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]

Core Points - Thiogenesis Therapeutics has activated a second clinical site in France for its Phase 2 clinical trial of TTI-0102, targeting MELAS patients [1][2] - The trial is enrolling 12 patients, with 8 receiving TTI-0102 and 4 receiving a placebo, and is set to conduct a three-month interim analysis in September 2025 [3][4] - Key clinical endpoints include the 12-Minute Walk Test, Fatigue Severity Scale, and WHOQOL-BREF Quality of Life assessment [4] Company Overview - Thiogenesis Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, focusing on sulfur-based therapeutics for pediatric and inherited mitochondrial diseases [9] - The lead product candidate, TTI-0102, is designed to improve upon first-generation thiol-based drugs by addressing issues like short half-life and side effects [6][7] - The company is publicly traded on the TSX Venture Exchange and OTCQX, and is also planning trials for other conditions such as Leigh syndrome and Rett syndrome [9] Disease Background - MELAS is an inherited mitochondrial disorder, often caused by a mutation in the MT-TL1 gene, with symptoms including seizures, muscle weakness, and fatigue [5] - It is estimated that approximately 4.1 out of 100,000 people worldwide are affected by MELAS [5] - The disease typically presents before the age of 20 and is considered an orphan disease despite its prevalence [5] Product Information - TTI-0102 is an asymmetric disulfide prodrug that acts as a precursor to cysteamine, a thiol compound involved in key biochemical reactions [6][7] - Prodrugs like TTI-0102 can enhance the bioavailability of active ingredients and reduce side effects, facilitating regulatory approval processes [8]

Core Insights - Thiogenesis Therapeutics has initiated a Phase 2 clinical trial for TTI-0102, targeting the treatment of MELAS, an inherited mitochondrial disease [1][3] - The trial aims to assess the safety, tolerability, efficacy, and pharmacokinetics of TTI-0102 over a 6-month period, involving 12 patients [2][3] - TTI-0102 is designed to increase intracellular antioxidants and amino acids, addressing deficiencies in MELAS patients [3][8] Company Overview - Thiogenesis Therapeutics is a clinical-stage biotechnology company based in San Diego, focusing on developing disulfides that enhance intracellular antioxidant production [1][10] - The company is publicly traded on the TSX Venture Exchange and OTCQX, with a focus on treating serious pediatric diseases with unmet medical needs [10] Clinical Trial Details - The Phase 2 trial is a multi-country, multi-center study conducted in the Netherlands and France, with a randomized, double-blind, placebo-controlled design [2] - A total of 12 patients will be enrolled, with 8 receiving TTI-0102 and 4 receiving a placebo, followed by an interim analysis after 3 months [2] Disease Background - MELAS is characterized by symptoms such as seizures, muscle weakness, and fatigue, often presenting before the age of 20, and is caused by mitochondrial DNA mutations [4] - The disease affects approximately 4.1 per 100,000 of the population, making it one of the most prevalent inherited mitochondrial disorders [4] Product Information - TTI-0102 is an asymmetric disulfide and prodrug that acts as a precursor to cysteamine, aimed at restoring mitochondrial function by increasing levels of glutathione and taurine [8] - The prodrug formulation addresses limitations of first-generation thiol-based drugs, including short half-life and adverse side effects [8][9]