CHDI Foundation is a privately-funded nonprofit biopharmaceutical research and development organization that is exclusively dedicated to developing therapies for Huntington’s disease (HD), a lethal autosomal dominant neurodegenerative disorder caused by expansion of CAG repeats in the huntingtin (HTT) gene. HD patient brains reveal a devastation of the caudate-putamen, and cortico-striatal-thalamo-cortical circuits seem to be particularly affected. Currently, no disease-modifying therapies are available. CHDI works collaboratively with academic institutions, contract research organizations (CROs) and biotechnology and pharmaceutical companies, with a general strategy of developing or de-risking therapeutic programs to the point where pharmaceutical companies will take them to market. A CHDI Outreach Team works to leverage pharmaceutical companies’ knowledge and assets that could be relevant to HD pathophysiology and provide our partners access to both CHDI’s domain knowledge as well as extensive in vitro and in vivo HD-relevant assays. Alterations in neuronal and synaptic function may underlie many of the early symptoms of HD; deficits in corticostriatal transmission have been well documented and are one of the earliest changes detected in HD rodent models. To investigate various phosphodiesterase (PDE) inhibitors and how they affect corticostriatal transmission and basal ganglia function, our Outreach Team investigated several advanced PDE inhibitors (PDE10i, PDE9i and PDE2i) in a collaboration with Pfizer. We will present our outreach strategy and an overview of our Pfizer collaboration, which has culminated in phase 2A and B clinical trials with a PDE10 inhibitor (PF-25454920) in HD patients.

Foundation-directed therapeutic development: Pfizer collaboration on PDE inhibitors

249th National Meeting (2015)

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