Insilico's AI-discovered, AI-designed IPF drug enters Phase 1 trials

AI, big data and real world evidence – the challenges and opportunities

Hong Kong-based Insilico Medicine has begun a Phase 1 trial of its AI-discovered and AI-designed anti-fibrotic small molecule inhibitor. 

Currently referred to as ISM001-055, the drug is meant to treat ideopathic pulmonary fibrosis using a relatively novel mechanism of action.

Insilico announced in February of last year that they had identified and developed the molecule in less than 18 months, using the company's target identification engine PandaOmics and its molecular generation engine Chemistry42. These platforms promise to use AI to greatly accelerate the timetable of drug discovery and development, and it's an area investors are quite bullish on.

“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," founder and CEO Alex Zhavoronkov said in a statement. "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.”

The drug entered Phase 0 human trials in late November, an exploratory microdose trial of eight individuals in Australia. That trial, conducted to establish a dose for the drug and assess safety, has now concluded.

The new Phase 1 trial will be a double-blind placebo trial and will include 80 healthy volunteers divided into 10 cohorts. The purpose of the trial will be to determine maximum tolerated dosage and establish dosage recommendations for future trials.

"It is a great pleasure to see our AI-discovered novel molecule with a novel mechanism enter the Phase I clinical trial." Feng Ren, chief science officer of Insilico Medicine, said in a statement. "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."

Insilico has been a major player in the AI drug discovery and development space for a few years ago, making news in 2019 for a Nature Biotechnology paper detailing how the team developed, synthesise, and preclinically validate a novel drug candidate in 46 days.

The company raised a $225 million Series C round last summer led by Warburg Pincus. In addition to developing its own drugs, the company also works with big pharma companies including Pfizer, Johnson & Johnson, and Teva.