论文部分内容阅读
Background:SOM Biotech owns a virtual screening in-silico technology able to identify new activities of already know drugs (www.sombiotech.com).Virtual screening (VS) is normally used to discover new chemical scaffolds in relation to a desired biological activity and may be based on a specific target or ligand (reference compound).The technology is based in 3D flexible superposition which considers 22 molecular interaction fields and has proved to be excellent for scaffold hopping.The drug database comes from a property library of products that have reached clinical or approved status.SOM was founded in 2009 and has already done more than 28 repurposed programs,with an 18% of success rate.Strategy:Through this approach SOM has focused in rare diseases because of the great medical need and because by repurposing drugs it is possible to reach fast the clinical proof of concept,and thus,the market sooner than with new chemical entities,a condition that is much appreciated in rare diseases which are normally life threating.We have focused in rare diseases as: Amyloidosis by TTR (ATTR),Huntington Disease,Phenylketonuria,Cystic Fibrosis,Amyotrophic Lateral Sclerosis,Acute Myeloid Leukemia,Glioma Multiforme,Spinbulbar Muscular Atrophy.Case Studies:We will present the case study of SOM0226,a repositioned product for the prevention and treatment of Transthyretin Amyloidosis (ATTR) which has jumped to Phase 2 proof of concepts in just two years after its discovery and is four times more potent/active than the most advanced new chemical entity (Tafamidis).Also,the case of SOM3355 for the treatment of chorea in Huntington patients will be discussed;a repurposed product with a better safety and compliance than the gold standard (Tetrabenazine).In conclusion drug reprofiling in rare indications can be a good strategy to reach the patients sooner,cheaper and with a better competitive price than standard approach.Advantages and disadvantages of this approach will also be discussed.