Ultragenyx Initiates New Development Program Studying Triheptanoin

Ultragenyx Initiates New Development Program Studying Triheptanoin (UX007) for the Treatment of Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

           Phase 2 Trial Initiation Planned for Year End

NOVATO, Calif., Aug. 5, 2013 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced its plan to develop triheptanoin (UX007) for the treatment of seizures in glucose transporter type-1 deficiency syndrome (Glut1 DS). Glut1 DS is a rare and severely debilitating disease caused by mutations in the
SLC2A1 gene, which encodes for a protein called glucose transporter
type-1 (Glut1). The disease is characterized by seizures, developmental delay and movement disorders. The Glut1 protein transports glucose from blood into the brain. Because glucose is the primary source of energy for the brain, this defect in the Glut1 transporter results in a chronic state of brain energy deficiency. The company is planning to initiate a Phase 2 trial studying triheptanoin in Glut1 DS by the end of 2013.

Triheptanoin, a triglyceride of medium, odd-carbon chain (C7) fatty acids, is intended as a substrate replacement therapy to restore energy metabolism in Glut1 DS patients by providing an alternative source of energy to the brain, in addition to providing anaplerotic substrates to resupply intermediates of the tricarboxylic acid cycle (TCA), a key energy-generating process. In recently published literature, triheptanoin was shown to deliver energy substrates to the brain in
Glut1 DS mice. In other published data, triheptanoin was shown to reduce seizures in mouse models of epilepsy.

Triheptanoin is being developed to address the needs of Glut1 DS patients who continue to have episodes of seizures despite the best available treatment. While there are currently no drugs approved for
Glut1 DS, the current standard of care consists of a ketogenic diet, which provides ketone bodies as an alternative energy source to glucose. However, seizures are not controlled completely in all patients, and many patients continue to have significant problems with developmental delay and motor dysfunction. In addition, due to the extreme dietary restrictions, some patients are not able to comply with the ketogenic diet.

"This UX007 development program is promising news for patients with
Glut1 DS as it may provide an alternative or complementary treatment option to the ketogenic diet," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We look forward to working with the investigators, patients and disease community in studying triheptanoin for Glut1 DS. Adding this new program to our existing pipeline is another example of our commitment to developing therapies that address significant unmet medical needs."

The company is working with the FDA to gain alignment on the path forward for initiating a global Phase 2 study. The proposed study is designed to assess the safety and efficacy of triheptanoin in Glut1 DS patients in a rigorous, controlled clinical trial, and evaluate the effects of triheptanoin on seizures as well as developmental delay and motor dysfunction.

About Glut1 DS and Triheptanoin

Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS) is a rare and severely debilitating disease characterized by seizures, developmental delay and a movement disorder. Glut1 DS is caused by a mutation in SLC2A1, encoding the Glut1 protein responsible for transporting glucose across the blood-brain barrier. Because glucose is the primary source of energy for the brain, this disorder results in a chronic state of energy deficiency in the brain. Glut1 DS is a rare disease with an estimated birth incidence of 1:90,000. The prevalence is estimated to be several thousand patients in the US. There are currently no approved treatments specific to Glut1 DS.

Triheptanoin 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 the tricarboxylic acid (TCA) cycle, a key energy-generating process. Ultragenyx is testing a highly purified form of triheptanoin, which is produced using a GMP-compliant process required to obtain drug approval from the FDA and other regulatory agencies.

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 inquiries, Bee Nguyen
         bnguyen@ultragenyx.com
         For Investor inquiries, Shalini Sharp
         ssharp@ultragenyx.com
 

Posted: August 2013

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