Advanced understanding of neurodegenerative diseases achieved by Trophos

Trophos SA, a clinical stage pharmaceutical company developing innovative therapeutics from discovery to clinical validation for indications with under-served needs in neurology and cardiology, announces today the conclusion of the pan-European MitoTarget Project, the EU funded orphan disease project, with submission of the final report to the EC.

The results obtained in this project included additional confirmation that mitochondrial dysfunction plays an important role in neuron death, and therefore the progression of neurodegenerative diseases.

The MitoTarget project contributed towards broader research required to discover which dysfunction/s is the leading cause of neurodegenerative diseases, key to finding effective drug targets in the future. It also further confirmed that Trophos’ lead drug candidate olesoxime, currently in an ongoing pivotal trial for the orphan neuromuscular disease Spinal Muscular Atrophy (SMA), targets mitochondria.

Various changes in mitochondrial function, distribution or number were found in all experimental models of neurodegenerative diseases including Alzheimer's Disease, Huntington's Disease, Hereditary Spastic Paraplegia (HSP), Amyotrophic Lateral Sclerosis (ALS), and even general aging. There were very promising results obtained in models of ALS, Huntington's Disease and Alzheimer's Disease.

Evidence produced by MitoTarget also proved mitochondrial targeted drugs should provide a part of the therapeutic strategy of neurodegenerative diseases. Combination therapeutics will be essential to attack multiple aspects of the disease including both the causes and the symptoms.

Trophos and the consortium have already published many of their results and further publications are planned describing results of the basic research as well as the clinical trial in ALS to aid all parties involved in future research to work towards eradicating and curing diseases that include SMA, Alzheimer's Disease, Huntington’s Disease as well as ALS.

The three-year project was initiated in 2007 with the creation of the consortium, commencing the main part of the activity in 2009, and was funded by the EU. Advocates included the British theoretical physicist, Stephen Hawking, CH, CBE, FRS, FRSA, and a patron of the Motor Neurone Disease Association.

Led by Trophos, the MitoTarget consortium consisted of 17 partners from four European countries (France, Germany, United Kingdom, and Belgium). Fourteen of the partners as well as a centre in Spain participated in the clinical trial element of the project and five basic research groups focused on understanding the underlying mechanisms of neurodegenerative diseases.

“Trophos has been deeply honored to have led the MitoTarget Consortium, which has made significant discoveries that will help future research into neurological diseases,” said Rebecca Pruss, MitoTarget project principle investigator and Trophos’ chief scientific officer. “The research section of the project made considerable progress that will greatly contribute to, and benefit future efforts to find therapies for Alzheimer’s, Parkinson’s, Huntington’s diseases, ALS and other neurodegenerative diseases including Multiple Sclerosis.”