Insilico Medicine ("Insilico"), an end-to-end artificial intelligence (AI)-driven drug discovery company, today announced that it has dosed multiple healthy volunteers in Phase I clinical trial evaluating ISM001-055, the first anti-fibrotic small molecule inhibitor generated by Insilico's AI-powered drug discovery platform for idiopathic pulmonary fibrosis (IPF).

The double-blind, placebo controlled single and multiple ascending dose Phase I clinical trial will evaluate the safety, tolerability, and pharmacokinetics of ISM001-055. Eighty (80) healthy volunteers will be enrolled in ten (10) cohorts comprising of five (5) single ascending doses and five (5) multiple ascending dose cohorts. The primary endpoints determine the maximum tolerated dose and establish dosage recommendations for potential Phase II clinical trials in the future.

It is a great pleasure to see our AI-discovered novel molecule with a novel mechanism enters the Phase I clinical trial.  We are committed to taking advantage of AI-driven drug discovery to rapidly advance the development of innovative therapeutics for unmet medical needs, especially in the areas of fibrosis, oncology, immunology, and neurology, and initiating this phase I study demonstrates the power of our AI platform in achieving that."

Feng Ren, Ph.D., Chief Science Officer of Insilico Medicine

In order to better understand compound distribution, establish a dose, and provide insights into ISM001-055's safety profile in humans, Insilico initiated a Phase 0 exploratory microdose trial of ISM001-055 in November 2021, which was conducted in Australia and included eight (8) healthy volunteers. The results identified a microdose of ISM001-055 with a favorable pharmacokinetics and safety profile.

"There are groups who try to do target discovery and groups who do chemistry, but Insilico goes end-to-end." said Michael Levitt, 2013 Nobel Laureate in Chemistry and member of Insilico Medicine's Scientific Advisory Board. "The best way to validate the performance of an AI system is to conduct comprehensive testing with novel targets and novel molecules. Human safety testing is crucial. I am delighted that Insilico's AI-powered, end-to-end anti-fibrotic program just passed microdose study in humans and has entered Phase I."

ISM001-055 is a small molecule inhibitor against a novel target discovered by target identification engine PandaOmics, and with a molecule structure designed by small molecule generation engine Chemistry42. The mechanism of action for the target is also relatively novel in fibrosis. Both PandaOmics and Chemistry42 are parts of Pharma.AI, Insilico's end-to-end AI-driven drug discovery and development platform.

"Modern deep learning technologies enable us to perform target identification using longitudinal biological data from healthy subjects and make inferences into a variety of diseases. This was the guiding principle for our anti-fibrotic program starting with identifying targets that may play a role in both aging and disease. It gives me great pleasure to announce that we have completed our first human in Phase 0 clinical trial and entered the full Phase I clinical trial with a once-a-day oral dose of our ISM001-055 anti-fibrotic where the target was identified using AI and the molecule was designed using AI, meaning it is AI-discovered and AI-designed", said Alex Zhavoronkov, PhD., Founder and CEO of Insilico Medicine.