Molecure files an Investigational New Drug (IND) application for lead clinical candidate OATD-01 with U.S. FDA ahead of a planned Phase 2 pulmonary sarcoidosis study

Molecure files an Investigational New Drug (IND) application for lead clinical candidate OATD-01 with U.S. FDA ahead of a planned Phase 2 pulmonary sarcoidosis study

·       OATD-01 is a first in class chitotriosidase 1 (CHIT1) inhibitor with disease modifying potential in sarcoidosis and other interstitial lung diseases

·       Clearance of the IND application will pave the way for Molecure to initiate the study in the United States becoming only the second Polish biotech company from Poland ever to do so

Warsaw, Poland 23 June 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 filed an Investigational New Drug (IND) application with the U.S. FDA for OATD-01, a first in class and potentially disease modifying Chitotriosidase (CHIT1) inhibitor. Clearance of the IND would allow Molecure to begin an international Phase 2 proof of concept study to evaluate OATD-01 for the treatment of pulmonary sarcoidosis. 

“Today’s IND filing is another landmark milestone for Molecure and its lead product OATD-01” said Molecure CEO Marcin Szumowski. “There are currently no approved therapies for sarcoidosis, and while steroids are often used, they have serious side effects and deliver only short-term benefits with little evidence of extended therapeutic efficacy. As a result, there is a significant unmet need for better treatments that can prevent disease progression. We believe that OATD-01, which has demonstrated efficacy in several pre-clinical models, could alter the fate of sarcoidosis patients by becoming the new standard of care for sarcoidosis. Clearance of our IND would allow Molecure to become only the second, Polish biotech company in history to initiate in the United States a multicenter Phase 2 study for an innovative drug, a major accomplishment for everyone involved in this programme."

Samson Fung, Molecure CMO and Member of the Management Board added, “This IND application brings us a step closer to a new treatment option for underserved sarcoidosis patients. We are excited about our plans to advance OATD-01 into a Phase 2 trial as we believe this highly promising new agent could play a key role in the treatment of this rare disease, improving symptoms as well as quality of life and potentially stopping disease progression. We anticipate beginning our Phase 2 trial in patients in the US and EU in the fourth quarter of this year.

This Phase 2 trial is expected to be a global, multi-center, randomized, double blind and placebo-controlled study to evaluate the safety and efficacy of OATD-01 in approximately 90 patients with active pulmonary sarcoidosis. The results of this double-blinded study will be available once the study is complete, expected in H1 2025. The innovative primary endpoint, already discussed and cleared by the FDA during the pre-IND meeting procedure is the response to a 12-week treatment with OATD-01 measured by the reduction of granulomatous inflammation in pulmonary parenchyma evaluated by PET/CT imaging.”

Molecure has received orphan drug designation (ODD) from the FDA for OATD-01 in the indications of sarcoidosis and idiopathic pulmonary fibrosis.

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.

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.

ENDS