Ultragenyx Gains Worldwide Rights for Triheptanoin (UX007)

NOVATO, Calif., July 11, 2013 (GLOBE NEWSWIRE) -- 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.

The company plans to initiate a Phase 2 clinical trial with UX007 later this year in patients with long-chain FAOD. The company is also completing a retrospective study of triheptanoin in 24 long-chain FAOD patients who have been receiving triheptanoin through an investigator-sponsored compassionate-use protocol for up to 13 years. Data from the retrospective study will be presented this fall.

"We are encouraged by the data we have seen to date from investigator-initiated studies, and are excited to expand the potential utility of UX007 in a global development program," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "Our next step is to build on these investigator programs with clinical data from sponsored trials of UX007 in patients with these devastating metabolic disorders."

In addition to advancing its own development program toward potential approval, Ultragenyx is supporting multiple independent investigator-sponsored and compassionate-use clinical trials in FAOD and other indications and is providing drug free of charge for these studies. UX007 is a pharmaceutical-grade product being developed by Ultragenyx under an exclusive supply agreement for triheptanoin with an experienced European manufacturer.

Ultragenyx is planning to develop other potential indications for UX007, including GLUT1 deficiency syndrome (also known as De Vivo disease), a genetic disorder that impairs brain energy metabolism.

About FAOD and UX007

Fatty acid oxidation disorders are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to break down fatty acids into energy. Several thousand patients are afflicted with FAOD in the U.S. and a number of fatty acid oxidation disorders are now included in newborn screening panels. Several thousand additional patients outside of the U.S. are also affected, and newborn screening for these disorders is becoming more common.

UX007 is a specially designed synthetic compound intended to provide patients with a medium-length, odd-chain fatty acid that is metabolized to replace intermediate substrates in fatty acid oxidation and in the tricarboxylic acid (TCA) cycle, a key energy-generating process. UX007, a highly purified form of triheptanoin, is produced using a GMP-compliant process intended to reduce taste and odor with the goal of enhancing patient compliance.

About Ultragenyx

Ultragenyx is a privately held, clinical-stage biotechnology company committed to bringing to market life-transforming therapeutics for patients with rare and ultra-rare metabolic genetic diseases. Founded in 2010, the company is rapidly building a diverse portfolio of products addressing diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no effective treatments. Ultragenyx has one product in Phase 2 clinical trials, UX001 for the treatment of hereditary inclusion body myopathy (HIBM). UX007 is planned to enter Phase 2 clinical trials for long-chain FAOD later this year. Another compound, UX003 for MPS 7 (Sly Syndrome), is expected to begin Phase 1/2 clinical trials later this year. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx' strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

About BRI

Established in 1984 in Dallas, Texas, Baylor Research Institute (BRI) promotes and supports research to bring innovative treatments from the laboratory workbench to the patient bedside. To achieve this bench-to-bedside concept, BRI focuses on basic science, clinical trials, health care effectiveness and quality of care research. Today, under the leadership of Michael Ramsay, MD, BRI is conducting more than 900 active research protocols with 350 research investigators, spanning more than 20 medical specialties, and has research and development projects in areas ranging from human immunology and orphan metabolic diseases to diabetes, cardiovascular disease and many other unmet medical needs. Its precision medicine arm offers a unique platform for identifying micro-array-based fingerprint signatures. The Baylor Health Care System offers to its research affiliate unique access to one of the largest patient bases potentially available for research in the US within a single institution. BRI has received full accreditation from AAHRPP. www.baylorhealth.edu/research/

CONTACT: Ultragenyx Pharmaceutical Inc. 415-483-8800 For Media inquiries, Bee Nguyen bnguyen@ultragenyx.com For Investor inquiries, Shalini Sharp ssharp@ultragenyx.comSource:Ultragenyx Pharmaceutical Inc.