Thiogenesis Announces Second Site Begins Enrolling in Phase 2 MELAS Clinical Trial and Provides Update

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]