Insilico Medicine Doses First Patient in Phase II Trial for AI-Designed IBD Therapy

Insilico Medicine has officially dosed the first patient in its Phase IIa clinical trial, known as BETHESDA, to evaluate the safety and efficacy of Garutadustat (ISM5411). This novel, oral drug is being developed as a potential treatment for ulcerative colitis, a form of inflammatory bowel disease (IBD) that impacts millions worldwide. The study is led by Professor Minhu Chen at the First Affiliated Hospital of Sun Yat-sen University and focuses on approximately 80 participants.

Garutadustat represents a significant departure from traditional IBD treatments that often rely on systemic immune suppression. Instead, this therapy acts as a gut-restricted PHD inhibitor designed to promote the repair of the intestinal barrier directly at the site of inflammation. This localized action aims to achieve mucosal healing while avoiding the side effects typically associated with systemic medications.

The discovery of Garutadustat highlights the unprecedented efficiency of generative artificial intelligence in the pharmaceutical sector. Utilizing the proprietary Pharma.AI platform and the Chemistry42 generative engine, Insilico identified the molecule as a preclinical candidate within just 12 months. During this accelerated timeline, only 115 compounds were synthesized and tested, a stark contrast to the thousands of molecules often required in conventional drug discovery.

This clinical milestone follows the publication of the drug's preclinical development in Nature Biotechnology, which detailed the AI-driven workflows used to design its unique structure. Previous Phase I studies conducted in Australia and China have already confirmed the drug's favorable safety profile and its ability to remain concentrated within the gastrointestinal tract. The transition into Phase II arrives during a period of rapid growth for the company, which recently listed on the Main Board of the Hong Kong Stock Exchange. As the BETHESDA trial progresses, researchers will monitor clinical remission, endoscopic improvement, and biomarker responses to determine if the AI-identified mechanism translates into meaningful benefits for patients.