Ultragenyx Receives Approval of Clinical Trial Application (CTA) for a Phase 1/2 Trial Testing UX003 in Mucopolysaccharidosis Type 7 (MPS 7)

NOVATO, Calif., Aug. 14, 2013 (GLOBE NEWSWIRE) -- 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.

"We are pleased with the rapid progress of the UX003 clinical development program which may finally provide a long-awaited therapy to treat MPS 7 patients," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "Other MPS enzyme therapies have been successfully developed already, and we are happy to be finally applying the same treatment strategy for affected MPS 7 patients."

The open label Phase 1/2 clinical study is designed to primarily evaluate safety, dose, and dose regimen in MPS 7 patients. The company expects to start enrolling patients later this year at a single center in the U.K. If the Phase 1/2 trial is successful, the company anticipates it would proceed with a pivotal Phase 3 trial in 2014.

About MPS 7

Mucopolysaccharidosis type 7 (MPS 7), originally described in 1973 by William Sly, MD (also known as Sly syndrome), is a rare genetic, metabolic disorder and is one of 40 different lysosomal storage disorders. MPS 7 is caused by the deficiency of β-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulfate and heparan sulfate. These complex GAG carbohydrates are a critical component of many tissues. The inability to properly break down GAGs leads to a progressive accumulation in many tissues and multi-system disease.

While its clinical manifestations are similar to MPS 1 and MPS 2, MPS 7 is one of the rarest among the MPS disorders and is likely underdiagnosed as are most rare diseases. MPS 7 has a wide spectrum of clinical manifestations and can present as early as at birth or in older patients with less rapidly progressive disease. There are no approved therapies for MPS 7 today.

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. For Media, Bee Nguyen For Investors, Shalini Sharp 415-483-8800Source:Ultragenyx Pharmaceutical Inc.