Using AI/ML Tools to Translate from Target ID to the Clinic

Irene Choi, the head of drug discovery at Verge Genomics, presented on the application of AI/ML in drug discovery, focusing on the Converge platform and the PIKfyve programme. With over 15 years of experience in neuroscience drug discovery, Irene led the PIKfyve programme to a successful phase one trial and had previously advanced multiple drugs to the clinic at Nektar Therapeutics. 

The Converge platform is a fully integrated drug discovery platform that uses all-in-human data to identify novel targets. It incorporates various types of data, both public and proprietary, to improve drug discovery. The platform's foundation is built on over 61 terabytes of human data, which is used to translate findings into preclinical models and clinical studies. 

Choi highlighted the PIKfyve small molecule inhibitor programme, which was in clinical trials for treating ALS (Amyotrophic Lateral Sclerosis). The programme used preclinical models with a disease signature to identify models that replicated the disease signature, demonstrating rescue in both patient-derived cell lines and animal models. The programme advanced from target identification to phase one in under five years, showcasing the efficiency of the Converge platform. 

The presentation also discussed the broader application of the Converge platform in other neurodegenerative diseases, including Parkinson's disease, frontal temporal dementia, and schizophrenia. Verge Genomics had established partnerships with major pharmaceutical companies like Lily and Alexion to support their drug discovery efforts. 

Choi emphasised the importance of leveraging human data and AI/ML technologies to improve drug discovery. The Converge platform's closed loop of data generation allowed for continuous improvement and accuracy over time. The platform's ability to identify and validate targets in human model systems and clinical studies was a key factor in its success. 

Choi's presentation demonstrated the significant impact of AI/ML technologies in drug discovery, particularly through the Converge platform and the PIKfyve programme. The integration of human data and the collaboration with pharmaceutical partners were crucial elements in advancing their drug discovery efforts.