VHIR and SOM Biotech develop drug for amyloidosis
It has received orphan drug designation from the US Food and Drug Administration (FDA)
The Vall d’Hebron Research Institute (VHIR) and SOM Biotech have signed a collaboration agreement to jointly develop a drug for amyloidosis. The research center and biopharmaceutical company aim to carry out phase IIa clinical trials on the SOM0226 compound to treat transthyretin-related amyloidosis (ATTR), a rare disease.
This is a case of drug repositioning: in 2011, SOM Biotech discovered a new use for the SOM0226 compound, which was already on the market, and applied for a patent to protect possible use to treat ATTR. They have now reached an agreement with the VHIR to develop this drug, with more than €2 millions already invested.
Transthyretin-related amyloidosis (ATTR) is a rare disease that affects the peripheral and nervous system and myocardium. The liver and other areas of the body begin to produce mutated transthyretin protein (TTR), which accumulates in various organs and leads to localized cell damage. Liver transplant is the only treatment currently available to prevent or curb the progress of this disease, as there are no drugs to treat it. The SOM0226 compound has received orphan drug designation from the US Food and Drug Administration (FDA).
SOM Biotech is a Catalan biopharmaceutical company established in the Barcelona Science Park (PCB) and focusing on repositioning (or reprofiling) drugs already on the market. This is a fairly uncommon specialization but offers clear economic advantages: the risk and investment needed to develop a new indication for a drug that has already been approved are much lower. SOM Biotech, created in 2009, won the 2013 EmprenedorXXI award in the “Creixes” category for consolidated business projects.
More information is available in the press release.
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