After the successful completion of a phase 0 microdose trial, ISM001-055, an antifibrotic small molecule inhibitor with potential application in idiopathic pulmonary fibrosis (IPF), has initiated its clinical evaluation journey in humans.

The announcement was recently made by the artificial intelligence (AI)-driven drug discovery company Insilico Medicine.

“We have completed our first human in phase 0 clinical trial and entered the full phase I clinical trial with an 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,” Alex Zhavoronkov, PhD, founder and chief executive officer of Insilico, said in a press release.

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Results from the phase 0 exploratory microdose trial, which enrolled 8 healthy volunteers, identified a microdose of ISM001-055 with a favorable pharmacokinetic and safety profile.

Read more about IPF experimental therapies

The phase 1 trial (NCT05154240) will enroll 80 healthy volunteers distributed into 10 cohorts: 5 with single ascending doses and 5 with multiple ascending doses. The aim of this double-blind, placebo-controlled, single and multiple ascending dose trial is to evaluate the safety, tolerability, and pharmacokinetics of ISM001-055.

The primary endpoints will be essential to determine the maximum tolerated dose of ISM001-055 and establish dosage recommendations for phase 2 studies.

“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, PhD, chief science officer of Insilico Medicine, stated.

ISM001-055 was developed against a novel target discovered by a target identification engine. The mechanism of action of ISM001-055 in fibrosis is also relatively new.

“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,” Michael Levitt, PhD, 2013 Nobel laureate in chemistry and member of Insilico Medicine’s Scientific Advisory Board, said.


AI-discovered anti-fibrotic product candidate enters phase 1 human clinical trial. News release. Insilico Medicine; February 24, 2022.

A phase 1, evaluate the safety, tolerability, and pharmacokinetics of INS018_055 in healthy subjects. December 13, 2021. Updated February 10, 2022. Accessed February 28, 2022.