Thiogenesis Reports Positive Interim Phase 2 Trial Results for MELAS and Announces Pipeline Advancements in Leigh Syndrome and Cystinosis

Core Insights - Thiogenesis Therapeutics reported positive interim results from its Phase 2 clinical trial of TTI-0102 for MELAS, achieving biological proof-of-concept, dose discovery, and biomarker improvement [1][2][5] MELAS Clinical Trial - The Phase 2 trial included nine patients, with six receiving TTI-0102 and three on placebo, focusing on biological proof-of-concept, dose discovery, and early efficacy [2] - Five active patients remain in the trial, with TTI-0102 well tolerated, although four patients under 50 kg left due to dose-dependent side effects, indicating a need for refined dosing protocols [3] - The interim analysis showed significant support for TTI-0102's mitochondrial antioxidant and restorative activity, with individual biomarker data remaining confidential until early 2026 [4] Future Plans and Pipeline Updates - The company anticipates final 6-month data from the MELAS cohort by January 2026 and plans to file an Investigational Medicinal Product Dossier (IMPD) to initiate a pivotal Phase 3 trial in Europe in 2026 [6][7] - For Leigh syndrome spectrum, the FDA accepted the IND application, with a Phase 2 trial expected to start in early 2026, incorporating dosing adjustments based on MELAS interim data [8] - The company plans to file an IND application for a Phase 3 trial in nephropathic cystinosis, comparing TTI-0102 to current cysteamine therapies, which have significant side effects [9][10] About TTI-0102 - TTI-0102 is a disulfide compound designed to overcome limitations of existing thiol-based drugs, offering potential for once-daily dosing and improved tolerability [15] - The compound targets mitochondrial dysfunction, with applications in MELAS and other pediatric diseases [16]