ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE) Files An 8-K Other EventsItem 8.01.Other Events.
On August 22, 2017, Ultragenyx Pharmaceutical Inc. (the “Company”) issued a press release announcing top-line results from the Phase 3 study of Ace-ER in GNE Myopathy (the “Ace-ER Release”).
On August 24, 2017, the Company issued a press release announcing the submission of a Biologics License Application with the Food and Drug Administration for Burosumab (the “BLA Release”).
A copy of the Ace-ER Release is filed herewith as Exhibit 99.1 and a copy of the BLA Release is filed herewith as Exhibit 99.2.
Item 9.01Financial Statements and Exhibits
(d) Exhibits
Exhibit No.Description
99.1Press Release, dated August 22, 2017
99.2Press Release, dated August 24, 2017
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Ultragenyx Pharmaceutical Inc. ExhibitEX-99.1 2 rare-ex991_6.htm EX-99.1 rare-ex991_6.htm Exhibit 99.1 Contact Ultragenyx Pharmaceutical Inc. Investors & Media Ryan Martins 415-483-8257 Ultragenyx Announces Top-Line Results from Phase 3 Study of Ace-ER in GNE Myopathy Study did not meet its primary endpoint Novato,…To view the full exhibit click here
About ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE)
Ultragenyx Pharmaceutical Inc. is a clinical-stage biopharmaceutical company. The Company is focused on the identification, acquisition, development, and commercialization of products for the treatment of genetic diseases. The Company is engaged in the identification, acquisition, development and commercialization of products for the treatment of rare and ultra-rare diseases segment. Its pipeline consists of two product categories: biologics, including a monoclonal antibody and enzyme replacement therapies, and small-molecule substrate replacement therapies. KRN23 is a fully human monoclonal antibody administered via subcutaneous injection. Recombinant human beta-glucuronidase (rhGUS), is an intravenous (IV), enzyme replacement therapy for the treatment of mucopolysaccharidosis 7 (MPS 7). It is developing UX007 for oral administration intended as a substrate replacement therapy. It is developing aceneuramic acid extended-release (Ace-ER) for the treatment of GNE myopathy.