- Ultragenyx Pharmaceutical Inc RARE has announced longer-term data from the Glycogen Storage Disease Type Ia (GSDIa) and Ornithine Transcarbamylase (OTC) Deficiency Phase 1/2 studies. Data were presented at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting.
- DTX401 (GSDIa) Program: Longer-term Phase 1/2 data demonstrate the durability of response, with sustained responses lasting more than 2.5 years since treatment.
- All nine patients continue to demonstrate improved glucose control while tapering or discontinuing oral glucose replacement with cornstarch and improving energy metabolism pathways.
- All patients in the prophylactic cohort in the Phase 1/2 study have demonstrated early, clinically meaningful cornstarch reductions ranging from 64% to 83%.
- Phase 3 study of DTX401 in GSDIa is expected to initiate early in the second half of 2021.
- DTX301 (OTC) Program: Longer-term Phase 1/2 data show durable metabolic control and sustained responses lasting more than three years since treatment.
- The six patients who previously demonstrated a response remain clinically and metabolically stable, including all three treated at the highest dose (1.7 x 10^13 GC/kg dose).
- DTX301 Phase 3 study will start in the second half of 2021.
- Across all cohorts of the study, no infusion-related adverse events and no treatment-related serious adverse events were reported. All treatment-related adverse events have been mild or moderate.
- The company also announced that its next-gen HeLa producer cell line manufacturing platform resulted in significant product yield increases.
- Price Action: RARE shares are up 3.26% at $110.19 during the market session on the last check Friday.
Loading...
Loading...
RAREUltragenyx Pharmaceutical Inc
$35.51-%
Edge Rankings
Momentum
24.66
Growth
Not Available
Quality
Not Available
Value
20.35
Price Trend
Short
Medium
Long
© 2025 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
Benzinga simplifies the market for smarter investing
Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.
Join Now: Free!
Already a member?Sign in
