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SOM Biotech announces Orphan Drug Designation in US for SOM0226

By 6 de February de 2014November 18th, 2020No Comments
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Dr. Raúl Insa. founder and CEO of SOM Biotech.
 06.02.2014

SOM Biotech announces Orphan Drug Designation in US for SOM0226

The US Food and Drug Administration (FDA) assigned Orphan Drug Designation on 24 December 2013 to SOM Biotech for SOM0226 for the treatment of Transthyretin Amyloidosis (ATTR). This compound is indicated for all variants of the disease: Familial Amyloid Polyneuropathy (FAP), Familial Amyloid Cardiomyopathy (FAC), Senile Systemic Amyloidosis (SSA) and CNS Selective Amyloidosis (CNSA). The Parc Científic de Barcelona is home to the headquarters of this biotechnology firm, which is one of the few companies in the world specializing in discovering and developing new therapeutic applications for drugs already on the market.

ATTR is a protein aggregation disorder, caused by abnormal extracellular deposition of transthyretin (TTR), a transport protein carrying retinol and thyroxin (T4) in blood and cerebrospinal fluid. The amyloidogenic process in ATTR is characterized by the dissociation of native TTR tetramers into unstable monomers that unfold and aggregate leading to the formation of amyloid fibrils. Deposits of fibrilar TTR and toxic intermediates cause nervous system and/or heart pathology resulting in death within 5-15 years of onset.

SOM0226 is a repositioned compound previously approved for an unrelated indication. It is a potent inhibitor of the TTR amyloidogenesis process and also has fibril disrupting activity, representing a therapeutic alternative with superior cost-effectiveness than existing treatments.

The compound has been validated in several efficacy assays for ATTR and it has been thoroughly tested in vivo and clinically for its currently marketed indication. As a repositioned drug, SOM0226 can bypass much of the early cost and time needed to bring a drug to market and is currently ready to initiate a clinical Phase II Proof of Concept study.

SOM Biotech is a startup pharmaceutical company based at the Parc Científic de Barcelona, founded in 2009, with the mission to carry out research, identification, experimental demonstration, intellectual property, clinical proof of concept and licensing of new applications and medical indications for marketed drugs (repositioning or reprofiling). The identification is performed by a technological solution of virtual screening developed by the own company.

SOM has a broad portfolio of products under development covering both rare diseases (for TTR amyloidosis, Huntington’s disease and glioblastoma multiforme) and other therapeutic areas (Alzheimer’s disease and benign prostatic hypertrophy). Currently, it has filed five new patents for new medical indications and collaborates with leading pharmaceutical companies worldwide.