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Thiogenesis Reports Positive Interim Phase 2 Trial Results for MELAS and Announces Pipeline Advancements in Leigh Syndrome and Cystinosis
Trial achieved biological proof-of-concept, dose discovery and biomarker improvement San Diego, ...
About this update from Thiogenesis Therapeutics Corp
[{"type":"text","content":"Thiogenesis Reports Positive Interim Phase 2 Trial Results for MELAS and Announces Pipeline Advancements in Leigh Syndrome and CystinosisTrial achieved biological proof-of-concept, dose discovery and biomarker improvementSan Diego, California--(Newsfile Corp. - November 4, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) (\"Thiogenesis\" or the \"Company\") a clinical-stage biotechnology company developing sulfur-based therapeutics for pediatric diseases, predominantly those involving mitochondrial dysfunction, today announced interim results from its Phase 2 clinical trial of TTI-0102 for Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes or MELAS, which is being conducted at Radboud University in the Netherlands and CHU Angers in France. The Company also provided updates regarding its progress and planning related to its Leigh syndrome spectrum and nephropathic cystinosis clinical programs.Positive Interim Data: MELAS Phase 2 Clinical TrialThe blinded interim analysis is based on the first three months of data from the Company's placebo-controlled Phase 2 MELAS clinical trial. The trial included nine patients: six randomized to Thiogenesis' proprietary TTI-0102 lead compound and three to placebo. The goals of the Phase 2 trial are to provide biological proof-of-concept, dose discovery, biomarker activity and early efficacy.The Phase 2 clinical trial continues with five active patients weighing over 70 kg, two receiving TTI-0102 and three on placebo. TTI-0102 has been well tolerated within the group, consistent with previous Phase 1 clinical trial results in healthy adults as announced in May 2022. The interim analysis also included data from four patients under 50 kg who left the study due to dose-dependent side effects, underscoring the need to refine dosing protocols. The Company plans to adopt two dosing regimens in upcoming clinical trials: the current regimen used for larger patients that is in-line with its ongoing Phase 2 MELAS study and a single reduced dosage for lighter patients. Individual biomarker and efficacy data remain confidential pending potential publication in early 2026. The interim data provide clear biological differentiation among the five active patients and indicate significant support for TTI-0102's mitochondrial antioxidant and restorative activity.\"The interi...