November 24, 2025 — Leads & Copy —
San Diego-based Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) is planning to initiate a Phase 3 pivotal clinical trial for its lead compound, TTI-0102, to treat nephropathic cystinosis.
According to the company press release, TTI-0102 is a next-generation sulfur-based prodrug designed to address limitations of current standard-of-care therapies for rare mitochondrial and metabolic diseases. The company expects to submit an Investigational New Drug (IND) application in early 2026.
Nephropathic 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. Current treatments, immediate release cysteamine (Cystagon®) and delayed-release cysteamine (Procysbi®), require multiple daily doses and have significant side effects.
Thiogenesis’ TTI-0102 aims to improve upon existing treatments with the potential for once-daily oral dosing, reduced GI intolerance, and improved tolerability across weight ranges. The drug is designed to have dual mechanistic activity, depleting cystine and enhancing intracellular antioxidant pathways.
The Phase 3 trial will be conducted under the FDA’s 505(b)(2) regulatory pathway, as TTI-0102 is reduced and metabolized in the GI tract, releasing cysteamine, the active ingredient in Cystagon® and Procysbi®.
Thiogenesis’ ongoing Phase 2 MELAS trial has provided insights into dosing strategies, demonstrating clear biomarker responses supporting thiol-mediated mitochondrial antioxidant activity, dose-dependent tolerability patterns, and a fully characterized exposure-response profile. These data have enabled Thiogenesis to refine dosing regimens and enhance the biological rationale of the cystinosis Phase 3 program, reducing typical development risk.
Dr. Patrice Rioux, Chief Executive Officer of Thiogenesis Therapeutics, said TTI-0102 was engineered to overcome the limitations of existing cysteamine formulations. Rioux added that with the potential for once-daily or reduced dosing and better tolerability profile, Thiogenesis believes TTI-0102 represents the next-generation therapy that patients and families need and would significantly increase their quality of life.
TTI-0102 is a sulfur-based disulfide prodrug consisting of two cysteamine molecules and one molecule of pantothenic acid (Vitamin B5). It is currently in clinical development for MELAS, Leigh syndrome, pediatric MASH, and nephropathic cystinosis.
Thiogenesis Therapeutics Corp. is publicly traded on the TSX Venture Exchange and in the U.S. on the OTCQX.
Brook Riggins, Director and CFO, can be reached at info@thiogenesis.com or by calling (888) 223-9165.
Source: Thiogenesis Therapeutics
