We have just learned that the phase 3 Aspire study for GTX-102 (apazunersen) in Angelman syndrome did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (MDRI).
As a community, we are disappointed by this news. Although this outcome is not what any of us hoped for, it does not change our commitment to helping our loved ones living with AS – a goal we share with the entire Angelman syndrome community around the world.
Clinical research is only possible because families are willing to step forward. Your contribution to this field is profound, and we are deeply grateful.
We have requested a community webinar from Ultragenyx, and will release any related information as soon as we have it.
If you are currently participating in an Ultragenyx clinical trial, please contact the physician who leads the trial (PI) with any questions and to discuss next steps.
If you have questions about the trial and this data, please contact patientinquiry@ultragenyx.com.