Oryzon has received European authorisation to initiate a Phase IIa study of vafidemstat in Phelan McDermid Syndrome (PMS). The study has been designed to enrol 12 adult patients at one Spanish site, with safety and tolerability as the primary focus over 12 weeks of treatment. Changes in anger, aggression and other symptoms are secondary measures, offering an early indication of activity in a condition with no approved drugs at present. The rationale draws on findings in preclinical research in mice lacking SHANK3, and earlier exploratory clinical data in autism spectrum disorder. The study is partially funded through Med4Cure, and a Spanish patient association will help identify participants.
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