How successful are AI-discovered drugs in clinical trials? A first analysis and emerging lessons

Drug Discov Today. 2024 Jun;29(6):104009. doi: 10.1016/j.drudis.2024.104009. Epub 2024 Apr 30.

Abstract

AI techniques are making inroads into the field of drug discovery. As a result, a growing number of drugs and vaccines have been discovered using AI. However, questions remain about the success of these molecules in clinical trials. To address these questions, we conducted a first analysis of the clinical pipelines of AI-native Biotech companies. In Phase I we find AI-discovered molecules have an 80-90% success rate, substantially higher than historic industry averages. This suggests, we argue, that AI is highly capable of designing or identifying molecules with drug-like properties. In Phase II the success rate is ∼40%, albeit on a limited sample size, comparable to historic industry averages. Our findings highlight early signs of the clinical potential of AI-discovered molecules.

Keywords: AI drug discovery; AI drug in clinical trials; AI-discovered drug; AI-native Biotech; Clinical trial success rate.

Publication types

  • Review

MeSH terms

  • Artificial Intelligence*
  • Clinical Trials as Topic* / methods
  • Drug Discovery* / methods
  • Drug Industry
  • Humans