SOM Biotech develops a drug for Amyloidosis in collaboration with VHIR

16/04/2014 12:04


The Vall d'Hebron Institute of Research (VHIR) and the biopharmaceutical SOM Biotech, based at the Barcelona Science Park (PCB), have signed an agreement to jointly develop the clinical phase of a drug for transthyretin Amyloidosis (ATTR). The collaboration starts with the execution of a proof of concept in 20 healthy voluntiers and patients affected by the polineuropatic form of the disease - which will confirm the efficacy and safety of compound SOM0226. The product, a repositioned drug developed by SOM Biotech, may be effective for all variants of the ATTR and has been designated recently orphan drug by the Food and Drug Administration (FDA) of United States.


The Amyloidosis related to transthyretin protein is a rare disease, progressive, severe and debilitating, that mainly affects the peripheral nervous system and myocardium and, in some cases, is inherited from parents to children. The disease occurs when the liver and other parts of the body produce a mutated transthyretin protein, causing a series of aggregates that are deposited in multiple organs. This extraordinary accumulation of abnormal protein - known as deposits of amyloid - provokes local cellular damage that is expressed with a variety of clinical symptoms, leading to the death of the patient over a period of between 5 and 15 years, after suffering a major heart and nervous problems.


"Currently on the world market only exists a pharmacological treatment for one of the early forms of polineuropatic transthyretin Amyloidosis.  The cost of this treatment is very high and only has approval for Europe, so there is a clear medical need that supports the development of new drugs for the treatment of this disease", says the neurologist Dr. Josep Gamez, head of the research group of the Peripheral Nervous System of the VHIR and coordinator of the Neuromuscular Disease Unit at the Service of Neurology of the Hospital Universitario Vall d’Hebron, where the clinical trial will take place.