Update on Two Clinical Programs Today, Thiogenesis Therapeutics (TTI.v), a clinical-stage biotechnology company, presented updates on its two clinical programs for mitochondrial diseases during the UMDF "Bench-to-Bedside" webcast.
MELAS
TTI is advancing its lead product, TTI-0102, with promising applications in treating Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes (MELAS) and Leigh Syndrome (LS).
For MELAS, TTI has submitted its Clinical Trial Application Part II in Europe, aiming to start a Phase 2 trial in early 2025 in the Netherlands and France. This trial will involve 12 patients, focusing on physical endurance and biomarker analysis.
Notably, MELAS is a rare mitochondrial disorder affecting 15,000 patients in the U.S. and 20,000 in the EU, and has symptoms like seizures, muscle weakness, and intellectual disability. TTI-0102 targets oxidative stress, a key factor in the disease.
Leigh Syndrome (LS)
For LS, TTI collaborated with a leading U.S. children's hospital and conducted a pre-IND meeting with the FDA. Following feedback, TTI plans to file an IND and launch a Phase 2a trial in early 2025. LS, diagnosed in infancy and affecting 1 in 40,000 births, has severe symptoms including motor skill loss and seizures. With no approved treatment, TTI-0102 offers potential relief by addressing oxidative stress.
Highlighting TTI's commitment to advancing mitochondrial disease treatments and supporting advocacy efforts through UMDF, CEO Patrice Rioux commented:
"It is a privilege to get the opportunity to support UMDF whenever possible as they provide invaluable services in increasing the profile of mitochondrial disease, patient and family support, and industry advocacy, therefore, I was thrilled to get the opportunity to present updates on our MELAS and Leigh syndrome clinical programs on the UMDF Webcast."
Full News Release: https://www.newsfilecorp.com/release/233143/Thiogenesis-Announces-Presentation-on-UMDF-Webcast-Featuring-Clinical-Trials-in-Mitochondrial-Disease
Posted on Behalf of Thiogenesis Therapeutics Corp.