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Thiogenesis Therapeutics Expands on Plans for Phase 3 Pivotal Trial of TTI-0102 in Nephropathic Cystinosis
IND expected in early 2026; TTI-0102 designed to improve tolerability & simplify dosing San ...
About this update from Thiogenesis Therapeutics Corp
[{"type":"text","content":"Thiogenesis Therapeutics Expands on Plans for Phase 3 Pivotal Trial of TTI-0102 in Nephropathic CystinosisIND expected in early 2026; TTI-0102 designed to improve tolerability & simplify dosingSan Diego, California--(Newsfile Corp. - November 24, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) (\"Thiogenesis\" or the \"Company\"), a clinical-stage biotechnology company developing next-generation sulfur-based prodrugs for rare mitochondrial and metabolic diseases, today announced plans to initiate a Phase 3 pivotal clinical trial of its lead compound TTI-0102 for the treatment of nephropathic cystinosis, an inherited lysosomal storage disorder requiring lifelong cystine-depleting therapy. The Company plans to submit an Investigational New Drug (IND) application in early 2026. About Nephropathic CystinosisNephropathic cystinosis is a rare, autosomal recessive lysosomal storage disorder caused by mutations in CTNS, leading to toxic intracellular cystine accumulation and progressive multi-organ damage. Without disease-modifying therapy, patients develop renal Fanconi syndrome, growth failure, and progression to end-stage renal disease. Lifelong cysteamine treatment slows, but does not eliminate disease progression, and tolerability challenges frequently lead to suboptimal adherence. There are two currently approved drugs for cystinosis; immediate release cysteamine (Cystagon®) and delayed-release cysteamine (Procysbi®). Both require multiple daily dosings, and have significant side effects. The global cystinosis population exceeds 2,000 patients worldwide, representing a market opportunity of over $300 million.A Next-Generation Approach to Cystine DepletionTTI-0102 is a next-generation cysteamine-based prodrug engineered to address longstanding limitations of current standard-of-care therapies including their, short half-life, side effects and dosing frequency. TTI-0102 is designed to potentially offer:Once-daily oral dosing, enabled by controlled prodrug metabolism and extended exposureReduced peak-related GI intolerance, a major limitation of current cysteamine productsImproved tolerability across weight ranges, supported by results from the Company's MELAS Phase 2 program Dual mechanistic activity: cystine depletion and enhancement of intracellular antioxidant pathways (glutathione and taurine), c...