Drug has shown potential in Friedreich ataxia and Alzheimer disease.

ViroPharma is paying Intellect Neurosciences $6.5 million up front for rights to its clinical-stage OX1 candidate, which is being developed for the treatment of Friedreich ataxia (FA). Under terms of the worldwide, exclusive deal ViroPharma will be able to develop and commercialize OX1 for the treatment, management, or prevention of any disease or condition covered by Intellect’s patents.

ViroPharma will also pay milestones that could amount to $120 million assuming successful advancement to market. The company will also pay a tiered royalty of up to a maximum percentage of low teens, based on annual net sales.

OX1, or indole-3-propionic acid (IPA), is a naturally occurring small molecule that has potent antioxidant properties that can protect against neurodegenerative disease, according to the companies. It is copper-binding drug compound that prevents oxidative stress and blocks formation of toxic Aß aggregates. Intellect Neuroscience says that it was developing OX1 as a disease-modifying treatment for AD and was working on establishing proof-of-concept for other diseases including Wilson disease.

In a recent Phase I study conducted in The Netherlands, OX1 was demonstrated to be safe and well tolerated at all dose levels tested. ViroPharma expects to initiate a Phase II study within 12 to 18 months after completion of longer-term toxicology studies. ViroPharma intends to file for orphan drug designation upon review of the Phase II data.

ViroPharma has two commercial products: Cinryze for hereditary angiodema and Vancocin for Clostridum difficile. Cinryze is also being developed for pediatric use in hereditary angiodema and as a subcutaneous formulation. Both programs are at the Phase II stage. ViroPharma’s lead investigational candidate is buccal midazolam for seizures in children and adolescents.

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