RARE's Phase III OTC Deficiency Study Data in Detail [Yahoo! Finance]
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: Yahoo! Finance
The candidate is designed to provide durable expression and activity of OTC following a single intravenous infusion. DTX301 enjoys the Orphan Drug designation in the United States and the EU and the Fast Track designation in the United States for treating OTC deficiency. Ultragenyx Pharmaceutical's phase III Enh3ance study enrolled 37 patients who were randomized equally to receive either DTX301 or placebo during the 36-week randomized control period. After this period, the study will be unblinded, and patients in the placebo arm will crossover to receive treatment. RARE's Phase III OTC Deficiency Study Data in Detail Per the data readout, at week 36 of the controlled phase of the Enh3ance study, patients treated with DTX301 achieved a statistically significant and clinically meaningful 18% reduction in 24-hour plasma ammonia compared with placebo, meeting one of the primary endpoints. Average ammonia levels among treated patients remained within the normal range through week 3
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News
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- RARE Shareholder Alert: Ultragenyx Pharmaceutical Inc. Securities Class Action Lawsuit Investors With Losses May Join — The Gross Law FirmGlobeNewswire
- RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud LawsuitPR Newswire
- RARE Investors Have Opportunity to Lead Ultragenyx Pharmaceutical Inc. Securities Fraud Lawsuit with the Schall Law FirmGlobeNewswire
RARE
Earnings
- 2/12/26 - Miss
RARE
Sec Filings
- 3/12/26 - Form 8-K
- 3/10/26 - Form 4
- 3/10/26 - Form 144
- RARE's page on the SEC website