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Ultragenyx Pharmaceutical Inc. (RARE)

NasdaqGS - NasdaqGS Real Time Price. Currency in USD
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101.71+2.35 (+2.37%)
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Previous Close99.36
Open100.21
Bid101.75 x 1100
Ask111.32 x 800
Day's Range98.76 - 102.17
52 Week Range66.14 - 179.65
Volume676,597
Avg. Volume450,748
Market Cap6.864B
Beta (5Y Monthly)1.91
PE Ratio (TTM)N/A
EPS (TTM)N/A
Earnings DateN/A
Forward Dividend & YieldN/A (N/A)
Ex-Dividend DateN/A
1y Target EstN/A
Fair Value is the appropriate price for the shares of a company, based on its earnings and growth rate also interpreted as when P/E Ratio = Growth Rate. Estimated return represents the projected annual return you might expect after purchasing shares in the company and holding them over the default time horizon of 5 years, based on the EPS growth rate that we have projected.
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  • Ultragenyx's Gene Therapies For Genetic Metabolic Disorders Show Longer-Term Durable Responses
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    Ultragenyx's Gene Therapies For Genetic Metabolic Disorders Show Longer-Term Durable Responses

    Ultragenyx Pharmaceutical Inc (NASDAQ: 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. See more from BenzingaClick here for options trades from BenzingaAffimed's AFM13 In Combination With NK Cells Show Improved Tumor Recognition, Killing In Preclinical StudiesHumanigen Shares Gain On FDA Emergency Filing Plans For COVID-19 Drug By June© 2021 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.