Molecure has dosed the first subject in the UK in its proof-of-concept in human Phase II KITE clinical trial of OATD-01, a potential treatment for active pulmonary sarcoidosis.
The Royal Infirmary in Edinburgh administered the first dose of the OATD-01, a chitotriosidase 1 (CHIT1) inhibitor or placebo, marking a significant milestone in the study of this multi-organ disease.
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This double-blind, randomised, placebo-controlled, multicentre trial is designed to assess the safety and efficacy of the oral CHIT1 inhibitor.
Nearly 100 subjects, including those who have and have not received other therapies, will be enrolled to receive either a 25mg daily fixed dose of OATD-01 or a placebo for 12 weeks.
The trial will span 20-30 centres across the European Union, Norway, the US, and the UK.
Simbec Orion, a contract research organisation, is tasked with the conduct of the study.
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By GlobalDataDue to the study’s double-blinding requirement, the final unblinded data are expected to be reported by the end of next year.
The response to a 12-week dosing with OATD-01 as evaluated by the degree of granulomatous inflammation decline in the lung parenchyma will be the trial’s primary endpoint.
After around 50 subjects complete trial participation, an independent committee will carry out a sub-analysis to evaluate the results and decide on the future course of the study, including the number of subjects required.
Molecure received orphan drug designation (ODD) from the US Food and Drug Administration (FDA) for OATD-01 to treat sarcoidosis and idiopathic pulmonary fibrosis.
Molecure CEO Dr Marcin Szumowski said: “Launched in the UK, the Phase II trial involving patients with active disease, the ‘proof-of-concept in human’ phase, is a significant milestone in the clinical development of our lead programme.
“It is a landmark moment also because it is the first time that a chitotriosidase 1 inhibitor has been offered to patients.
“The results of this study will be strategically important for further value creation and commercialisation of OATD-01, and we look forward to the results of the study in 2025.”
Last June, the company submitted an investigational new drug (IND) application to the FDA for commencing a Phase II trial of OATD-01 to treat pulmonary sarcoidosis.
