Ultragenyx reports positive longer-term Phase 3 data for DTX401 gene therapy in GSDIa

Ultragenyx Pharmaceutical Inc.

DTX401 met primary endpoint at Week 48: mean cornstarch reduction 41% vs 10% placebo (p<0.0001).
At Week 96, DTX401 and crossover groups each achieved mean 61% reduction in daily cornstarch from baseline.
Two-thirds of participants eliminated at least one nighttime cornstarch dose; fasting tolerance improved through Year 2.
83% of DTX401 group and 95% of crossover reported improved disease burden at Week 96 per PGIC.
Open-label cohort (n=3) achieved 95% mean cornstarch reduction at Week 24; all discontinued cornstarch by Week 36.

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