Ultragenyx Announces Preliminary Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7

Ultragenyx Announces Preliminary Data From Phase 1/2 Study of Recombinant Human Beta-Glucuronidase in Mucopolysaccharidosis 7

Data Presented at American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting

NOVATO, Calif., March 27, 2014 (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 the presentation of preliminary data from the Phase 1/2 study of recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome).

Ultragenyx Announces First Patient Enrolled in Phase 2 Study of Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome

Novato, CA, March 11, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced the first patient enrolled in the Phase 2 study of triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS), at Columbia University. Glut1 DS, also known as De Vivo disease, is a rare, severely debilitating disease characterized by seizures, developmental delay, and movement disorder.

Ultragenyx Announces Presentation of Data From a Single Patient Treated With Recombinant Human Beta-Glucuronidase at 10th Annual World Lysosomal Disease Network Symposium

NOVATO, Calif., Feb. 12, 2014 (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 data presentations from a single patient treated with recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis 7 (MPS 7, Sly syndrome).

Ultragenyx Announces Initiation of Phase 2 Study for Patients with Long-Chain Fatty Acid Oxidation Disorders

Open-label study to assess safety and clinical effects of triheptanoin

NOVATO, CA – February 11, 2014 – Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that the first patient has been enrolled in an open-label Phase 2 study to assess safety and clinical effects of triheptanoin, also known as UX007, in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to break down and convert long chain fatty acids into energy.

Ultragenyx Appoints Clay Siegall, Ph.D. and Matthew Fust to Board of Directors

NOVATO, Calif., Feb. 6, 2014 (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 the appointment of Clay B. Siegall, Ph.D. and Matthew Fust to the company’s Board of Directors effective January 30, 2014. Dr. Siegall, currently President, Chief Executive Officer and Chairman of the Board of Seattle Genetics, Inc., and Mr. Fust, formerly Executive Vice President and Chief Financial Officer of Onyx Pharmaceuticals, Inc. will serve as independent directors for Ultragenyx. With these additions, Ultragenyx extends gratitude and appreciation for the counsel provided by Mr. Ben Auspitz, who has resigned from his position on the Board effective January 30, 2014.

Ultragenyx Announces Closing of Initial Public Offering and Exercise of Underwriters’ Option to Purchase Additional Shares

NOVATO, Calif., Feb. 5, 2014 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the closing of its initial public offering of 6,624,423 shares of common stock at an initial public offering price of $21.00 per share, which includes the exercise in full by the underwriters of their option to purchase up to 864,054 additional shares of common stock. All of the shares of common stock were offered by Ultragenyx. The company’s common stock is listed on The NASDAQ Global Select Market under the ticker symbol “RARE.” Ultragenyx estimates net proceeds from the offering to be approximately $126.4 million, after deducting underwriting discounts and commissions and estimated offering expenses.

Ultragenyx Announces Pricing of Initial Public Offering

NOVATO, CA – January 30, 2014 – Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced the pricing of its initial public offering of 5,760,369 shares of its common stock at an initial public offering price of $21.00 per share, before underwriting discounts. The shares are expected to begin trading on The NASDAQ Global Select Market on January 31, 2014 under the ticker symbol “RARE.” All of the shares of common stock are being offered by Ultragenyx. In addition, Ultragenyx has granted the underwriters a 30-day option to purchase up to an additional 864,054 shares of common stock to cover over-allotments, if any.

Ultragenyx Announces Results from Phase 2 Study of Sialic Acid Extended-Release Treatment in Hereditary Inclusion Body Myopathy

Upper extremity muscle strength composite shows statistically significant difference between the 6 gram and 3 gram dose groups

Novato, CA — December 20, 2013 — Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline results from a 48-week Phase 2 clinical study of sialic acid extended-release (SA-ER, UX001) tablets in 46 patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disease. SA-ER is designed to replace the deficient sialic acid substrate in patients with HIBM. Patients were initially randomized to either receive placebo, 3 grams or 6 grams of SA-ER per day. After 24 weeks, placebo patients crossed over to either 3 grams or 6 grams total daily dose, on a blinded basis, for an additional 24 weeks. The final analysis compared change at week 48 from baseline for the combined groups at 6 grams versus 3 grams of SA-ER.

Ultragenyx Investigational New Drug Application for Triheptanoin for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome is in Effect

Phase 2 Clinical Study Expected to Begin in the First Half of 2014
NOVATO, CA – December 17, 2013 – Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, announced today that its Investigational New Drug (IND) application
filing for triheptanoin (UX007) for the treatment of glucose transporter type-1 deficiency syndrome (Glut1 DS) is in effect. The IND allows the company to proceed with its clinical development program for triheptanoin in Glut1 DS in the United States. The company plans to initiate a Phase 2 trial studying triheptanoin in Glut1 DS in the first half of 2014.

Ultragenyx Announces the Presentation of Data from a Single Dose Phase 1 Study, Conducted by Kyowa Hakko Kirin Co. Ltd. (KHK), of KRN23 in X-linked Hypophosphatemia (XLH) in Adults

KRN23 increased phosphate levels and was safe and well tolerated
NOVATO, CA – October 6, 2013— Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of data from a first-in-human, randomized, double-blind, placebo-controlled, single dose study of a human monoclonal anti-FGF23 antibody (KRN23) in X-linked hypophosphatemia in adults. X-linked hypophosphatemia (XLH) is an inherited metabolic bone disease characterized by short stature and skeletal deformities. A Phase I study (US-02) was conducted by KHK with KRN23 to assess its safety and tolerability and to measure changes in biochemical markers in adult patients with XLH. Thirty-eight adults with XLH were randomized to receive single doses of KRN23 or placebo via intravenous (IV) (0.003 to 0.3 mg/kg) or subcutaneous (SC) (0.1 to 1.0 mg/kg) routes.