The biopharmaceutical company Oryzon Genomics, a member of Catalonia.health, has announced its participation as a sponsor of the first-ever Phelan-McDermid syndrome (PMS) burden of illness study. The study is led by CureShank, a research advocacy organization founded by families of individuals affected by PMS, whose mission is to accelerate the development of treatments for the disorder.
This study, recently launched, will collect data from families of individuals living with PMS, health insurance claims and insights from clinical experts, to help quantify the true economic impact of PMS, inform the development of new therapies, and guide future market access strategies.
The company is preparing a Phase II trial in people with PMS, expected to start in the coming months, with its LSD1 inhibitor. This study, named HOPE-2, will evaluate the safety and efficacy of vafidemstat in PMS. HOPE-2 will be conducted in the European Union and will be partially financed by funds received under the EU IPCEI MED4CURE grant in 2025.
Jordi Xaus, Oryzon’s Chief Scientific Officer, explained: “Consistent with Oryzon’s commitment to rare diseases, we are proud to support CureShank’s important initiative. This study not only evaluates the burden of Phelan-McDermid syndrome but also helps identify the key drivers of that burden - critical insights that can guide new interventions and accelerate the development of much-needed treatments to improve the quality-of-life to patients and their families”.
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