AI-Discovered Drug Reverses Aging Markers in Study, Biotech Says

An experimental lung disease drug developed using artificial intelligence showed promise in reversing biological signs of aging, pointing to broader potential use of the treatment, according to its developer.

In a mid-stage study, Insilico Medicine’s drug rentosertib helped reduce biological age as measured by six different aging clocks examining chemical changes in the body, according to an analysis published in Nature Biotechnology on Monday. Those who were given a placebo saw little change in their biological age on those same clocks, the Hong Kong-listed company said.

“If your biological age is lower, you are likely to die later,” the AI drug discovery startup’s Chief Executive Officer, Alex Zhavoronkov, said in an interview. He cautioned that the results are still early and the long-term health effects of the drug on larger groups of people are unknown. The analysis included 42 patients with a chronic lung disease.

Aging clocks are tools that estimate someone’s biological age, as opposed to chronological age, reflecting how well their cells and tissues are functioning. The six aging clocks used in the study — developed by teams at Insilico, Harvard University, China’s Peking University and others — each use different metrics and methods.

While longevity enthusiasts have long embraced the repurposing of existing medications to delay aging, efforts to develop novel anti-aging drugs have often ended in failure. But with the advent of GLP-1 treatments whose benefits now appear to extend far beyond weight loss and diabetes, major pharmaceutical players are increasingly showing interest in the field.

Companies including Insilico have chosen to focus on dual-use therapies that can tackle age-related ailments while helping people live longer along the way. The latest study is part of a Phase 2 trial that also measured rentosertib’s efficacy in treating idiopathic pulmonary fibrosis. Rentosertib is currently in a late-stage clinical trial in China for the incurable lung disease.

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Insilico plans to run similar analyses for at least half of the drugs in its pipeline.

AI promises to compress drug research timelines, increase chances of success and propose ideas humans may not otherwise have. Insilico first identified the target behind rentosertib as one implicated in aging with an AI tool that trawls through large amounts of data to rank potential targets. It then designed and refined the drug using another that screens molecules virtually and suggest ways to optimize them.

For its analysis, Insilico collected blood from patients at various time points during the trial and partnered with external researchers to analyze changes in proteins. Four weeks after the initial dose, patients taking rentosertib saw their average biological age dropped by as much as six years, although the benefit narrowed later in the trial.

The study offers a template for future anti-aging drug development, Zhavoronkov said. While it’s currently impossible to win regulatory approval specifically for aging, he suggested the results would open the door for doctors to prescribe or test rentosertib for aging — or age-related diseases — once the drug is approved for other uses.