Results from a murine model investigating bitopertin for the treatment of EPP

Mark Fleming, MD, Boston Children’s Hospital, Boston, MA, presents the findings from a mouse model which was used to investigate the potential use of bitopertin for the treatment of erythropoietic protoporphyria (EPP). Patients with EPP are hypersensitive to the sun and experience an accumulation of protoporphyrin IX (PPIX) in the blood, which can lead to fatal liver disease. Results from this study demonstrated that bitopertin can reduce PPIX accumulation and liver damage, and this agent will soon be explored in a Phase I trial. This interview took place at the 64th ASH Annual Meeting and Exposition congress held in New Orleans, LA.

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