Disc Medicine, Inc. presented Phase II data for bitopertin in erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) showing consistent and sustained suppression of a metabolite that can cause those two hematological diseases – potentially confirming a new purpose for a neuropsychiatric drug candidate that Roche Holding AG studied in schizophrenia roughly a decade ago. As a result, patients in the Disc’s mid-stage study are showing what the company calls “profound” improvements in sunlight tolerance.
Analysts lauded the findings following the readout on 9 June at the European Hematology Association 2023 conference in Germany, even though the data are an interim readout from a small open-label study