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A clinical-stage drug discovery and development company based in Barcelona, Spain, is offering a repurposed drug for the treatment of chorea movements associated with Huntington’s disease. It is a small molecule that is currently undergoing Phase 2a proof-of-concept. The company is looking for license agreements.
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.
The technology is a repurposed drug with an already established safety profile for its primary indication. Compared to the available treatments for Huntington’s disease on the market, which have black box warning, it is safe and may also be used in Huntington’s disease patients with neurological afflictions such as depression, psychosis and aggressive behaviour.
The AI platform has demonstrated success through the company’s first asset for Transthyretin Amyloidosis which reached Phase 2a completion in five years since its discovery in 2011 and was licensed for worldwide rights to a US-based pharmaceutical company. Recently, the technology has yet again proven its predictive accuracy through the Phase 2a milestone achieved by the company’s second asset for Huntington’s disease in the 3rd quarter of 2018.
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The company seeks to collaborate with biotechnology and/or pharmaceutical companies that wish to expand their pipeline through licensing the company’s clinical- or preclinical-stage assets.