The administration of ISM001-055 for the treatment of idiopathic pulmonary fibrosis (IPF) has started in healthy volunteers. 

“We are very pleased to see Insilico Medicine’s first antifibrotic drug candidate entering into the clinic,” Feng Ren, PhD, chief scientific officer of Insilico Medicine said in a press release.

ISM001-055 is a small molecule that inhibits a novel biological target. It has been discovered using the company’s end-to-end artificial intelligence (AI)-powered drug discovery platform.

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“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,” Dr. Ren said.

“We have leveraged our end-to-end AI-powered drug discovery platform, including the usage of generative biology and generative chemistry, to discover novel biological targets and generate novel molecules with drug-like properties.”

Read more about IPF overview

ISM001-055 has already been shown in preclinical studies to significantly improve the activation of myofibroblasts, which contributes to fibrosis.

The microdose trial that is being conducted in Australia aims to characterize the pharmacokinetic profile of intravenously administered ISM001-055 in humans. 

“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,” Alex Zhavoronkov, PhD, founder and chief executive officer of Insilico Medicine said.

“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 Medicine initiates first-in-human study of ISM001-055, a novel drug discovered using Insilico’s proprietary end-to-end artificial intelligence platform. News release. Insilico Medicine; November 30, 2021.