Ultragenyx reports positive Phase 1/2 data for GTX-102 in Angelman syndrome; Phase 3 Aspire study well powered

Ultragenyx Pharmaceutical Inc.

Week 48 Bayley-4 Cognition GSV mean change +6.7 vs minimally important difference +5 (n=40).
Phase 3 primary endpoint Bayley-4 Raw score mean change +10.9 from baseline.
Study >95% powered to detect treatment effect even with up to 3x higher sham response.
MDRI net response +2.0 (p<0.0001); 80% of patients achieved net improvement in at least one domain.
Phase 3 Aspire will enroll ~120 patients with full maternal UBE3A deletion; 48-week primary analysis.

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