Oxford Drug Design has announced new in vivo results that further validate its emerging first‑in‑class oncology programme, marking a significant step forward for the Oxford-based AI drug discovery company.
Using its GenAI platform, the company has completed in vivo validation of a novel therapeutic mechanism designed to target multiple tumour types.
In a genetically engineered mouse model replicating the earliest mutational events in colorectal cancer, its lead compound showed statistically significant anti‑tumour activity with efficacy comparable to rapamycin, a benchmark therapy, while demonstrating no detectable toxicity.
Rapamycin and related drugs are often limited by the emergence of KRAS mutations that render them ineffective.
Oxford Drug Design’s findings indicate a potential way to bypass this challenge. In advanced 3D tumour models derived from RAS‑driven colorectal cancers, the company’s compound induced cancer cell death in scenarios where rapamycin failed. These models represent two of the most aggressive human colorectal cancer subtypes that carry KRAS mutations associated with poor clinical outcomes.
The work was conducted by the Cancer Research UK Scotland Institute as part of an ongoing grant‑funded collaboration. The lead molecules originate from Oxford Drug Design’s proprietary chemical scaffolds, refined through its in‑house structural biology capabilities. This marks the company’s third first‑in‑class programme to achieve clear in vivo validation using its AI‑driven platform.
Paul Finn, CSO of Oxford Drug Design, 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 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”.
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 said: “The collaboration – led by Dr Valeria Pavet and Dr Andrew Campbell – 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.”
