Ultragenyx's Gene Therapies For Genetic Metabolic Disorders Show Longer-Term Durable Responses

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.

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