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August 5, 2014
09:29 EDTRAREUltragenyx announces license agreement with UniQuest, new U.S. patent
Ultragenyx Pharmaceutical announced a license agreement with UniQuest Pty Limited for intellectual property related to the treatment of refractory epilepsy and other seizure-related and neurologic disorders with triheptanoin. The intellectual property originated from research on epilepsy and other neurologic models conducted at The University of Queensland. Separately, a United States patent has been issued with claims directed to compositions of triheptanoin above a certain level of purity. The patent term expires in October 2025. A second U.S. patent with claims directed to compositions of triheptanoin in a dosage unit was issued in August 2013 and expires in June 2020. These patent terms do not include any potential for patent term extension of up to five additional years. Similar claims are either granted or being pursued by Ultragenyx in other territories outside the U.S.
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September 15, 2014
09:04 EDTRAREUltragenyx announces positive results from Phase 1/2 study of KRN23
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September 3, 2014
09:14 EDTRAREUltragenyx announces positive interim data from Phase 1/2 study of rhGUS
Ultragenyx Pharmaceutical announced the presentation of positive interim data from the Phase 1/2 study of recombinant human beta-glucuronidase, or rhGUS, UX003, an investigational therapy for the treatment of mucopolysaccharidosis 7, or MPS 7, Sly syndrome. The data are being presented at the Society for the Study of Inborn Errors of Metabolism, or SSIEM, Annual Symposium in Innsbruck, Austria. The Phase 1/2 open-label clinical study is assessing the safety, efficacy, and dose of rhGUS administered every other week via intravenous infusion in three patients. A 12-week primary analysis phase evaluating 2 mg/kg of rhGUS every other week is being followed by dose-exploration and long-term extension.

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