Ultragenyx Announces Data Presentations of rhGUS and rhPPCA at 12th Annual WORLD Symposium™

NOVATO, Calif., March 01, 2016 -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced upcoming presentations of data highlighting recombinant human beta-glucuronidase (rhGUS, UX003) and recombinant human protective protein cathepsin-A (rhPPCA, UX004) at the Lysosomal Disease Network’s (LDN) 12^th Annual WORLDSymposium^TM March 1-4 in San Diego, California. At the symposium, Ultragenyx CEO Emil Kakkis, M.D. Ph.D., will receive the 2016 LDN Award for Innovation and Achievement.

The following three posters will be presented on Tuesday, March 1 from 4:30-6:30pm PT and Wednesday, March 2 from 4:00-6:00pm PT:

rhGUS (UX003) in Mucopolysaccharidosis 7 (MPS 7)

Poster #173: Enzyme Replacement Therapy with Investigational rhGUS (UX003) in an Infant with Non-immune Hydrops Fetalis (NIHF) and Mucopolysaccharidosis Type VII (MPS VII)

• Authors: H.A. Lau, S. Parmar, M. Kazachkov, R. Shah, J. Wells, N. Yachelevich, A. Chopra, C. Haller, E. Kakkis

Poster #174: Evidence of Early Bone Response after Initiation of ERT in a 3-Year Old Patient with MPS VII

• Authors: H.A. Lau, S. Parmar, M. Ferraris, C. Haller, S. Agarwal, E. Kakkis

rhPPCA (UX004) for Galactosialidosis

Poster #LB-13: Recombinant Human Protective Protein/Cathepsin A: An Update on the Development of an Enzyme Replacement Therapy for Galactosialidosis

• Authors: V. Koppaka, J. Cadaoas, S. Cullea, E. Gomero, C. Guzman, H. Hu, K. Jayashankar, M. Machado, G. Morris, R. Mosca, A. Natesan, A. d'Azzo, M. Vellard

About MPS 7

Mucopolysaccharidosis 7 (MPS 7, Sly syndrome), originally described in 1973 by William Sly, M.D., is a rare genetic, metabolic disorder and is one of 11 different MPS disorders. MPS 7 is caused by the deficiency of beta-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 results in a 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. MPS 7 has a wide spectrum of clinical manifestations and can present as early as at birth in a severe form called non-immune hydrops fetalis. There are no approved therapies for MPS 7 today. The use of enzyme replacement therapy as a potential treatment is based on 20 years of research work in murine models of the disease. Enzyme replacement as a strategy is well established in the MPS field as there are currently four approved enzyme replacement therapies for other MPS disorders: MPS 1 (Aldurazyme®, laronidase), MPS 2 (Elaprase®, idursulfase), MPS 4A (Vimizim™, elosulfase alfa), and MPS 6 (Naglazyme®, galsulfase).

About Galactosialidosis

Galactosialidosis is a rare lysosomal storage disease for which there are no currently approved drug therapies. Similar to MPS patients, patients with galactosialidosis present with both soft tissue storage in the liver, spleen, and other tissues, as well as connective tissue (bone and cartilage) related disease. As with MPS 7, an enzyme deficiency results in accumulation of substrates in the lysosomes, causing skeletal and organ dysfunction, and death.

About Ultragenyx

Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built 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 approved therapies.

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.

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Ryan Martins
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