Molecure has signed an agreement to conduct a Phase II clinical trial for its lead molecule OATD-01, taking another important step in the development of this program.
- The agreement with Simbec-Orion includes organizing and fully executing a proof-of-concept Phase II clinical trial in patients with pulmonary sarcoidosis.
- The Phase II proof-of-concept study of OATD-01 will be conducted as multi-center and international clinical trial in the United States and the European Union and is expected to start in Q4 2023.
- OATD-01 is a first-in-class chitotriosidase 1 (CHIT1) inhibitor that modulates macrophage function and has broad therapeutic potential for the treatment of inflammatory and fibrotic diseases.
- Molecure has filed an Investigational New Drug (IND) application with the US Food and Drug Administration (FDA) for a Phase II study in the United States.
- Molecure has received Orphan Drug Designation (ODD) from the FDA for OATD-01 in sarcoidosis and idiopathic pulmonary fibrosis.
Warsaw, 3rd July 2023 – Molecure S.A. (“Molecure”: WSE: MOC) a clinical stage biotechnology company that uses its world leading medicinal chemistry and biology capabilities to discover and develop first in class small molecule drug candidates that directly modulate unexplored protein and RNA targets to treat multiple incurable diseases, announced that it has entered into an agreement with Orion Sante Sarl (Simbec-Orion), based in France, to organize and conduct a comprehensive Phase II clinical trial for OATD-01.
The OATD-01 Phase II trial will be conducted in the US and several European Union countries. Phase II is planned as a double-blind, placebo-controlled, clinical trial with a fixed dose of compound OATD-01 administered once daily for 12 weeks. The study is designed to evaluate the clinical efficacy, pharmacokinetics, pharmacodynamics, and safety of OATD-01 in patients with active pulmonary sarcoidosis. The value of the agreement with Simbec-Orion is approximately €10.1 million (PLN 45 million – according to the average exchange rate of the National Bank of Poland published on 3rd July 2023, i.e., PLN 4.43).
“The signing of an agreement with Simbec-Orion, an experienced international CRO, marks another important step in recent days towards the initiation of a Phase II trial of OATD-01 in sarcoidosis patients. We recently announced the submission of an IND application to the US Food and Drug Administration (FDA) for this trial, which was preceded by a meeting with the regulator to discuss the dossier and the design of the clinical trial, including its innovative endpoints. We have provided the FDA with more than 20,000 pages of documentation from preclinical studies conducted to date and from the completed Phase I study in 2020. In the near future, we will submit a similar clinical trial application to the EMA under the European Centralised Procedure. We expect to enroll the first patient in the trial in early Q4 2023. The trial will enroll approximately 90 patients with active pulmonary sarcoidosis in the US and Europe, including Poland. This phase of the trial, known as “proof-of-concept in humans”, is critical to the validation of the chitinase platform as it is the first time a chitinase inhibitor has been administered to patients worldwide. Positive results from this phase could pave the way for many other indications with significant unmet medical needs worldwide, such as other interstitial lung diseases or non-alcoholic steatohepatitis (NASH), where similar molecular mechanisms lead to disease development,” – said Marcin Szumowski, CEO of Molecure S.A.
“Sarcoidosis is a rare but highly debilitating disease that significantly reduces the quality of life and, in some cases, leads to death of patients. It remains an unmet clinical need, and the needs of patients are often overlooked. We are, therefore, committed to initiating this trial as soon as possible as we believe that OATD-01 can play an important role in helping patients, not only by alleviating their symptoms but also by improving their quality of life and potentially halting the progression of the disease. Our innovative compound OATD-01, by blocking chitotriosidase 1, is the first-in-class potential drug using this mechanism to modify macrophage function. Our planned study includes an innovative primary efficacy endpoint, consulted with the FDA in a pre-IND meeting. This is the response to 12-week administration of OATD-01, verified by PET/CT imaging, reduction in size and number of granulomas in patients’ lungs. We prepared the study design, including the recruitment criteria and its endpoints, in collaboration with key opinion leaders in fibrotic and pulmonary diseases. We hope that with our most advanced molecule, we will soon be able to bring help to patients with sarcoidosis, fulfilling our mission,” – said Dr. Samson Fung, MD, Chief Medical Officer, and Board Member of Molecure S.A.
Simbec-Orion was selected following a competitive tender organized by Molecure.
OATD-01, is an oral, once-daily, first-in-class highly selective CHIT1 inhibitor for the treatment of sarcoidosis. CHIT1 is a promising molecular target through its role in transforming resident, anti-inflammatory macrophages into pro-inflammatory and pro-fibrotic types. The inhibition of CHIT1 by OATD‑01 has been shown to reduce inflammation & fibrosis.
OATD-01 has demonstrated potent anti-inflammatory and antifibrotic effects in various disease models and has high therapeutic potential in diverse inflammatory and fibrotic diseases with high unmet medical needs such as sarcoidosis, as well as idiopathic pulmonary fibrosis (IPF) and NASH.
Molecure has received orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis.
Sarcoidosis is a systemic disease of unknown cause that is characterized by the formation of immune granulomas in various organs, mainly the lungs and the lymphatic system.
Sarcoidosis is a global disease, affecting both men and women with a prevalence of about 5–50 in 100,000 with 70% of patients aged between 25 and 45 years.
The most severe and frequent complication of sarcoidosis is the occurrence of pulmonary fibrosis. This is usually associated with significant impairment of pulmonary function. Pulmonary fibrosis results in the majority of deaths related to sarcoidosis in western countries.
Molecure is a clinical stage biotechnology company that uses its world leading medicinal chemistry and biology capabilities to discover and develop first-in-class small molecule drug candidates that directly modulate the function of underexplored protein and RNA targets to treat multiple incurable diseases.
Molecure has generated a diverse pipeline of seven distinct programs with the support of leading academic life science institutions.
Molecure’s most advanced in-house drug candidate is OATD-01, a first in class dual chitinase inhibitor for the treatment of interstitial lung diseases, such as sarcoidosis and idiopathic pulmonary fibrosis, that is Phase II ready. A Phase II trial in patients with sarcoidosis is expected to start in 2023.
Our second proprietary candidate is OATD-02, an oral, potent and selective first in class, dual arginase inhibitor (ARG1 and ARG2) for the treatment of cancer, which advanced to Phase I clinical development in Q1 2023.
Molecure’s headquarters and laboratories are located in Warsaw, Poland with an additional laboratory facility in Łódź. The company is listed on the Warsaw Stock Exchange (ticker: MOC).
For more information, please visit molecure.com/en/
Simbec-Orion is an experienced, full-service Contract Research Organisation (CRO), with offices across the UK, Europe, and the United States. Established for over 45 years, providing clinical trial expertise to a wide range of small to mid-sized biotech and pharmaceutical partners across Europe, North America and beyond.
Across the organisation, scientific teams leverage both a wide therapeutic experience in clinical pharmacology, as well as more specialist expertise in Phase I-IV oncology and rare disease. Simbec-Orion’s adaptable, highly experienced teams continuously take a quality-first approach, whilst maintaining the short communication lines and Senior Leadership Team oversight only achievable with a lean management structure. Learn more at www.simbecorion.com