Insilico Medicine's AI-Designed TEAD Inhibitor Receives IND Approval and Orphan Drug Designation from FDA

Insilico Medicine, a clinical-stage biotechnology company specializing in generative AI-driven drug discovery, has announced that its novel compound, ISM6331, has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA). This approval follows the FDA's earlier grant of Orphan Drug Designation (ODD) to ISM6331 for the treatment of mesothelioma, a rare and aggressive cancer primarily caused by asbestos exposure.

ISM6331 is a potent non-covalent small molecule inhibitor targeting the TEAD protein family, key regulators within the Hippo signaling pathway, which is implicated in tumor progression, metastasis, and drug resistance.

The development of ISM6331 was significantly accelerated by Insilico's AI-powered generative chemistry engine, Chemistry42. The AI system rapidly generated three promising series of compounds, which were further optimized to produce ISM6331. Preclinical studies have demonstrated the compound's broad anti-tumor activity, high safety margin, and favorable pharmacokinetic properties.

See also: Insilico Medicine’s Generative AI Patent Provides Advantage in AI Drug Discovery Race

The FDA's Orphan Drug Designation grants ISM6331 various benefits, including seven years of market exclusivity upon approval, potential tax credits, and eligibility for specific grants, all aimed at fostering the development of treatments for rare diseases affecting fewer than 200,000 people in the United States.

With the IND clearance, Insilico Medicine plans to initiate clinical trials in the U.S., targeting mesothelioma and other cancers associated with abnormalities in the Hippo pathway. The company continues to leverage its Pharma.AI platform, which integrates AI across biology, chemistry, and clinical development, to expedite the discovery and development of novel therapeutics. ISM6331 is one of nine molecules in Insilico's pipeline to have received IND approval.

Topics: AI & Digital