AI drug discovery company Oxford Drug Design has successfully completed in vivo validation of a novel therapeutic approach targeting multiple tumour types.

In studies using a genetically engineered mouse model that replicates the earliest mutational events in colorectal cancer, Oxford Drug Design’s lead compound demonstrated statistically significant anti-tumour activity with efficacy comparable to that of rapamycin – a benchmark therapy – while showing no detectable signs of toxicity.

Oxford Drug Design’s studies showcased a way to get around well-known limitations of rapamycin and similar drugs, in which tumours acquire KRAS mutations that render these therapies ineffective.

In advanced 3D tumour models derived from RAS-driven colorectal cancers, the company’s compound induced cancer cell death in settings where rapamycin failed.

These models reflect the two most aggressive human colorectal cancer subtypes (Consensus Molecular Subtypes 3 and 4) that carry KRAS mutations associated with poor clinical outcomes.

This programme marks the company’s third first-in-class therapeutic effort to achieve clear in vivo validation using its AI-driven discovery platform.

Oxford Drug Design is now planning to extend the same differentiated capabilities into additional disease areas with significant unmet medical need.

Dr 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”.