The Vall d'Hebron Institute of Research (VHIR) and the biopharmaceutical SOM Biotech, based at the Parc Científic de Barcelona (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 that affects the peripheral nervous system and myocardium. 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 accumulation (deposits of amyloid) provokes local cellular damage that is expressed with a variety of clinical symptoms, leading to the death over a period of between 5 and 15 years.

“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. José 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.

SOM Biotech studies have shown that SOM0226 represents a therapeutic alternative superior to other products in development. It is a potent inhibitor of the amyloidogenic process with an innovative dual mechanism of action; stabilizing the TTR protein and also disrupting the fibers once deposited in tissues (thus stopping the progression and reversing the disease), which may be effective for the four forms of the ATTR, including the advanced stages.

On the other hand, SOM0226 is a repurposed drug which has previously been approved for another indication, so the risk and the level of investment to develop this new ATTR indication is reduced. The market price of this drug would mean a considerable reduction of costs for public health systems, compared to the few current treatment options and, at the same time, it would be a much more accessible for the ill population.

An investment of more than €2.000.000

The new therapeutic activity of the SOM0226 for the treatment of transthyretin Amyloidosis was discovered by SOM Biotech through its property virtual screening platform, which is able to identify new therapeutic activities of already-marketed drugs. Later on, SOM0226 was experimentally validated in several non-clinical models in laboratories from Spain, Europe and the United States. The positive results obtained made it possible to protect the new use of SOM0226 for the treatment of transthyretin Amyloidosis through an international patent.

Achieving this goal has been an effort over three years and an investment of more than €2.000.000, which has been possible thanks to private investment and public grants/loans of the Agency for the Company Competitiveness (ACC1Ó) of the Generalitat of Catalonia, the Spanish Patent and Trademark Office, and the Ministries of Health and Economy.

“Despite the considerable efforts in research, there is no other drug in the process of health approval for transthyretin Amyloidosis, where an alternative effective therapy is of great need for patients. The only available treatments are a drug discovered in the United States with the exorbitant price of €140,000 a year and liver transplantation, only suitable for a particular population with a very high economic cost. Collaboration with VHIR and the results of this joint project will allow progress in the development of new pharmacological, effective and safe treatments for ATTR patients, with an acceptable cost-effectiveness”, says Dr. Raúl Insa, founder of SOM Biotech.

“This is a very good chance for VHIR to go walk with a company like SOM Biotech that risks and committs to collaborate with a leading clinical trials center, an initiative that should open the door to future alliances with other companies. In addition, in this case acquires greater relevance if we take into account that it is a disease with a high economic and social impact for the health system”, says Xavier Molina, head of the VHIR Promotion Area.

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