Insilico used its Pharma.AI platform to identify a promising drug candidate from more than 20 disease targets and dozens of deep-learning-generated molecules.

Insilico Medicine, an end-to-end artificial intelligence (AI)-driven drug discovery company, has dosed a volunteer in Australia with ISM001-055, a potentially first-in-class small molecule inhibitor of a novel biological target discovered by its Pharma.AI platform for the treatment of Idiopathic Pulmonary Fibrosis (IPF). It represents the first trial in humans of this drug candidate. (Fierce Biotech)

IPF is a chronic lung disease that causes a progressive and irreversible decline in lung function. Insilico completed the entire discovery process from target discovery to preclinical candidate nomination within 18 months on a budget of $2.6 million.

“What impresses me the most about this achievement is not only the lower cost or faster speed of development but, also it demonstrated the Pharma AI platform can overcome the low probability of getting to this stage,” said Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine. “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. The failure rates in preclinical target discovery are very high and even after the targets are validated in animal models, over half of Phase 2 clinical trials fail primarily due to the choice of target. Target discovery is the fundamental grand challenge of the pharmaceutical industry. With ISM001-055 we used end-to-end AI connecting biology, chemistry in order to assess activity and safety in multiple preclinical models.”

Insilico said in a blog that the AI-powered process took only a small fraction of cost and time for a typical preclinical program which is estimated to be around $430 million out-of-pocket expenses and above $1 billion capitalized, and takes anywhere from three to six years to finalize.

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