NeuroVive Pharmaceutical AB, a leader in mitochondrial medicine, has signed a preclinical collaboration agreement with the Children’s Hospital of Philadelphia (CHOP) and Marni J. Falk, M.D., a US key opinion leader in the mitochondrial medicine field.
Dr. Falk’s research team at CHOP will evaluate compounds from NeuroVive’s research programme, NVP015, in certain experimental disease models. The research team will study energy metabolism and disease development in models of mitochondrial complex I dysfunction.
Marni J. Falk. is an attending physician and director of the mitochondrial disease clinical center at CHOP, US, which is a large center for children and adults with mitochondrial disorders and a leading mitochondrial disease research center. Dr. Falk’s experience spans all the way from early experimental research through to clinical development and covers the whole drug development process. CHOP is one of the world’s largest children’s hospitals and has consistently been ranked as one of the best hospitals for children in the US.
“We feel fortunate to be able to initiate this collaboration with a well-recognized institution like CHOP and Dr. Marni Falk, a well-esteemed leader in the mitochondrial disease field. This is an important step in further advancing and strengthening our NVP015 program, as well as in the development of novel treatment opportunities for people with mitochondrial medicine disorders”, commented NeuroVive’s CMO Magnus Hansson.
The NVP015 discovery programme is based on a concept from NeuroVive’s CSO, Dr. Eskil Elmér and collaborators to create a cell permeable prodrug of the endogenous energy substrate succinate. A prodrug is an inactive drug which is not activated until when entering the cell and the chemical structure is altered. A successful candidate from this discovery programme in paediatric mitochondrial disorders would classify as an orphan drug.
One of the most common causes of mitochondrial diseases relates to complex I dysfunction, which occurs when 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 such as Leigh Syndrome and MELAS, both of which are very serious diseases with symptoms such as muscle weakness, epilepsy, and other severe neurological manifestations. Mitochondrial diseases usually present early in life and deteriorate progressively.
NeuroVive Pharmaceutical AB is a leader in mitochondrial medicine. The company is committed to the discovery and development of medicines that preserve mitochondrial integrity and function in areas of unmet medical need.