The Spanish company has developed an efficient and predictive artificial intelligence (AI) technology for drug reprofiling.
Through its AI technology, the company has built its own pipeline of assets in the Central Nervous System (CNS) orphan disease space—the company’s therapeutic focus.
The company’s pipeline consists of clinical-stage assets for Huntington’s disease and Adrenoleukodystrophy, as well as preclinical-stage assets for Adrenomyeloneuropathy, Phenylketonuria and Niemann-Pick disease.
The current most advanced asset is for Huntington’s disease which is in Phase 2a, with preliminary clinical data expected in September 2019.
The technology is a VMAT-2 inhibitor being developed as a prevention and treatment of chorea movements associated with Huntington’s disease. It may also be used for hyperkinetic movement disorders associated with Tourette’s syndrome, tardive dyskinesia and hemiballism.
The company’s business model involves drug discovery, preclinical/clinical development and out-licensing after Phase 2a proof-of-concept. The company is seeking pharmaceutical and biotechnology companies for a license agreement and is flexible with reference to deal structure, depending on the prospective licensee.