San Diego, California — January 13, 2026 — Leads & Copy — Thiogenesis Therapeutics, Corp. announced that an abstract outlining preliminary results from its Phase 2 clinical trial of TTI-0102 for Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS) in the EU has been accepted as Late-Breaking News for presentation at the Mitocon Conference 2026, which will take place in Pisa from January 23-26, 2026.
The poster entitled, “Pharmacokinetics and Pharmacodynamics of TTI-0102 in MELAS,” will be presented during the Poster Session on January 23rd, and will summarize preliminary pharmacokinetic and pharmacodynamic findings from Thiogenesis’ Phase 2 MELAS study. The abstract was selected through Mitocon’s Late-Breaking News review process by the conference’s Scientific Committee.
The Phase 2 MELAS clinical trial is a randomized, double-blind, placebo-controlled, multi-center study designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of oral TTI-0102 in patients with MELAS. Interim results from the study demonstrated biological proof-of-concept and biomarker activity supporting TTI-0102’s mechanism of action.
Patrice Rioux, MD, Ph.D., Chief Executive Officer and Co-Founder of Thiogenesis, said that being selected for a late-breaking presentation at Mitocon underscores the scientific importance of the data generated in the company’s Phase 2 MELAS study. He added that the pharmacokinetic and pharmacodynamic profile observed to date is consistent with TTI-0102’s proposed mechanism of action and supports continued clinical development in MELAS and related mitochondrial disorders, including the company’s upcoming Phase 2a clinical trial in Leigh syndrome spectrum in the U.S.
Mitocon is the leading Italian patient advocacy organization dedicated to mitochondrial diseases. The organization promotes research, education, and collaboration among clinicians, scientists, and patient communities. Its annual international conference is a recognized as a leading forum for the presentation of emerging scientific and clinical advances in mitochondrial medicine.
MELAS is an inherited mitochondrial disorder, most often caused by a mutation of m.3243A>G in the MT-TL1 gene in mitochondrial DNA. Initial symptoms usually include seizures, vomiting, headaches, muscle weakness, loss of appetite and fatigue. Oxidative stress, including deficiencies in glutathione and taurine, play an important role in mitochondria dysfunction and are potential pathological mechanisms of mitochondrial disorders, making for viable targets for the treatment of MELAS and other mitochondrial diseases. There are estimated to be approximately 4.1/100,000 of the population with MELAS worldwide.
Leigh syndrome spectrum is a rare, inherited genetic disease that affects the power plant of the cell, the mitochondria. It is usually diagnosed in infancy and occurs in an estimated 1/40,000 live births. Symptoms include weak sucking/breastfeeding, loss of motor and communication skills, poor muscle development, respiratory issues, weakness/fatigue and seizures. There are currently no approved drugs for Leigh syndrome spectrum.
TTI-0102 is Thiogenesis’ lead product candidate and a next-generation cysteamine-based prodrug designed to address limitations associated with first-generation thiol therapies, including short half-life, gastrointestinal side effects, and dosing constraints. As a prodrug, TTI-0102 is metabolized following ingestion, enabling controlled release of cysteamine with the potential for improved tolerability and once-daily dosing. TTI-0102 is being evaluated across multiple indications associated with mitochondrial dysfunction and oxidative stress.
Thiogenesis Therapeutics, Corp. is a clinical-stage biopharmaceutical company with operations based in San Diego, California. The company is traded on the TSX Venture Exchange and in the U.S. on the OTCQX. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol-active compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Thiogenesis’ lead product candidate, TTI-0102 has an active Phase 2 clinical trial in MELAS, an IND-cleared Phase 2a clinical trial planned in Leigh syndrome spectrum, a Phase 2 clinical trial planned in pediatric MASH and a Phase 3 clinical trial planned in nephropathic cystinosis.
Brook Riggins, Director, and CFO can be reached at info@thiogenesis.com or (888) 223-9165.
Source: Thiogenesis Therapeutics
