Ultragenyx Announces Positive Topline Results and DMC Review from First Cohort of Phase 1/2 Clinical Study of DTX301, an Investigational Gene Therapy in Ornithine Transcarbamylase (OTC) De...
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
Ureagenesis normalized in one patient and further increased by 24 weeks Cohort 2 patient enrollment to begin in March 2018; data expected in second half of 2018 NOVATO, Calif., March 07, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced positive longer-term safety and efficacy data from the first dose cohort of the Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. “Longer term data from the first, lowest-dose cohort show that patient 1 maintained and substantially increased levels of ureagenesis through 24 weeks and we view these results as a promising indication of the potential and durability of DTX301. Based on these positive results at 24 weeks, this patient opted to discontinue all alternate pathway medication three weeks ago per t
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News
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) had its price target lowered by analysts at Canaccord Genuity Group Inc. from $111.00 to $109.00. They now have a "buy" rating on the stock.MarketBeat
- Here's What Analysts Are Forecasting For Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) After Its First-Quarter Results [Yahoo! Finance]Yahoo! Finance
- Ultragenyx (RARE) Q1 Loss Wider Than Expected, Revenues Rise Y/Y [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) had its "overweight" rating re-affirmed by analysts at Cantor Fitzgerald. They now have a $107.00 price target on the stock.MarketBeat
- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) had its price target lowered by analysts at Wedbush from $48.00 to $47.00. They now have a "neutral" rating on the stock.MarketBeat
RARE
Earnings
- 5/2/24 - Miss
RARE
Sec Filings
- 5/3/24 - Form 4
- 5/3/24 - Form 10-Q
- 5/2/24 - Form 8-K
- RARE's page on the SEC website