Recently, the 4B06-01 project with AI-empowerment from target discovery to the whole process of clinical trials has successfully enrolled all 64 patients. This project is a Proof-of-Concept Trial for the treatment of amyotrophic lateral sclerosis (ALS) conducted in Peking University Third Hospital. The double-blind dosing phase is expected to be completed in August 2024, and the one-year clinical treatment observation is scheduled to be completed in February 2025.

This AI-assisted Investigator-Initiated Trial (IIT) was conceptualized by Professor Lu Bai of Tsinghua University, and led by Professor Fan Dongsheng, a renowned ALS clinical expert from Peking University Third Hospital as the Principal Investigator, in close collaboration with multiple partners including 4B Technologies, Insilico Medicine, Gnosis Healthineer, and Beijing Academy of Artificial Intelligence (BAAI) among others. By innovative application of AI technology in trial design such as patient selection and drug efficacy evaluation, this approach not only significantly shortened patient recruitment time, achieving precise screening and enrollment of all patients within 1 year, but also maximizes the utilization of the clinical data from participants to address the challenge of insufficient sample size for rare diseases.

4B06-01, based on drug “repurposing” principle, represents an example of accelerated precision medicine development program enabled by AI technologies and collaborative partnerships.  During the course of progression, Insilico Medicine applied PandaOmics algorithm to screen  publicly available international databases and discovered novel potential  targets for ALS; 4B Technologies leveraged its expertise and capability in translational and clinical medicine in neurological diseases to complete the target identification and candidate drug selection, rapidly enabling initiation of the IIT; Gnosis Healthineer and BAAI developed EEG AI analysis methods to support precise patient selection for ALS; the team of Peking University Third Hospital integrated multiple resources by combining traditional clinical research methods with innovative approaches, and incorporating various AI techniques to observe disease progression and evaluate drug efficacy, thereby effectively advancing the trial.

This collaborative effort explores a pioneering model for drug development, aiming to enhance the success rate of ALS clinical trials and reduce R&D costs.