How successful are AI-discovered drugs in clinical trials? A first analysis and emerging lessons
Open accessAbstract
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.
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AI-discovered drugs have experienced similar levels of phase 2 trial failure as non-AI-discovered drugs.
“AI-discovered drugs have experienced similar levels of phase 2 trial failure as non-AI-discovered drugs20,21”
From A generative AI-discovered TNIK inhibitor for idiopathic pulmonary fibrosis: a randomized phase 2a trial (2025) · cites this paper as [21]
Few novel AI-discovered or AI-designed drugs have reached human clinical trials.
“Despite substantial progress in artificial intelligence (AI) for generative chemistry, few novel AI-discovered or AI-designed drugs have reached human clinical trials.”
From A generative AI-discovered TNIK inhibitor for idiopathic pulmonary fibrosis: a randomized phase 2a trial (2025) · cites this paper as [21]
No AI-discovered drug has so far progressed through phase 3 trials.
“none has so far progressed through phase 3 trials21.”
From A generative AI-discovered TNIK inhibitor for idiopathic pulmonary fibrosis: a randomized phase 2a trial (2025) · cites this paper as [21]
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GenAI and the Future of Creativity in Science and ArtPreprint
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Clinical trials gain intelligence
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- Authors
- KP Jayatunga, M., Ayers, M., Bruens, L., Jayanth, D., & Meier, C.
- Affiliations
- Boston Consulting Group (United States) (US)
- Corresponding author
- Meier, Christoph
- ORCID
- Meier, Christoph
- Venue
- Drug Discovery Today
- Publisher
- Elsevier BV
- Published
- 2024-06
- Volume
- 29
- Issue
- 6
- Pages
- 104009
- ISSN
- 1359-6446 (ISSN-L)
- OpenAlex
- W4396521393
- Cited by
- 166
- References
- 20
- Citations by year
- 2026: 71 · 2025: 76 · 2024: 18 · 2023: 1
- FWCI
- 34.98
- Type
- journal article
- Language
- en
- Open access
- hybrid
- www.sciencedirect.com
- Licence
- Locations
- Drug Discovery Today (publishedVersion, cc-by-nc-nd)
- PubMed (publishedVersion)
- Topics
- Physical Sciences › Computer Science › Computational Theory and Mathematics › Computational Drug Discovery Methods
- Health Sciences › Medicine › Health Informatics › Artificial Intelligence in Healthcare and Education
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Protein Degradation and Inhibitors
- Keywords
- drug discovery, clinical trial success rates, drug-likeness, machine learning in drug discovery
- MeSH terms
- Artificial Intelligence, Clinical Trials as Topic, Drug Industry, Humans, Drug Discovery
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