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Program for mitochondrial disorders


Medical problem

Primary mitochondrial disorders are congenital hereditary metabolic diseases that affect cellular energy conversion. These diseases can express in widely differing ways, and are viewed as syndromes depending on the combination of symptoms and research discoveries. One of the most common causes of mitochondrial diseases relates to Complex I Dysfunction, i.e. energy conversion in the first of the five protein complexes in the mitochondrion that are involved in effective energy conversion does not function normally. This is apparent in disorders including Leigh’s Syndrome and MELAS, both of which are very serious diseases with symptoms such as muscle weakness, epileptic fits and other severe neurological manifestations. These diseases often first express early in life and deteriorate progressively. Many organs and tissue types can be affected.

Treatment objective

The energy shortage in mitochondrial disorders exacerbates as the body’s need for energy increases, in cases of infection and fever, for example. This energy shortage can cause serious symptoms and require intensive care; at present, there is no specific therapy to improve energy supply to the body’s organs. NeuroVive’s energy regulators are being developed to satisfy this need for energy during these episodes and are designed to circumvent the impaired metabolism. By alleviating the episodes and limiting the organ damage arising as a result of the energy shortage, the complications from these diseases are mitigated. NeuroVive also develops a substance that has mitochondrial protective effects mediated through inhibition of cyclophilin D. In an experimental model this has been shown to prevent muscle fiber weakness in mitochondrial myopathy.

Market potential

About 10 people per 100,000 have a mitochondrial disorders, which often express first in early childhood. Accordingly, pharmaceuticals for treating primary mitochondrial disease have orphan drug status, which means greater potential for market approval than traditional pharmaceuticals. The value of orphan drugs is several billion Swedish kronor, and the yearly cost of therapy for a single patient can be in the range of SEK 200,000 to SEK 1.5 m.

Development platform

NeuroVive's project in clinical phase I, KL1333 is a potent modulator of the cellular levels of a central coenzyme in the cell’s energy metabolism. KL1333 has in preclinical studies been demonstrated to increase mitochondrial energy output, reduce lactate accumulation, diminish the formation of free radicals, and to have long-term beneficial effects on energy metabolism. It is in clinical development stage for chronic oral treatment of primary genetic mitochondrial disorders such as MELAS, KSS, CPEO, PEO, Pearson, MERRF and Alpers syndrome.

NVP015 is being developed as an energy-regulating preparation for specific intravenous acute treatment of conditions involving cellular energy crisis. The objective is to develop an orphan drug for a series of relatively unusual childhood diseases, and as an acute therapy for compromised mitochondrial function caused by pharmaceuticals. There is also potential usage for large patient groups, where the body could benefit from extra energy production, in extended surgery and intensive care, for example.

Using our proprietary cyclophilin inhibitor platform, compounds are being developed for the treatment of mitochondrial myopathy. The NVP025 project is targeting cyclophilin D and therefore has a different and complementary mode of action.