Interim Report January-September 2020
Mitochondrial myopathies (muscle diseases) are among the most common manifestations of primary mitochondrial disorders. The individual conditions that compose mitochondrial myopathies include MELAS (mitochondrial encephalopathy, lactic acidosis and stroke-like episodes), PEO (progressive external ophthalmoplegia), KSS (Kearns-Sayre syndrome) and MERRF syndrome (myoclonic epilepsy with ragged red fibers). The clinical hallmark of a mitochondrial myopathy includes muscle weakness, exercise intolerance and fatigue and often present with other symptoms of mitochondrial genetic disorders. The severity of the disease can range from generally progressive weakness to death. There is a major unmet medical need for new and effective treatment options for mitochondrial myopathies since there is no specific treatment for these serious diseases.
NVP025 is focused on chronic treatment of mitochondrial myopathies by utilizing a potent cyclophilin inhibitor from Abliva’s Sangamide class of compounds. Mitochondrial myopathies manifest in the MELAS, PEO, KSS, and MERRF syndromes. The goal is to develop a treatment that protects the mitochondria in the muscles from disturbed calcium handling and subsequent muscular dystrophy, thus preventing the muscle weakness associated with these diseases. In collaboration with the Karolinska Institute in Stockholm, Abliva has demonstrated that cyclophilin inhibitors can slow the disease progression and increase survival rates in an experimental mitochondrial myopathy model.