ORYZON to Sponsor First Phelan-McDermid Syndrome (PMS) Burden of Illness Study

Core Insights - Oryzon Genomics is sponsoring the first Phelan-McDermid syndrome (PMS) burden of illness study, led by CureShank, to assess the economic impact of PMS and guide therapy development [1][2][3] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics, particularly in CNS disorders and oncology [6] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [6] Study Details - The PMS study aims to collect data from families, health insurance claims, and clinical experts to quantify the economic burden of PMS and inform future market access strategies [2][8] - The study will help identify key drivers of the burden, which can guide new interventions and accelerate treatment development [3] Disease Context - PMS is a neurodevelopmental disorder caused by mutations in the SHANK3 gene, leading to developmental delays, intellectual disabilities, and behavioral issues [4] - There are currently no approved pharmacological treatments for PMS, highlighting the need for effective therapies [4] Clinical Trials - Oryzon is preparing a Phase II trial named HOPE-2 to evaluate the safety and efficacy of vafidemstat in PMS, expected to start soon [5] - Vafidemstat has shown promise in preclinical models for reversing social-behavior and aggression phenotypes associated with ASD [5][7]