ever administration of a chitotriosidase 1 (CHIT1) inhibitor is a significant milestone in the clinical development of Molecure's leading program.
*Warsaw, 21st May 2024 – *Molecure S.A. (‘Molecure’, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical stage, leveraging its globally unique expertise in medicinal chemistry and biology to explore and develop first-in-class small molecule drugs that directly modulate protein activity and mRNA function for the treatment of multiple incurable diseases, announces today 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.

*Marcin Szumowski, Molecure’s CEO* said: “An important milestone has been reached in the Phase II clinical trial of OATD-01 with the first patient administration in March this year at the Royal Infirmary in Edinburgh. We are pleased to receive further regulatory approvals from selected EU countries and Norway to conduct this breakthrough clinical study. The first dose is expected to be administered to patients in the selected countries in the third quarter of this year. The Phase II clinical trials to be conducted in both the USA and Europe, the two most commercially important markets, will enable us to collect the necessary data that, if positive, will confirm clinical proof of concept in a pulmonary sarcoidosis patient population that currently has few treatment options with limited efficacy in disease modification. OATD-01 has demonstrated remarkable potential in preclinical studies, suggesting it could redefine the standard of care for pulmonary sarcoidosis. The results of these studies will be crucial for further development and commercialization of OATD-01 in this and other potential indications such as NASH/MASH, IPF or inflammatory bowel disease (IBD). We look forward to presenting the unblinded results of this study in late 2025.”

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 Q1 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.

*About OATD-01*

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.

*About sarcoidosis*

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.

*About Molecure S.A.*

Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, leveraging its unique expertise in medicinal chemistry and biology to search for and develop first-in-class small molecule drugs that, through direct modulation of previously unexplored protein and RNA targets, can provide therapy for many incurable diseases.

Molecure has generated a diversified portfolio of seven distinct programs with the support of leading academic institutions worldwide, including Yale University, Rutgers University, the Flemish Institute for Biotechnology (VIB) in Ghent, the University of Michigan, and the International Institute of Molecular and Cell Biology in Warsaw (MIBMiK).

The most advanced drug candidate developed by Molecure is OATD-01, a first-in-class CHIT1 inhibitor for the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis.

The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancers, with Phase I clinical trials commencing with the first patient administration in the first quarter of 2023.

Molecure's headquarters and laboratories are located in Warsaw and Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

Detailed information can be found on: https://molecure.com/pl/

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