OATD-01 is a first-in-class small molecule chitinase inhibitor that successfully completed Phase I clinical trials in healthy volunteers.
OATD-01 inhibits the activity of chitinases (CHIT1 and AMCase), which are enzymes that break down chitin, a component of many organisms, which is not produced by the human body. However, our Company among other academic and clinical research groups has shown that macrophages, or cells that are activated during inflammation, produce an increased amount of chitinases. This often correlates with the course of a number of inflammatory diseases, and the activity of chitinases is closely related to the processes of organ fibrosis, especially in the lungs. Our scientists have shown that removing a gene that encodes one of the chitinases leads to a much milder course of inflammation and fibrosis in preclinical models. We also confirmed that by blocking chitinases with OATD-01, we can significantly reduce the burden of disease in the same animal models. Thanks to this, our compound can find potential use in idiopathic pulmonary fibrosis, sarcoidosis or asthma, diseases that still pose a major therapeutic problem.
OATD-01 has an orphan drug designation (ODD) status for the treatment of idiopathic pulmonary fibrosis and sarcoidosis by the U.S. Food and Drug Administration (FDA).
Idiopathic Pulmonary Fibrosis (IPF) is a disease of the group of interstitial lung diseases, with an undetermined etiology and poor prognosis. It is characterized by progressive pulmonary fibrosis leading to respiratory failure. The average survival of patients from diagnosis is 3 to 5 years. In recent years, two drugs for IPF therapy have been registered – pirfenidone and nintedanib. These drugs limit the decline of lung capacity, that is, slow down the progression of the disease, but they are not fully efficacious, hence IPF still represents an important unmet medical need.
Sarcoidosis is a systemic disease, manifested by the formation of nodules called granulomas composed of inflammatory cells, which can be localized in various organs, most often in the lungs. In most cases, granulomas are eliminated spontaneously or as a result of treatment with oral corticosteoroid anti-inflammatory drugs. In about 30% of patients, however, the disease progresses, there is no response to treatment and pulmonary interstitial fibrosis occurs leading to an impairment of lung performance. There is currently no effective advanced, progressive sarcoidosis therapy.