AI drug discovery in 2026: from first phase III trials to cancer vaccines
Developing a new medicine traditionally takes 10 to 15 years, and around nine out of ten candidates that enter clinical trials fail. Artificial intelligence is being applied to every stage of this process: identifying biological targets, designing molecules, predicting toxicity, selecting patients and personalising treatment. In 2026, two programmes provided the clearest clinical evidence yet of what this approach can achieve.
How AI discovers drugs
AI drug discovery typically proceeds in two stages. First, target discovery: models analyse genomic, proteomic and clinical data to identify proteins that drive a disease. Second, generative chemistry: models trained on known molecules and their properties propose new chemical structures predicted to bind the target, be safe and be manufacturable. Promising candidates are synthesised and tested in the laboratory, and the results feed back into the models. This cycle can compress years of medicinal chemistry into months.
Rentosertib: an AI-discovered drug in phase III
Insilico Medicine used its generative AI platform both to identify the target, the kinase TNIK, and to design the molecule rentosertib for idiopathic pulmonary fibrosis (IPF), a progressive scarring of the lungs. In a phase IIa study, patients receiving 60 mg once daily showed a mean improvement in forced vital capacity (FVC) of 98.4 mL after 12 weeks, compared with a mean decline of 20.3 mL on placebo. In 2026 the company launched the phase III GENESIS-IPF-3 trial, a randomised, double-blind, placebo-controlled study of 320 patients at 47 centres over 52 weeks, and dosed the first patient in September 2026 (News-Medical, 2026; Bio-IT World, 2026).
Personalised mRNA cancer vaccines
Every tumour carries mutations that produce abnormal proteins, called neoantigens, which the immune system can learn to recognise. A personalised cancer vaccine is designed for a single patient: the tumour and healthy tissue are sequenced, algorithms identify and rank the neoantigens most likely to trigger an immune response, and an mRNA vaccine encoding them is manufactured for that patient. Moderna's intismeran autogene encodes up to 34 neoantigens.
In August 2026, Moderna and Merck announced that the phase III INTerpath-001 trial of intismeran autogene combined with the immunotherapy Keytruda met its primary endpoint of recurrence-free survival and its key secondary endpoint of distant metastasis-free survival in 1,137 patients with resected high-risk melanoma (BioPharm International, 2026). Five-year data from the earlier phase IIb study showed a 49% reduction in the risk of recurrence or death (hazard ratio 0.51).
AI across the medicine lifecycle
- Vaccination programmes: forecasting demand, optimising cold-chain logistics and identifying under-vaccinated populations.
- Medicine assistants: explaining prescriptions, checking interactions and supporting adherence for patients.
- Clinical trials: matching patients to trials and predicting enrolment and outcomes.
Brand opportunity: biotech and health domains
AiDrug.ai is a category-defining name for an AI drug discovery company or platform. CancerVaccines.ai fits a biotech, a patient information service or an investment platform focused on personalised oncology. MyDrug.ai suits a personal medicine assistant, Immunization.ai a vaccination intelligence service, and IVG.ai a short brand for a biotech or health technology company.
Read also about AI health assistants for patients. This article is for information only and is not medical advice.
References
- News-Medical (2026). Generative AI-driven drug rentosertib enters phase III trial for IPF.
- Bio-IT World (2026). Insilico Medicine launches phase III trial for AI-developed drug.
- Insilico Medicine (2026). First patient dosed in GENESIS-IPF-3.
- BioPharm International (2026). Merck and Moderna intismeran autogene phase 3 melanoma results.
- Practical Dermatology (2026). INTerpath-001 meets dual phase 3 melanoma endpoints.
Frequently asked questions
Has an AI-discovered drug reached phase III?
Yes. Insilico Medicine’s rentosertib, whose target and molecule were both found with generative AI, entered the phase III GENESIS-IPF-3 trial for idiopathic pulmonary fibrosis in 2026.
What is a personalised cancer vaccine?
A vaccine designed for one patient: the tumour is sequenced, algorithms select its most immunogenic neoantigens, and an mRNA vaccine encoding them trains the immune system to attack the cancer.
Is AiDrug.ai for sale?
Yes. AiDrug.ai, CancerVaccines.ai, MyDrug.ai, Immunization.ai and IVG.ai are for sale on 4ai by offer.
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