A new pharmacological therapy for the mitochondrial disease Leigh syndrome

Reference number 2018-03231
Coordinator NeuroVive Pharmaceutical AB
Funding from Vinnova SEK 5 000 000
Project duration November 2018 - November 2020
Status Ongoing
Venture SWElife Phase 2 2017
Call 2018-02544-en

Purpose and goal

The project objective is to develop a new pharmacological therapy for chronic oral treatment of patients with the mitochondrial disease Leigh syndrome. Leigh syndrome is a severe inherited genetic mitochondrial disease primarily affecting the central nervous system and other organ systems with high energy requirements. It usually presents in the first year of life, and is normally fatal within a few years. There are no pharmacological therapies available for Leigh syndrome and related disorders.

Expected results and effects

Most cases of Leigh syndrome are due to dysfunction in complex I (CI) of the mitochondrial electron transport system. The drug compound we develop in this project is specifically targeting this dysfunction. A drug targeting Leigh syndrome could ameliorate clinical progression of disease, reduce morbidity, and decrease mortality in this patient group. Subsequently NV354 has the potential to receive approval for use in other mitochondrial diseases, thereby serving a much broader patient population.

Planned approach and implementation

The team will progress the project through TRL5 and prepare it for IND/CTA application and Phase I first-in-human clinical trials. The project will also include activities in order to have a clinical stage candidate drug with a solid business case. Important relationships have been built with key academic investigators, KOLs, care givers and patient organisations which will be further strengthened.

External links

The project description has been provided by the project members themselves and the text has not been looked at by our editors.