AI startup’s drug breakthrough targets lung disease treatment

Artificial intelligence has reached a milestone in drug development as Insilico Medicine announces the first successful mid-stage clinical trial of an AI-designed drug. The company’s experimental treatment for idiopathic pulmonary fibrosis—a deadly lung disease with limited treatment options—demonstrated improved lung function in patients receiving the highest dose. This breakthrough represents a significant advancement in AI-driven pharmaceutical innovation, potentially reducing drug development timelines from years to months.

The big picture: Insilico Medicine’s drug rentosertib showed encouraging results in treating an incurable lung disease, marking what the company calls the first time an AI-created drug targeting an AI-identified biological pathway has succeeded in a mid-stage clinical study.

• The highest dose of rentosertib improved lung function in patients with idiopathic pulmonary fibrosis, a condition characterized by lung scarring that progressively impairs breathing.
• Biomarker analysis confirmed the drug worked as predicted by the AI system, specifically reducing a protein that artificial intelligence had hypothesized was linked to the disease.

Why this matters: The results validate AI’s potential to dramatically accelerate drug discovery, with Insilico claiming it reduced the typical timeline from 4.5 years to just 18 months for developing a clinical-ready drug candidate.

• CEO Alex Zhavoronkov described the findings as “one of the best results people have ever seen” for this lung condition.
• The breakthrough comes amid billions in investment from pharmaceutical companies and startups betting on AI’s ability to make drug development more efficient.

Behind the numbers: The study included only 71 patients, all from China, and ran for a relatively short 12 weeks, raising questions about the robustness of the findings.

• Some patients taking the drug experienced worsening conditions, while several others developed liver injury that researchers acknowledged will require monitoring in future trials.
• Zhavoronkov maintains the side effects are manageable and the drug is “reasonably safe” given the serious nature of the disease it treats.

Looking ahead: Despite promising initial results, rentosertib still faces a lengthy path to potential regulatory approval.

• The company is formulating plans for larger trials needed to substantiate the drug’s efficacy, with approval at least two years away under the best-case scenario.
• Insilico is currently running a parallel mid-stage study in the United States and plans to continue clinical trials in both China and the US.
• The company, which filed to list in Hong Kong, views the data as a demonstration of its broader AI drug discovery platform, which includes more than 30 programs.