EMA Approves Oryzon's Application for Phase II HOPE-2 Study in Phelan-McDermid Syndrome

The European Medicines Agency (EMA) has approved Oryzon Genomics' clinical trial application (CTA) for the Phase II HOPE-2 study. This single-arm, open-label trial will evaluate vafidemstat in 12 adult patients with Phelan-McDermid syndrome (PMS) in Spain. Vafidemstat is an LSD1 inhibitor in clinical development for central nervous system disorders, with preclinical findings suggesting it could modulate neural plasticity, neuroinflammation, and neuronal excitability.