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NOVATO, Calif., March 8, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the Phase 1 study of UX001 for hereditary inclusion body myopathy (HIBM) has been completed.  UX001 is an extended release formulation of sialic acid intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency.  UX001 is the first program from the company's pipeline to enter the clinic since its founding in 2010. The FDA Office of Orphan Products Development has granted orphan drug designation for UX001 for the treatment of HIBM.

"Ultragenyx continues to make excellent progress in advancing and expanding our clinical development pipeline with the completion of our first clinical study," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "We deeply appreciate the participation and enthusiastic support of the HIBM patient community for our Phase 1 study. We anticipate releasing the results from this study within the next couple of months, and will look forward to presenting the data at a scientific meeting in the fall. We plan to initiate a Phase 2 trial of SA-ER in patients with HIBM in the second quarter of this year."

The Phase 1 clinical study evaluated the pharmacokinetics (PK) and safety of UX001 in 28 HIBM patients.  The study tested five different single-dose levels in each group of six subjects.   Subjects then underwent repeat dosing at four dose levels over 7 days to establish the steady-state pharmacokinetics and safety of repeat doses of UX001.  Ultragenyx anticipates data from the Phase 1 study in April 2012.   

About HIBM 

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease.  HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market.  The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases.  Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy.

The company is led by an experienced management team in rare disease therapeutics.  Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.