Thiogenesis Initiates Phase 2 Clinical Trial in Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes ("MELAS")

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]