A couple of comments on Ultragenyx’s planned Phase 3 trial -
The primary endpoint accepted by the FDA for this Phase 3 trial is improvement in cognition assessed by the Bayley-4 cognitive raw score. Key secondary endpoint will be the Multi-domain Responder Index (MDRI) across all five domains of cognition, receptive communication, behaviour, gross motor function, and sleep.
Assessment scales selected by Neuren for its Phase 2 trial were quite different from those used by Ultragenyx in its Phase 2 trial and did not include these two accepted endpoints.
Angelman syndrome happens when part of the maternal copy is either deleted or damaged. An estimated 70% of diagnosed AS cases occur due to gene deletion. The other 30% of diagnosed cases relate to a mutation/variant in the maternal copy of the UBE3A gene or are of unknown cause.
Ultragenyx is including only those patients who have documented genetic confirmation of full maternal UBE3A gene deletion causing Angelman syndrome in the region of 15q11.2-q13.
Neuren’s Phase 2 study included those who had clinical diagnosis of AS with a documented disease-causing genetic etiology known to impact maternally derived UBE3A expression in brain.
Ultragenyx has also met with the European Medicines Agency, receiving acceptance of the overall Phase 3 study design, dosing and evaluations. A further meeting with Japan’s Pharmaceuticals and Medical Devices Agency to inform and discuss the Phase 3 study design is expected in the coming weeks .
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