Insilico Medicine, an end-to-end artificial intelligence (AI)-driven drug discovery company, announced the initiation of a first-in-human microdose trial of ISM001-055, a potentially first-in-class small molecule inhibitor for idiopathic pulmonary fibrosis (IPF). This chronic lung disease leads to a progressive and irreversible decline in lung function. The trial, conducted in Australia, aims to characterize the pharmacokinetic profile of ISM001-055 in humans.
ISM001-055 has shown promising results in preclinical studies, including in vitro biological studies, pharmacokinetic, and safety studies. It significantly improved myofibroblast activation, which is crucial in the development of fibrosis. The novel target of ISM001-055 is potentially relevant to a broad range of fibrotic indications.
Feng Ren, Ph.D., CSO of Insilico Medicine, highlighted the significance of this milestone, stating that ISM001-055 is the first AI-discovered novel molecule based on an AI-discovered novel target to enter clinical trials. The company leveraged its AI-powered drug discovery platform, including generative biology and generative chemistry, to discover novel biological targets and generate novel molecules with drug-like properties.
Insilico Medicine's achievement in completing the discovery process from target discovery to preclinical candidate nomination within 18 months on a budget of $2.6 million is notable. Michael Levitt, 2013 Nobel Laureate in Chemistry and member of Insilico Medicine’s Scientific Advisory Board, praised the achievement for its reproducibility and revolutionary method.
Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine, emphasized the platform's ability to overcome the low probability of reaching this stage, highlighting the challenge of target discovery in the pharmaceutical industry. The company's AI platform connects biology and chemistry to assess activity and safety in multiple preclinical models.
Prof. John Kao, Head of Institute for Translational Research of HKSTP, shared the excitement about Insilico Medicine's transition from drug discovery to drug development, marking an important milestone in translational research.
