Insilico’s AI-designed molecule enters Phase 2 testing

Clinical trial of oral INS018_055 is running in parallel in the US and China

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Insilico Medicine made its entry into Phase 2 clinical testing with INS018_055, a small molecule it discovered and designed using artificial intelligence (AI), as a potential treatment for idiopathic pulmonary fibrosis (IPF).

Patients have now received the first dose of oral INS018_055 in a randomized placebo-controlled Phase 2 trial running in parallel in the U.S. and China.

This “is not only an important step for Insilico, but also a milestone for AI-driven drug discovery and development,” Feng Ren, PhD, Insilico’s chief scientific officer and co-CEO, said in a press release.

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The trial is designed to test how safe and well tolerated INS018_055 is when taken for 12 weeks (about three months). They’re also testing its pharmacokinetics (how it moves within the body) and preliminary efficacy in four groups of patients.

The company plans to enroll an additional 60 patients from multiple locations across the U.S. and China to test the small molecule in a larger population.

INS018_055, which interacts with a yet-undisclosed target, is expected to act as an anti-fibrotic, or a therapy that slows the rate of tissue scarring (fibrosis) in the lungs, which is a hallmark of pulmonary fibrosis.

“With demonstrated potential against both fibrosis and inflammation, INS018_055 could offer another option for patients worldwide,” Ren said.

The small molecule was discovered with the company’s Pharma.AI platform, which uses PandaOmics algorithms to select a disease target from a large pool of data. It then uses Chemistry24 to design a small molecule that may interact with the selected target.

The safety and pharmacological properties of single and multiple-ascending doses of INS018_055 were tested in a previous Phase 1 trial (NCT05154240) in New Zealand involving 78 healthy volunteers.

All side effects related to treatment were mild and went away by the trial’s end. INS018_055 also was well tolerated. An additional group of 48 healthy volunteers entered another Phase 1 trial in China, according to the company.

Insilico: ‘Validation of our generative AI platform’

“When we started developing generative AI for drug discovery, I never expected to see the clinical and preclinical results we have today,” said Alex Zhavoronkov, PhD, Insilico’s founder and co-CEO. “We will explore the efficacy for patients of AI-discovered and designed treatments in clinical trials, which is a true validation of our generative AI platform.”

Earlier this year, the U.S. Food and Drug Administration granted INS018_055 orphan drug designation for the treatment of IPF, which helped to support its move into Phase 2 testing.

“We are eager to continue to advance this potentially first-in-class therapy forward to help patients in need and show the value of generative AI in drug discovery and development,” Zhavoronkov said.

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