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Thiogenesis Therapeutics Expands on Plans for Phase 3 Pivotal Trial of TTI-0102 in Nephropathic Cystinosis
Newsfile· 2025-11-24 14:00
Core Insights - Thiogenesis Therapeutics plans to initiate a Phase 3 pivotal clinical trial for TTI-0102, targeting nephropathic cystinosis, with an Investigational New Drug (IND) application expected in early 2026 [1][10] Group 1: Company Overview - Thiogenesis Therapeutics is a clinical-stage biotechnology company focused on developing sulfur-based prodrugs for rare mitochondrial and metabolic diseases [10] - The company is publicly traded on the TSX Venture Exchange and OTCQX, with operations based in San Diego, California [10] Group 2: Product Details - TTI-0102 is a next-generation cysteamine-based prodrug designed to improve tolerability and simplify dosing compared to existing therapies [3][4] - The drug consists of two cysteamine molecules and one molecule of pantothenic acid (Vitamin B5), allowing for once-daily dosing with reduced peak-related toxicity [9] Group 3: Clinical Trial Insights - The Phase 3 trial will utilize established endpoints and trial design informed by biomarker and tolerability data from the ongoing MELAS clinical study [5][7] - The trial aims to address limitations of current cysteamine therapies, which require multiple daily doses and are associated with significant side effects [2][13] Group 4: Market Opportunity - The global population of cystinosis patients exceeds 2,000, representing a market opportunity of over $300 million [2]