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Retrophin, Inc. (RTRX) To Secure Development And Commercialization Rights On Promising New Autism, Schizophrenia Treatment

Retrophin, the biopharma developer focused on debilitating and life-threatening neurological diseases in unmet and underserved categories, announced signing a deal today which grants the company a 120-day exclusivity window in which to negotiate a royalty-bearing U.S. license for the intellectual property needed to develop, manufacture, and sell an exciting new Autism and Schizophrenia treatment. The deal was signed with an undisclosed, major pharmaceutical company in exchange for an upfront, non-refundable fee, as well as obligation to pay an additional fee upon execution of a suitable license agreement.

For a company that has already pioneered extensive territory in neurological disease, this deal is a major victory. With four current treatments in various stages of development, typified by their lead compound, RE-021 (originally developed for hypertension), which is designed to treat a rare and severe (mostly pediatric) kidney nephropathy affecting some 50k patients in the U.S. alone. This license would put RTRX in control of a potentially break out product that could offer new and significant therapeutic options to patients that suffer from schizophrenia and autism spectrum disorders. With RE-021 slated to start enrollment in a pivotal Phase 2 clinical trial this year, securing this new product license (especially in so broad a category as autism spectrum disorders) is huge news that could even dwarf the rest of the company's portfolio, despite how good it looks.

President, Founder and CEO of RTRX, Martin Shkreli, emphasized the abundant extant data from numerous studies conducted using the product and assured investors that the company's own rigorous due diligence confirmed an extremely high probability here for "significant utility" in the given indications.

Retrophin looks to be the perfect company to pick up the ball and run with it here given their established presence in childhood neurological disease. The company's other three products currently under development are:

RE-001 - designed to combat the most common and most severe form of muscular dystrophy, Duchenne Muscular Dystrophy, which affects nearly 1 in 3.6k male newborns

RE-003 - a recombinant fusion protein targeting the most common genetic cause of infant mortality, Spinal Muscular Atrophy, which affects some 112k infants in the U.S. and Europe alone

RE-024 - which has shown nice results in preclinical animal models at St. Jude's Children's Research Hospital as a substitute for phosphopantothenate therapy used to treat pantothenate kinase-associated neurodegeneration, a rare but life-threatening neurological disorder resulting in the inability to metabolize vitamin B5

With solid data on the benefits of this new product for autistic people and patients suffering from schizophrenia to go off of, RTRX should make short work of the development and commercialization cycle, meaning Retrophin could soon have a winning autism spectrum disorder drug ready for market. Taken into consideration alongside the rest of RTRX's portfolio, investors will want to keep a close eye on this deal and how the company progresses with the execution of the license.

For more information on Retrophin, visit

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