Insilico Medicine, a China-based biotechnology company at the forefront of generative artificial intelligence (AI), has announced that it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its pipeline candidate ISM6331. This potential best-in-class candidate drug is designed to treat mesothelioma, a rare and aggressive form of cancer.
Mesothelioma, primarily caused by exposure to asbestos, is known for its propensity to spread to the surfaces of organs, nerves, and blood vessels within the body. The median survival time after diagnosis is a stark 9 to 12 months. Current traditional treatment methods, including surgery and radiotherapy, offer limited long-term benefits, highlighting an urgent need for innovative therapies for mesothelioma patients.
ISM6331 is a potent non-covalent small molecule inhibitor with a novel molecular structure. It was discovered and optimized using Insilico Medicine’s proprietary chemical generation engine, Chemistry42, and targets the transcription enhancing related domain (TEAD) protein family. TEAD, a key regulatory factor of the Hippo pathway, plays a significant role in tumor progression, metastasis, cancer metabolism, immunity, drug resistance, and other critical processes. Preclinical studies have demonstrated that ISM6331 exhibits broad-spectrum anti-tumor effects across multiple cell lines, is effective at low doses in animal models, and possesses favorable safety and ADMET (Absorption, Distribution, Metabolism, Excretion, and Toxicity) profiles.- Flcube.com
