ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE) Files An 8-K Regulation FD Disclosure

ULTRAGENYX PHARMACEUTICAL INC. (NASDAQ:RARE) Files An 8-K Regulation FD Disclosure
Item 7.01

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Regulation FD Disclosure.

On January 7, 2019, Ultragenyx Pharmaceutical Inc. (the “Company”) posted an updated Corporate Presentation to its website at www.ultragenyx.com in the “Events & Presentations” subsection of the “Investors” tab.

The information set forth under Item 7.01 and in the Corporate Presentation shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, except as shall be expressly set forth by specific reference in any such filing.

On January 4, 2019, the Company issued a press release (the “Release”) announcing positive topline results from the first cohort of the Phase 1/2 clinical study of DTX401, a gene therapy for the treatment of glycogen storage disease type Ia.

A copy of the Release is filed as Exhibit 99.1.

Item 7.01

Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.

Description

99.1

Press Release, dated January 4, 2019

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Ultragenyx Pharmaceutical Inc. Exhibit
EX-99.1 2 rare-ex991_6.htm EX-99.1 rare-ex991_6.htm Exhibit 99.1 Contact Ultragenyx Pharmaceutical Inc. Investors & Media Danielle Keatley 415-475-6876   Ultragenyx Announces Positive Topline Results from First Cohort of Phase 1/2 Clinical Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia   DTX401 Response Observed in All Three Patients,…
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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.

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