Molecure has submitted a new application for approval to conduct a Phase II clinical trial for clinical candidate OATD-01 in the countries of the European Union and Norway
- Molecure has resubmitted an application for approval to conduct a Phase II clinical trial for OATD-01 in Denmark, France, Greece, Germany and Norway. The company has proposed Denmark as the rapporteur Member State for the application evaluation process.
- In the previous coordinated evaluation procedure, the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products refused the request, and the consequence of this refusal was that, despite the approval of the ethics committees in Greece, Germany, France, Denmark, Poland and Norway, it was not possible to conduct the clinical trial in the requested European Union countries because Poland was reported in the request as the rapporteur Member State.
- Following approvals from the US Food and Drug Administration (FDA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) and the relevant ethics committees in those countries to conduct a Phase II clinical trial for OATD-01, Molecure is progressing smoothly in preparation for the start of the clinical trial and is finalising the contracting of sites in the US and UK.
Warsaw, 9th February 2024. – The company Molecure S.A. („Molecure”, WSE ticker: MOC), a biotechnology company that discovers and develops drugs to the clinical development stage and leverages its globally unique expertise in medicinal chemistry and biology to search for and develop first-in-class small-molecule drugs that directly modulate protein activity and mRNA function for the treatment of multiple incurable diseases, has submitted a new Phase II clinical trial application for OATD-01 in patients with active pulmonary sarcoidosis in the countries of the European Union and Norway, as announced, and Denmark has been proposed as the rapporteur Member State for the application evaluation process.
In the previous proceedings, Poland was the rapporteur Member State, in accordance with the EMA’s recommendations and the Company’s proposal, but due to the strict limits imposed by Polish law on the dose of ionising radiation a patient can receive in a clinical trial, Poland ultimately refused to allow the trial to proceed, despite the Company providing documentation indicating that the radiation level in diagnostic imaging could be reduced below the limit indicated in the Ordinance of the Minister of Health of 11 January 2023 on the conditions for the safe use of ionising radiation for all types of medical exposure (Journal of Laws 2023, item 195), and indication of the specific authorised technology that makes this possible.
„As announced, we have resubmitted an updated application for approval to conduct the clinical trial in the European Union countries and Norway. We have proposed Denmark as a rapporteur Member State in the new application, not least because the prevalence of this disease in Scandinavian countries, including Denmark, is one of the highest in relation to other European countries. We hope that our proposal will proceed smoothly, not least because the relevant authorities in the individual countries have already taken note of our proposal in the first procedure and the relevant ethics committees in all countries have agreed to the study. Following the approval of our application, we expect the first administration in the European Union and Norway to take place at the end of the second and beginning of the third quarter of this year. We were very keen to enable patients in Poland suffering from sarcoidosis to take part in the trial and thereby give them access to an innovative therapy with significant potential. We regret that we ultimately had to abandon the clinical trial in Polish centres due to the difficulty of performing the full range of medical procedures foreseen in the protocol, including PET/CT imaging. This diagnostic method is crucial for assessing the efficacy of the investigational drug and we chose it after consultation with a broad international group of advisors and key opinion leaders in pulmonology and nuclear medicine. It is worth noting that the first administration of OATD-01 will soon take place in both the US and the UK, where we have already received approvals from the relevant regulators and are completing the contracting of the last study sites. Poland, being the only EU country with such restrictive limits on diagnostic imaging that cannot be justified scientifically, effectively excludes many patients with non-oncological diseases from access to potentially groundbreaking experimental therapies that save their lives and health. 'We would be happy to return to Poland if the current limits are changed or the Polish regulator agrees to use certified technology that reduces this irradiation.” – comments Marcin Szumowski, CEO of Molecure S.A.
As designed, the Phase II clinical trial for OATD-01 is a multicentre, double-blinded, randomised, placebo-controlled study to assess safety and efficacy in approximately 90 patients with active pulmonary sarcoidosis. Due to the double-blinding requirement of the study, publication of the final unblinded results will follow its completion and is anticipated in the second half of 2025.
The study has an established innovative primary efficacy endpoint. This is the level to which OATD-01 will reduce granulomatous inflammation in the pulmonary parenchyma after 12 weeks of administration, as assessed by PET/CT imaging.
OATD-01 has shown disease-modifying potential in preclinical studies. Molecure believes this molecule could become a new medical standard for the treatment of pulmonary sarcoidosis.
OATD-01 is an orally administered 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 demonstrated potent anti-inflammatory and anti-fibrotic activity in various disease models and has high therapeutic potential in a variety of inflammatory and fibrotic diseases, the treatment of which represents an unmet medical need; such diseases include sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and non-alcoholic steatohepatitis (NASH)).
Molecure has received orphan drug designation (ODD) from the US FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis.
Sarcoidosis is a multi-organ disease of unknown aetiology characterised by the formation of granulomatous structures in various organs, mainly in the lungs and lymphatic system.
Sarcoidosis is a globally prevalent disease, affecting both men and women with an estimated incidence of 5-50 cases per 100,000 people, with 70% of patients being between 25 and 45 years of age.
The most serious and common complication of sarcoidosis is pulmonary fibrosis. It is usually associated with significant impairment of lung function. Pulmonary fibrosis accounts for the majority of sarcoidosis-related deaths in Western countries.
About Molecure S.A.
Molecure S.A. is a biotechnology company that discovers and develops drugs to the clinical development stage, using its own 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, could provide therapies for many incurable diseases.
Molecure has generated a diverse portfolio of seven distinct programmes with the support of leading academic research 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 the treatment of interstitial lung diseases such as sarcoidosis and idiopathic pulmonary fibrosis, which is currently entering Phase II clinical trials. The Phase II trial in sarcoidosis patients, a proof-of-concept study, will begin in Q1 2024 in the US and UK, and will also continue in the EU and Norway following approval from European regulators.
The second drug candidate is OATD-02, an oral, selective, first-in-class, dual arginase inhibitor (ARG1 and ARG2) being developed for use in cancer treatment, with a Phase I clinical trial commencing with first patient administration in Q1 2023.
Molecure’s headquarters and laboratories are located in Warsaw and Lodz. The company is listed on the Warsaw Stock Exchange (ticker: MOC).
Detailed information can be found on: https://molecure.com/pl/