Prediction of Clinical Trials Outcomes Based on Target Choice and Clinical Trial Design with Multi‐Modal Artificial Intelligence

Abstract

Drug discovery and development is a notoriously risky process with high failure rates at every stage, including disease modeling, target discovery, hit discovery, lead optimization, preclinical development, human safety, and efficacy studies. Accurate prediction of clinical trial outcomes may help significantly improve the efficiency of this process by prioritizing therapeutic programs that are more likely to succeed in clinical trials and ultimately benefit patients. Here, we describe inClinico, a transformer‐based artificial intelligence software platform designed to predict the outcome of phase II clinical trials. The platform combines an ensemble of clinical trial outcome prediction engines that leverage generative artificial intelligence and multimodal data, including omics, text, clinical trial design, and small molecule properties. inClinico was validated in retrospective, quasi‐prospective, and prospective validation studies internally and with pharmaceutical companies and financial institutions. The platform achieved 0.88 receiver operating characteristic area under the curve in predicting the phase II to phase III transition on a quasi‐prospective validation dataset. The first prospective predictions were made and placed on date‐stamped preprint servers in 2016. To validate our model in a real‐world setting, we published forecasted outcomes for several phase II clinical trials achieving 79% accuracy for the trials that have read out. We also present an investment application of inClinico using date stamped virtual trading portfolio demonstrating 35% 9‐month return on investment.