Ultragenyx Investigational New Drug Application for UX007 is Active

NOVATO, Calif., Aug. 26, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. announced today that its Investigational New Drug (IND) application filed for UX007 is now active. UX007 is a substrate replacement therapy intended for use in treatment of long-chain fatty acid oxidation disorders (LC-FAOD). It is designed to replace deficient substrates in the fatty acid oxidation pathway and in the tricarboxylic acid (TCA) cycle, the key energy-generating processes in LC-FAOD patients. The active IND allows the company to proceed with its clinical development program for UX007 in LC-FAOD in the US.

Emil D. Kakkis, M.D., Ph.D., Ultragenyx's Chief Executive Officer commented, "The IND filing represents an important milestone in the UX007 clinical development program. We continue to make rapid progress in this program and are committed to developing UX007 as a treatment for patients with LC-FAOD. We look forward to starting our first clinical study in patients severely affected by LC-FAOD."

The company plans to initiate an open label Phase 2 clinical trial later this year for LC-FAOD in patients 6 years old and up, who, despite current therapy, continue to have one or more serious complications including episodes of muscle rupture, severe periods of low blood glucose, liver disease, and heart failure. The study will evaluate the safety and efficacy of UX007 in approximately 20 LC-FAOD patients. It consists of a 4-week observational period for baseline condition on current standard therapy followed by 6 months of treatment with UX007, and a long term extension. As requested by the FDA in a pre-IND meeting, the company is also conducting a 9-month juvenile, non-rodent chronic toxicology study which will enable treatment of patients under 6 years old.

About LC-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. It is estimated that several thousand patients are afflicted with FAOD in the US, where the fatty acid oxidation disorders are now diagnosed by newborn screening. It is also estimated that several thousand patients outside of the US are affected, where newborn screening for these disorders is becoming more common. LC-FAOD patients are currently treated by the avoidance of fasting, low-fat/high carbohydrate diets, carnitine supplementation and medium chain triglyceride (MCT) oil. Despite current therapy, many patients still have significant metabolic events including hospitalizations due to LC-FAOD and significantly increased mortality.

UX007 (triheptanoin) is a purified form of a specially designed synthetic triglyceride compound and is produced using a GMP-compliant process required to obtain FDA drug approval. UX007 is intended to provide patients with medium-length, odd-chain fatty acids that are metabolized to replace intermediate substrates in fatty acid oxidation downstream of their genetic block in fatty acid metabolism. UX007 is also metabolized to a substrate that replaces deficient intermediates in the TCA cycle, a key energy-generating process. Together, the substrates produced by UX007 during metabolism are intended to improve energy production in FAOD patients.

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 product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no effective treatments.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's 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.

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