Thiogenesis Receives Confirmatory Guidance on IMPD for Pediatric MASH Phase 2a Clinical Trial in Europe and Core Patent Granted in EU

Core Insights - Thiogenesis Therapeutics has received scientific advice from the European Medicines Agency (EMA) supporting its submission for an Investigational Medicinal Product Dossier (IMPD) for a Phase 2a clinical trial in pediatric patients with metabolic dysfunction-associated steatohepatitis (MASH) [1][2] - The trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of TTI-0102 in children aged 10 to 17 with biopsy-confirmed MASH [2] - Thiogenesis has also been granted a European Union patent for its asymmetric disulfide prodrugs of cysteamine, which secures its competitive position in mitochondrial and metabolic disease indications until 2038 [3] Company Overview - Thiogenesis Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California, focusing on developing thiol-based therapies for mitochondrial diseases and pediatric metabolic conditions [8] - The company is publicly traded on the TSX Venture Exchange and OTCQX, with its lead product candidate, TTI-0102, currently in clinical trials for various serious pediatric conditions [8] Product Details - TTI-0102 is a third-generation disulfide prodrug of cysteamine, designed to address challenges associated with earlier thiol-based drugs, including short half-life and adverse side effects [5][6] - The prodrug aims to enhance the bioavailability of cysteamine, which is crucial for producing antioxidants and restoring mitochondrial function [6][7] Industry Context - Pediatric MASH is a severe form of fatty liver disease affecting approximately 7-14% of children and adolescents in the EU, translating to 5-10 million patients [4] - The condition is characterized by liver inflammation and damage due to fat accumulation, with oxidative stress playing a significant role in its progression [4]