Molecure submits application to the European Medicines Agency (EMA) to initiate a phase II clinical trial in the European Union and Norway to investigate OATD-01 for the treatment of pulmonary sarcoidosis
- OATD-01 is a first-in-class inhibitor of chitotriosidase 1 (CHIT1) with disease-modifying potential in sarcoidosis and other interstitial lung diseases
- EMA approval will enable clinical trials to be conducted in centers in the European Union, including several sites in Poland, and in Norway
- In July 2023, Molecure received approval from the U.S. Food and Drug Administration (FDA) to conduct a phase II clinical trial for OATD-01 in the U.S., expected to start in the fourth quarter 2023
- In September, the Central Institutional Review Board approved the Company’s application, which enables the qualification of sites conducting clinical trials in the USA
Warsaw, September 8, 2023 – Molecure S.A. (“Molecure”, GPW ticker: MOC), clinical stage biotechnology company developing first-in-class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases, has applied to the European Medicines Agency (EMA) for permission to initiate a phase II study for OATD-01, a first-in-class and potentially disease-modifying chitotriosidase 1 (CHIT1) inhibitor. EMA approval will enable Molecure to start a phase II clinical trial in the European Union and in Norway to evaluate the safety and efficacy of OATD-01 for the treatment of pulmonary sarcoidosis.
“This is an important step for Molecure, bringing us closer to the start of a phase II clinical trial for OATD-01 in Europe. The European Union – after the USA – is the largest regulated market where we intend to conduct a clinical trial for our innovative CHIT1 inhibitor. said Marcin Szumowski, President of the Management Board of Molecure S.A.
“Administration of the drug to the first patient with pulmonary sarcoidosis is scheduled for the fourth quarter 2023. We will start the study in the USA at the same time, having received FDA approval in July, and where we have the approval of the American bioethics committee to qualify medical centers where the study will be carried out.
“The clinical development of our leading program is entering a very important clinical proof-of-concept stage and the protocol of this study has gained wide approval among eminent specialists in the field of lung diseases. We expect the completion of the US study together with the final report in 2025. I am convinced that OATD-01, which has been shown to modify the course of the disease in preclinical studies, has the potential to become the new standard of care for pulmonary sarcoidosis. This is another step forward in the implementation of our mission – to improve the health and quality of life of patients struggling with incurable diseases. We believe that our drug, if trials are successful, could change the fate of patients with pulmonary sarcoidosis” – added Mr. Szumowski.
The phase II clinical trial for OATD-01 is designed as a multicenter, randomized, double-blind, placebo-controlled trial to evaluate safety and efficacy in approximately 90 patients with active pulmonary sarcoidosis.
For the efficacy study, an innovative primary endpoint was established with the FDA, i.e. response to 12-week administration measured by the degree of reduction of granulomatous inflammation in the lung parenchyma, assessed by PET/CT imaging. In the middle of the study, i.e. for about 50 patients, an intermediate checkpoint is planned in order to assess it statistically and decide on the further course of the study in terms of the number of patients – this should take place 2024. The disclosure of the final trial results is anticipated in 2025.
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 represents a promising molecular target due to its role in converting local anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. Blocking CHIT1 activity by OATD-01 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 a variety of inflammatory and fibrotic diseases that represent an unmet medical need, such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH).
Molecure has obtained an FDA designation of ODD (orphan drug designation) for OATD-01 in indications of sarcoidosis and idiopathic pulmonary fibrosis.
Sarcoidosis is a multi-organ disease of unknown etiology, which is characterized by the formation of granulomatous structures in various organs, mainly in the lungs and lymphatic system.
Sarcoidosis is a globally occurring disease, affecting both men and women with an estimated incidence of 5-50 cases per 100,000 inhabitants, with 70% of patients between the ages of 25-45.
The most serious and common complication of sarcoidosis is pulmonary fibrosis. This is usually associated with significant impairment of lung function. Pulmonary fibrosis is the cause of most sarcoidosis-related deaths in Western countries.
For further information, please contact:
Molecure S.A. (PR & IR)
(+48) 728 728 143
Frazer Hall, Sandi Greenwood
+44 (0)203 928 6900
About Molecure S.A.
Molecure S.A. is a biotechnology company discovering and developing drugs to the clinical stage, which uses its own unique competences in the field of 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 be the therapy of many incurable diseases.
Molecure has generated a diverse portfolio of seven distinct programs with the support of leading academic scientific institutions around the world, 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 use in the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is ready to enter phase II clinical trials. The start of a phase 2 trial in sarcoidosis patients is expected in early Q4 2023.
The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) for use in cancer treatment, whose phase I clinical trial began with its first administration to a patient in Q1 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 at: https://molecure.com/pl/