The U.S. Food and Drug Administration Gives the Green Light to Molecure's Flagship Program - Phase II Clinical Testing of OATD-01 is Ready for Launch

Press release

The U.S. Food and Drug Administration Gives the Green Light to Molecure's Flagship Program - Phase II Clinical Testing of OATD-01 is Ready for Launch

• Molecure can proceed with clinical trials in the U.S.A - Phase II proof-of-concept study will be the first to treat patients suffering from pulmonary sarcoidosis with OATD-01
• In the coming weeks Molecure will also seek the European Medicine Agency's approval to conduct phase II clinical testing of OATD-01 in the European Union
• OATD-01 is the first-ever chitotriosidase 1 (CHIT1) inhibitor with disease modifying potential in sarcoidosis and other interstitial lung diseases
  
  

Warsaw, 24 July 2023, - Molecure S.A. ("Molecure": WSE: MOC) a 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, announces that it 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 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." - says Marcin Szumowski, CEO of Molecure S.A.

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. The Molecure team has also participated in numerous seminars and conferences such as the World Association for Sarcoidosis and Other Granulomatous Disorders (WASOG) 2023 - the largest international meeting place focused on the future of sarcoidosis and other granulomatous disorders. At WASOG we presented the disease modifying potential of OATD-01 to global industry opinion leaders and we are confident that that this will help us get more U.S. and EU research faculties involved in our trials, resulting in faster patient recruitment." - adds Marcin Szumowski.

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 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 end point - 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.

About OATD-01

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.

About Sarcoidosis

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.

About Molecure

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 Lódz. The company is listed on the Warsaw Stock Exchange (ticker: MOC).

For more information, please visit molecure.com/en/

For further information, please contact:

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