Idiopathic Pulmonary Fibrosis (IPF) is a chronic, scarring lung disease characterized by a progressive and irreversible decline in lung function. Affecting approximately 5 million people worldwide, IPF carries a poor prognosis, with a median survival of 3 to 4 years. Current approved treatments, including antifibrotic drugs, can slow disease progression but do not stop or reverse it, leaving a significant unmet need for more effective, disease-modifying therapies.
ISM001-055 is a potentially first-in-class small molecule targeting TNIK developed utilizing generative AI. In IPF, the activation of TNIK drives pathological fibrosis in the lungs, contributing to the progressive decline in lung function. By inhibiting TNIK, ISM001-055 aims to halt or reverse fibrotic processes, offering a disease-modifying treatment for patients with IPF. The history of discovery, design and development including target discovery, generative chemistry, multiple
in-vitro and
in-vivo experiments as well as the results of Phase I clinical studies in human volunteers were published in a
Nature Biotechnology article in March 2024. The medicinal chemistry portion of ISM001-055’s discovery was published in the
Journal of Medicinal Chemistry in October 2024. Potential additional roles of TNIK in age-related diseases were published in
Cell Trends in Pharmacological Sciences in June 2024.
Insilico will be initiating discussions with regulatory bodies based on these encouraging results and will be pursuing a pivotal trial of ISM001-055 in IPF patients. In addition, complete Phase IIa data from this study will be presented at an upcoming medical conference and published in reputable medical journals.