Sparks - Oryzon Genomics

Oryzon Genomics has announced that it will sponsor the first Phelan-McDermid syndrome (PMS) burden of illness study. The study, which was recently launched, and is being led by CureShank (a research advocacy organisation focused on expediting life-changing therapies for PMS), is collecting data from families of PMS patients, health insurance companies and taking insights from clinical experts. The ultimate aim is to quantify the economic impact of PMS, while informing the development of new treatment options, and guiding future market access strategies.

Oryzon’s lead CNS drug candidate vafidemstat has been designed to address agitation and aggression, which is a common feature of PMS. Since there are currently no approved pharmacological treatments for PMS, there could be a sizable opportunity for Oryzon in this space. The company plans to launch its Phase II HOPE-2 trial in PMS patients by end-2025, partially financed by funds received under the EU IPCEI MED4CURE grant.