SOM Biotech, located at the Parc Científic de Barcelona, has initiated the clinical phase of an innovative oral therapy for transthyretin amyloidosis (ATTR): compound SOM0226, a reprofiled drug developed entirely by the biopharmaceutical company. The phase IIa proof-of-concept trial in humans is being conducted at the Vall d'Hebron University Hospital, coordinated by Dr. Josep Gámez, group leader of the Peripheral Nervous System Research Unit at Vall d'Hebron Research Institute (VHIR). The hospital will assume, jointly with the biotech, the risks and return on investment of the development of the drug, with the recent support from the Spanish Ministry of Economy.

Transthyretin amyloidosis is a rare disabling disease with a severe prognosis that primarily affects the peripheral nervous system and the heart and, in some cases, is inherited from parents to children. The disease develops when the liver and other parts of the body produce the mutated protein transthyretin (TTR), causing a number of aggregates that are deposited into multiple organs. This excessive accumulation of protein, known as amyloid deposits,causes local cell damage that presents with a variety of clinical symptoms. These symptoms include severe cardiac and neurological problems that lead to the death of the patient within a period of 5 to 15 years.

The new therapeutic activity of SOM0226 for the treatment of amyloidosis was discovered by SOM Biotech by means of a proprietary computational virtual screening platform. Subsequently, the drug was validated experimentally in several non-clinical models in laboratories throughout Spain, Europe and US. The positive results achieved endorsed to obtain an international patent of the new use of SOM0226 for the treatment of transthyretin amyloidosis.

Achieving this goal has required great effort for over three years and an investment of 2.000.000 € which has been made possible 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, Economy and   Industry.

“Currently there is no other drug undergoing marketing approval for transthyretin amyloidosis in Europe. The only treatment available is a drug discovered in the United States (with the exorbitant price of more than 140,000 € per patient and per year) and liver transplantation, suitable only for a particular population group and with very high social and health costs. Our innovative product is generating much interest from major international pharmaceutical companies. In the recently BioEurope conference held in Frankfurt, SOM Biotech presented the project to over 40 companies who showed an interest in continuing the development of the drug in order to reach the market as quickly as possible. The results of the phase IIa clinical trial will be crucial in consolidating these collaborations and will facilitate to license out the product by the second quarter of 2015”, says Dr. Raúl Insa, founder and CEO of SOM Biotech.

Objective: prevent, fight against and revert the disease

SOM0226 is a potent inhibitor of the amyloidogenic process with an innovative dual mechanism of action: as stabilizer of the transthyretin protein and disruptor of the fibers once they are deposited into the tissues. To cut it short, it stops progression and reverts the disease. This drug may also be effective against the 4 variants of transthyretin amyloidosis (polyneuropathic, cardiac, senile systemic and the CNS selective) including the more advanced stages of the disease. As a consequence, “this new therapeutic alternative will not only be able to confront Transthyretin amyloidosis but will also be able to prevent its progression, avoiding the formation of amyloid fibers”, remarks Dr. Gámez.

The phase IIa proof-of-concept clinical trial in humans will evaluate the activity of SOM0226 in stabilizing the protein, confirming both the safety and efficacy of the compound against the disease. The study will be conducted in 20 individuals, 15 of whom are affected by the polyneuropathic variant of this disease and 5 healthy volunteers.

According to Dr. Gámez, the pharmacokinetic and economic characteristics of this drug make it the first choice for treatment of asymptomatic patients: “SOM0225 will allow the access to a most effective treatment and cheaper to a major number of patients”. Also, VHIR researcher ensures that “the indications will extend to patients that are not candidates for a hepatic transplant, because of the age; to patients living in countries with less resources, or when the transplant is not viable or with long waiting lists; to patients already transplanted in which the progression of the disease is clear and, as a preventive measure, to carriers in age of risk to develop the disease”.

“The inclusion of the first patients in the clinical trial was a total success resulting from the joint effort and collaboration with the Vall d’Hebron Hospital, where a clear sensitivity to those affected by this very debilitating disease has always been a priority. In parallel, SOM Biotech is developing a new pharmaceutical form that will allow the administration of a once daily single dose of the drug, thus improving, the convenience of administration of the new treatment”, says Dr. Insa.