FierceBiotech selected Ultragenyx Pharmaceutical as one of the 2013 Fierce 15 Companies.

What makes Ultragenyx fierce: The most recent addition to the pipeline is KRN23, an antibody designed to treat rare cases of X-linked hypophosphatemia that is now wrapping a Phase I/II study at Kyowa Hakko Kirin. At the time KRN23 showed up on Ultragenyx's radar, it was the only rare-disease program at the Japanese pharma company, says CEO Emil Kakkis. When the deal was done just days ago, it was Ultragenyx's fourth midstage therapy.

 
Treatment with triheptanoin appears to reduce the frequency and length of hospitalizations associated with LC-FAOD disease.

NOVATO, CA –September 16, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company, announced the release of positive data from a retrospective protocol-driven medical record review oftriheptanoin treatment of patients with long-chain fatty acid oxidation disorders(LC-FAOD). LC-FAODpatients have severe morbidity and mortality with frequent complications and hospitalizations. The study evaluated the impact of triheptanoin treatment on the rate and extent of hospitalizationsin 20 of 24 patients who have been treated with triheptanoin for up to 13 years as part of a compassionate use protocol and consented to be part of the retrospective study. The study compared the incident rate for the major medical events before and after triheptanoin treatment, including the total number of hospitalizations and hospital days per year due to all causes,muscle rupture (rhabdomyolysis), hypoglycemia, or cardiomyopathy.

 
Exclusive partnership will advance global clinical development of
KRN23, an antibody for a rare bone disease

Novato, CA – September 3, 2013--Ultragenyx Pharmaceutical Inc.today announced it has entered into a collaboration and license agreement with Kyowa Hakko Kirin Co., Ltd. (KHK),to develop and commercialize KRN23. KRN23 is a recombinant fully human monoclonal IgG1 antibody intended to treat X-linked hypophosphatemia (XLH). KHK is currently completing a Phase 1/2 study in adults with XLH in the US and Canada. The two companies plan to initiate a pediatric XLH program in 2014.

 
Ultragenyx Receives Approval of Clinical Trial Application (CTA)
for a Phase 1/2 Trial TestingUX003 in Mucopolysaccharidosis Type 7 (MPS 7)

NOVATO, CA – August 14, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company, received Clinical Trial Application (CTA) approval from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the U.K. to conduct a Phase 1/2 clinical trial of UX003, recombinant human β-glucuronidase, in MPS 7, a rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the enzyme β-glucuronidase. MPS 7 is a severe multi-system disease resulting in cellular and organ dysfunction. There is no approved drug therapy.

 
Option Exercised for Ex-North American Territories

Novato, CA –July 11, 2013--Ultragenyx announced that it has expanded its exclusive license from Baylor Research Institute (BRI)in Dallas, Texas,to develop and commercialize triheptanoin outside of North America. The global license includes rights to patents, patent applications and other intellectual property related to the composition and formulation of UX007 as well as its use in treating a number of diseases including fatty acid oxidation disorders (FAOD), the lead indication being developed by the company.

 
Study to continue to 48 weeks, followed by extension study testing higher dosage

Novato, CA—July 3, 2013—Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, announced interim 24-week data from a 48-week Phase 2 clinical study of UX001 in 47 patients with hereditary inclusion body myopathy (HIBM), a progressive muscle-wasting disease. The study compared treatment with a total daily dose of 6 grams or 3 grams of UX001 with placebo. UX001, an oral sialic acid extended-release (SA-ER) tablet, is designed to replace the deficient sialic acid substrate in patients with HIBM.

 
NOVATO, CA – May 15, 2013 - Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced a Phase 1/2 study of UX003 for mucopolysaccharidosis type 7 (MPS 7, or Sly Syndrome). UX003 is a recombinant human β-glucuronidase intended as an enzyme replacement therapy (ERT) for the treatment of MPS 7, an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme β-glucuronidase and a severe multi-system disease. MPS 7 has no approved therapies.

First patient enrolled in integrated program designed to improve knowledge of rare disease

NOVATO, CA –April 8, 2013 -Ultragenyx Pharmaceutical Inc. today announced the launch of a unique Disease Monitoring Program (DMP) for Hereditary Inclusion Body Myopathy (HIBM), also known as GNE Myopathy. The goal of the HIBM-DMP is to improve the body of knowledge about this rare disease and its typical course. This novel program is being conducted in partnership with the University of Newcastle's TREATNMD organization, a global neuromuscular physician network in Newcastle, England.The main objectives of the HIBM-DMP are to expand knowledge of the clinical presentation, progression and variation of HIBM patients; identify and qualify biomarkers and other efficacy measures; inform the design and interpretation of clinical studies of investigational products for HIBM and eventually to optimize patient management.