Thiogenesis Therapeutics Corp. is advancing clinical programs for two rare mitochondrial diseases, MELAS and Leigh Syndrome (LS). CEO Patrice Rioux presented updates on these programs at the United Mitochondrial Disease Foundation (UMDF) Webcast on December 9, 2024.
Phase 2 Trial Planned for MELAS
The company's Clinical Trial Application Part II in Europe for MELAS has been submitted in December 2024, paving the way for a Phase 2 clinical trial in the Netherlands and France, expected to begin in the first quarter of 2025. The randomized, placebo-controlled trial will involve 12 patients, with 8 receiving Thiogenesis' lead product candidate, TTI-0102, and 4 receiving a placebo. The primary endpoint will assess the impact of TTI-0102 on patient walking endurance using a 12-minute walk test. Relevant biomarkers will be measured as secondary endpoints to evaluate the drug's effects on the underlying pathophysiology of MELAS.
MELAS is a rare, inherited mitochondrial disorder resulting from mutations in mitochondrial DNA. Symptoms include seizures, muscle weakness, fatigue, loss of motor skills, and intellectual disability. The estimated prevalence of MELAS is approximately 15,000 patients in the US and 20,000 in the EU. Oxidative stress is a key pathological mechanism in mitochondrial diseases, and TTI-0102 is designed to counteract this stress.
Advancing TTI-0102 for Leigh Syndrome
Thiogenesis is also collaborating with a leading US children's hospital to develop a treatment for LS. Following a pre-IND meeting with the FDA in November 2024, the company is preparing to file its IND for a Phase 2a clinical trial of TTI-0102 in LS. If the IND is cleared, the trial is expected to commence in the first quarter of 2025.
Leigh Syndrome is a rare inherited genetic disease resulting from mutations in both mitochondrial and nuclear DNA, affecting approximately 1 in 40,000 births. Initial symptoms include impaired sucking/breastfeeding capability, loss of motor and communication skills, respiratory issues, poor muscle development, loss of appetite, and seizures. Oxidative stress is a distinguishing feature of the disease, and there is currently no approved treatment for LS.
About TTI-0102
TTI-0102 is a novel thiol compound being developed by Thiogenesis to treat mitochondrial diseases. It is designed to reduce oxidative stress, a key factor in the pathology of MELAS and Leigh syndrome.
