Oxford Drug Design has announced further progress in the development of an innovative cancer therapy, marking a significant milestone for the Oxford-based AI drug discovery company.
In an announcement made on 12 January 2026, the company confirmed it has successfully completed in vivo validation of a potential first-in-class therapeutic approach targeting multiple tumour types.
The work was carried out using Oxford Drug Design’s proprietary generative AI (GenAI) discovery platform.
The results were demonstrated in studies using a genetically engineered mouse model that replicates the earliest mutational events in colorectal cancer.
In these studies, Oxford Drug Design’s lead compound showed statistically significant anti-tumour activity, with efficacy comparable to rapamycin, a benchmark therapy, while showing no detectable signs of toxicity.
Rapamycin and related drugs are known to have limitations, as tumours frequently develop KRAS mutations that reduce treatment effectiveness. Oxford Drug Design’s research suggests a potential solution to this challenge.
In advanced 3D tumour models derived from RAS-driven colorectal cancers, the company’s compound successfully induced cancer cell death in cases where rapamycin failed.
These tumour models reflect the two most aggressive human colorectal cancer subtypes, Consensus Molecular Subtypes 3 and 4, which are associated with poor clinical outcomes. The experimental work was undertaken by the Cancer Research UK Scotland Institute, as part of a previously announced grant-funded collaboration.
The lead molecules are drawn from Oxford Drug Design’s expanding portfolio of proprietary chemical scaffolds, further optimised through in-house structural biology capabilities.
This programme represents the company’s third first-in-class therapeutic effort to achieve clear in vivo validation using its AI-driven discovery platform.
Commenting on the achievement, Dr Paul Finn, Chief Scientific Officer, said:
“We continue to develop this breakthrough programme successfully against major tumours, applying our integrated expertise in generative AI and target biology. A significant milestone has been achieved and our rapidly advancing efforts are now focused on bringing this potential first-in-class treatment into the clinic”.
Professor Sarah Blagden, Oxford University Professor of Experimental Oncology, Lead of the Oxford Cancer Trials Office and Member of the Oxford Drug Design Scientific Advisory Board, added:
“These latest results of Oxford Drug Design are impressive. The relative strength of the lead candidate over rapamycin in mutated colorectal cancers points to broad clinical potential for this exciting, novel therapy. I look forward to further progress as it approaches human trials”
Dr Alan D. Roth, CEO of Oxford Drug Design, commented:
“Together with our CRUK Scotland Institute collaborators, our progress in this innovative approach against major tumours continues to validate the accuracy of our dual discovery platform, now poised to deliver superiority over established treatments in oncology.
“We continue our efforts to benefit patients with this breakthrough programme while building our pipeline with further first-in-class treatments.”
Professor Owen Sansom, Director of the CRUK Scotland Institute, Scientific Director of the CRUK Scotland Centre and Director of the National Mouse Genetic Network, UK, said:
“The collaboration – led by Dr. Valeria Pavet (Head of the Translational Research Collaborations Unit at CRUK SI) and Dr. Andrew Campbell (Research Team Lead, CRUKSI) – with Oxford Drug Design is an excellent example of the synergistic benefits arising from combining excellent academic and commercial expertise to advance a novel therapeutic approach.
“We look forward to continuing working together as the project advances towards the clinic.”
