A piece of news
It is a rare disease that affects the nervous system and heart, with only one treatment in the world
SOM Biotech, based at the Barcelona Science Park (PCB), and the Vall d’Hebron Research Institute (VHIR), have successfully completed a Phase IIa proof-of-concept clinical trial in humans to assess the efficacy and safety of an innovative oral treatment for transthyretin amyloidosis (ATTR), a rare disease that affects the peripheral nervous system and heart. The results have proven the efficacy of drug SOM0226, stabilizing 100% of the protein transthyretin in the plasma of affected patients.
ATTR is caused by a mutation of the transthyretin protein (TTR) normally produced by the liver and other organs. Patients with this condition, however, have a genetic mutation that means the liver doesn’t properly produce the protein and leads to a series of aggregates that, when they build up in other organs, lead to a variety of clinical symptoms including heart and neurological problems leading to death within 5 to 15 years. In Spain an estimated 400 people are affected by this disease.
To carry out this drug-reprofiling study on SOM0226, developed fully by SOM Biotech, the biopharmaceutical company signed an agreement with the hospital to jointly assume both the risks and the financial return. The clinical trial began a year ago under the leadership of Dr. Josep Gámez, head of the VHIR Peripheral Nervous System Research Unit, with 17 participants, including affected patients and healthy individuals. None of them showed any adverse side effects.
“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,” explained Dr. Gámez. Raúl Insa, founder and CEO of the biopharmaceutical firm, says that compound SOM0226 saves time and money, as it is a reprofiled drug. “We’ve demonstrated the 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. We expect that the excellent results yielded by this trial will allow us to successfully complete a license in a short time.”
More information is available in the press release