Shots:
- Ultragenyx has completed enrolment in its P-III (Aspire) trial to assess GTX-102 (apazunersen) in Angelman syndrome pts with confirmation of full maternal UBE3A gene deletion; Aurora study in more ages & genotypes to begin in H2’25
- Trial will evaluate GTX-102 (intrathecal, 8mg QM for 3mos., then up to 14mg Q4M) vs a sham treatment in Angelman syndrome pts (n=129, 4-17yrs.), which may cross over to treatment after completing Wk. 48 assessments; trial is expected to complete by H2’26
- Trial will evaluate cognitive improvement per Bayley-4 cognitive raw score as the 1EP, with 2EPs incl. Multi-domain Responder Index (MDRI) across cognition, receptive communication, behavior, motor function, & sleep
Ref: Globenewswire| Image: Ultragenyx| Press Release
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