A new pharmacological therapy for the mitochondrial disease Leigh syndrome

Purpose and goal

** Denna text är maskinöversatt ** The project, with the overall goal of developing a treatment for patients with mitochondrial diseases caused by dysfunction in the first complex (CI) in the mitochondrial electron transport chain, has developed a substance from the preclinical characterization stage to the almost complete preclinical development. The project´s goal, to take NV354 until clinical testing, is expected to take place at the turn of the year 2021/2022.

Expected results and effects

** Denna text är maskinöversatt ** NV354 has been produced in kg quantities under quality controlled conditions (GMP) and has been found to be efficient and have a satisfactory safety margin with respect to toxicity. Due to supplementary toxicological studies, the development and manufacture of a clinical trial product has been delayed and will be carried out after the end of the Swelife project.

Planned approach and implementation

** Denna text är maskinöversatt ** The schedule for planned activities during WP1-3 has proceeded as expected or slightly faster than expected. Due to a deviating finding in a standard GLP study during WP3, further studies were needed to ascertain the relevance of the deviating finding. This has led to several additional studies. These have now been completed and the standard GLP tox studies required to be able to start clinical trials in healthy volunteers have now been completed.