Insilico Medicine has announced the commencement of a first-in-human microdose trial for ISM001-055, a potentially first-in-class small molecule inhibitor discovered using its end-to-end artificial intelligence (AI)-powered drug discovery platform, Pharma.AI. The trial, which is being conducted in Australia, marks a significant milestone as it represents one of the first AI-discovered novel molecules based on an AI-discovered novel target to enter clinical development. The drug is being developed for the treatment of idiopathic pulmonary fibrosis (IPF), a chronic lung disease characterized by progressive and irreversible decline in lung function.
ISM001-055: A Novel Approach to Fibrosis
ISM001-055 is designed to inhibit a novel biological target identified by Insilico's AI platform. Preclinical studies have demonstrated promising results, including significant improvement in myofibroblast activation, a key process in the development of fibrosis. These findings suggest that ISM001-055 could be relevant to a broad range of fibrotic indications beyond IPF.
AI-Driven Drug Discovery
Insilico Medicine's achievement highlights the potential of AI in accelerating drug discovery. According to Insilico, the entire process, from target discovery to preclinical candidate nomination, was completed within 18 months on a budget of $2.6 million. This timeline and cost are significantly lower than traditional drug discovery approaches.
"We are very pleased to see Insilico Medicine’s first antifibrotic drug candidate entering into the clinic. We believe this is a significant milestone in the history of AI-powered drug discovery because to our knowledge the drug candidate is the first ever AI-discovered novel molecule based on an AI-discovered novel target," said Feng Ren, Ph.D., CSO of Insilico Medicine.
The microdose trial aims to characterize the pharmacokinetic profile of ISM001-055 in humans. The drug is administered intravenously to healthy volunteers.
Expert Perspectives
Michael Levitt, 2013 Nobel Laureate in Chemistry and member of Insilico Medicine’s Scientific Advisory Board, commented, "AI is a way to search for information and look for signals for drug discovery... This achievement didn't happen by chance and is a reproducible method and procedure which is revolutionary."
Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine, added, "There are very few examples of a pharmaceutical company discovering a new target for a broad range of diseases, designing a novel molecule, and initiating human clinical trials. To my knowledge, nobody has achieved this with AI to-date."
