NeuroVive’s Complex I Deficiency discovery program, also known as NVP015, is focused on the generation of novel drug candidates that target mitochondrial energy regulation in a number of mitochondrial disorders associated with complex I deficiency such as Leigh Syndrome.
The research presented last week focuses on the role of NeuroVive’s succinate prodrug candidates in mitochondrial respiration, a key component of mitochondrial energy production. Using a model of healthy cells and cells from a Leigh Syndrome patient, the research team demonstrated that succinate prodrug candidates increased mitochondrial respiration. The research was developed by NeuroVive’s scientific team, including young PhD researcher Sarah Piel who presented the findings to leaders in the field of mitochondrial medicine.
“We are delighted with the progress our Complex I Deficiency discovery program is making,” said Dr. Eskil Elmér, Chief Scientific Officer at NeuroVive “These findings support our hypothesis that succinate prodrugs are a strong candidate for the treatment of complex I Deficiency disorders and allow us to take the next step in candidate development with confidence.”
The next steps in the Complex I Deficiency discovery program involves the synthesis and scaled up manufacture of the most promising candidates, pharmacokinetic studies and early formulation and in vivo proof of concept work. It is anticipated that NeuroVive will select a final drug candidate for further development in 2016.
“Complex I deficiency disorders are rare and can be fatal as there are no existing treatments available to patients,” said Jan Nilsson, Chief Executive Officer. “We believe that our prodrugs may develop into treatments for such disorders and that the potential orphan drug indications offer a significant commercial opportunity.”
About NeuroVive’s Complex I Deficiency discovery program
This discovery platform is based on an idea by NeuroVive co-founder Dr. Eskil Elmér and collaborators to create a cell permeable pro-drug of the endogenous energy substrate succinate. Successful delivery will make succinate available to complex II in the respiratory chain supporting production of energy-carrying ATP molecules in spite of complex I disorders. A successful candidate from this discovery program directed towards paediatric mitochondrial disorders would be eligible for orphan drug status.
About Mitochondrial Complex I Disorders
Mitochondrial disease affects at least 1 in 8000 children, usually presenting at birth or early in life. Complex I deficiency is usually a progressive neuro-degenerative disorder and is responsible for a variety of clinical symptoms, particularly in organs and tissues that require high energy levels, such as brain, heart, liver, and skeletal muscles. Complex I deficiency is caused by mutations in mitochondrial or nuclear genes coding for the subunits of complex I and is the most common mitochondrial disorder presenting in childhood, accounting for up to 30% of cases. These rare disorders include Leber’s hereditary optic neuropathy (LHON), mitochondrial encephalopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonus epilepsy with ragged-red fibers (MERRF) and Leigh Syndrome.
NeuroVive Pharmaceutical AB (publ) is a leading mitochondrial medicine company committed to the discovery and development of highly targeted candidates that preserve mitochondrial integrity and function in areas of therapeutic need. NeuroVive’s business approach is driven by value-adding partnerships with leading mitochondrial research institutions and commercial partners across the globe.
NeuroVive’s portfolio consists of two clinical projects in acute kidney injury (AKI) and traumatic brain injury (TBI) with candidates in clinical and preclinical development and two drug discovery platforms. The NeuroSTAT® product is currently being evaluated in a Phase II study in traumatic brain injury. CicloMulsion® is being evaluated in an ongoing Phase II study, CiPRICS, in acute kidney injury during major surgery. NeuroVive’s shares are listed on NASDAQ OMX, Stockholm, Sweden.
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It is also possible to arrange an interview with NeuroVive’s CEO Jan Nilsson at the above contacts.
NeuroVive Pharmaceutical AB (publ)
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NeuroVive Pharmaceutical AB (publ) is required to publish the information in this news release under The Swedish Securities Market Act. The information was submitted for publication on the 2 November 2015, at 08.30 CET.