Molecure S.A. has received clearance from the U.S. Food and Drug Administration (FDA) on its Investigational New Drug (IND) application to conduct phase II clinical testing of OATD-01.
The announcement follows the company closing a secondary public offering and entering into subscription agreements for all 2,776,000 H-shares offered by way of private placement, within the authorized capital. The issue price was set at PLN 18, bringing the gross value of the offering to approximately PLN 50 million ($12 million).
OATD-01 is the first-ever chitotriosidase 1 (CHIT1) inhibitor with disease-modifying potential. The first pulmonary sarcoidosis patients in this phase II study are scheduled to begin receiving treatment in Q4 2O23.
“We are extremely excited to share this great news – not only for us, but for every patient looking for a better and more effective way of treating sarcoidosis. This marks the beginning of a new chapter in the development of our flagship project, as we enter human proof-of-concept studies and begin treating pulmonary sarcoidosis patients with OATD-01. As we have confirmed in a range of preclinical studies, OATD-01 has the ability to modulate macrophage activity, meaning it has the potential to be disease modifying and treat various inflammatory and fibrotic diseases which develop based on a similar molecular mechanism.
“In the coming weeks we are also planning to file for approval of phase II clinical trials with the European Medicines Agency (EMA). That would make it possible for us to begin testing OATD-01 in the European Union – including Poland. We expect to conclude these phase II clinical studies in mid-2025 with the publication of a report analyzing the headline data,” said Marcin Szumowski, CEO of Molecure.
“In recent months we’ve put a lot of effort into raising the profile of our clinical research plans with OATD-01 internationally – a process designed to build relationships with renowned clinical experts who specialize in lung diseases (including sarcoidosis), as well as foundations and other organizations which build communities to support patients suffering from a range of difficult diseases that have a significant and negative impact on the quality of their lives.”
OATD-01 has displayed disease-modifying abilities in preclinical trials and has the potential to become the new standard of care for treating pulmonary sarcoidosis.
The phase II clinical trial of OATD-01 is expected to be a multi-center, randomized, double-blind, placebo-controlled study assessing the drug’s safety and effectiveness in treating approximately 90 pulmonary sarcoidosis patients. As a result of the double-blind requirement, the study’s final unblinded results will be published after its conclusion which is scheduled for the first half of 2025.
The study has an innovative primary efficacy endpoint – the level to which OATD-01 is able to reduce granulomatous inflammation in the pulmonary parenchyma over a 12-week period based on PET/CT scan results. This endpoint was agreed with the FDA following a pre-IND meeting.
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 and 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.
The proceeds raised through the offering will co-finance the implementation of the company’s strategic plans for 2023 to 2025, particularly the clinical development of its two flagship programmes, namely OATD-01 and OATD-02 (a first-in-class dual arginase inhibitor for cancer).
Additionally, efforts will be intensified in a portfolio of early-stage programs, including breakthrough small-molecule drug technology that modulates mRNA translation and therapeutics targeting previously unexplored protein targets. These initiatives are bolstered by advanced machine learning and generative artificial intelligence (GenAI) methods.
Molecure estimates that the capital expenditure associated with the strategy from mid-2023 to the end of 2025 will amount to approximately PLN 250 million ($60 million).