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From left to right: Gisela Gili, Dr. Josep Gámez, Dr. Raul Insa, Maria Salvadó and Alba Montoliu (Photo: VHIR).

SOM Biotech and Vall d’Hebron successfully complete a clinical trial in patients with TTR amyloidosis

SOM Biotech and the Vall d'Hebron Research Institute (VHIR) have successfully completed a Phase IIa proof-of-concept clinical trial in humans to evaluate the efficacy and safety of an innovative oral therapy for transthyretin amyloidosis (TTA), a rare disease that affects the peripheral nervous system and heart. To carry out this trial, the hospital and the Catalan biotech, with headquarters at the Barcelona Science Park (PCB), signed an risk-sharing agreement under (RSA) which they jointly assumed the risks and economic returns of the project.


The trial of compound SOM0226, a reprofiled drug developed entirely by the biopharmaceutical company, was initiated a year ago under the coordination of Dr. Jose Gámez, head of the Peripheral Nervous System Research Unit of the Vall d’Hebron Research Institute (VHIR). 17 subjects participated in the trial, including patients affected by the polyneuropathic variant of the disease and healthy individuals.

The results have demonstrated excellent drug efficacy for the treatment of amyloidosis, as it has been able to stabilize the 100% of protein transthyretin (TTR) present in plasma in all patients treated. Moreover, the trial has also shown a high safety profile, since there have been no drug-related adverse effects.

“We have shown that compound SOM226 has a high potential to stabilize protein transthyretin in patients and therefore, it can slow down the progression of this invalidating minority disease”, says Dr. Gámez.

According to Dr. Raul Insa, founder and managing director of SOM Biotech: “We have achieved one of the goals most highly desired by any company in the pharmaceutical sector: to demonstrate efficacy in patients of a totally “in-house” discovered drug. In addition, being a minority disease. with little or no treatment, our satisfaction is twofold. On the other hand, SOM0226, is a reprofiled drug, which accelerates the various stages of its development, saving time and costs until it arrives on the market. We expect that the excellent results yielded by this trial will allow us to successfully complete a license in a short time.”

The new therapeutic activity (reprofiling) of SOM0226 for the treatment of amyloidosis was discovered by SOM Biotech through a proprietary virtual screening computational platform. Subsequently, the drug was validated experimentally in several non-clinical models in medical centers in Spain, Europe and the United States. The positive results permitted the protection of SOM0226 by means of an international patent.

Bringing the drug to clinical phase has been made possible as a result of the great efforts made over three years and an investment of 2.000.000 € obtained through private capital and public grants and loans from the Agency for Enterprise Competitiveness (ACC1Ó) of the Generalitat de Catalunya, the Spanish Patent and Trademark Office, and the Spanish Ministries of Health, Social Welfare and Equality, Economy and Competiveness and Industry, Energy and Tourism.

A delegation of SOM Biotech, led by Dr. Raúl Insa, had the opportunity to present the preliminary results of the clinical trial at the 1st European Congress on Hereditary ATTR Amyloidosis, which took place on November 2 and 3, 2015 in Paris. The work received numerous appraisals, achieving one of the first awards for best presentation.

Great hope for patients

Amyloidosis associated to transthyretin occurs when the liver and other parts of the body produce the mutated protein transthyretin (TTR), causing a number of aggregates which are deposited in multiple organs. This extraordinary accumulation of protein aggregates known as amyloid deposits causes local cell damage leading to a variety of clinical symptoms, including cardiac and neurological problems leading to the death of the patient over a period of between 5 and 15 years.

According to Dr. Gámez, “the test results are very satisfactory and suggest that SOM Biotech´s drug is able to slow the progression of all of the forms in which this disease occurs. Until now, only one treatment exists in the world for the treatment of transthyretin amyloidosis, but is only indicated in the early phase of one of the variants of the disease.”

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