Ultragenyx Pharmaceutical Inc. (RARE) will be presenting encouraging data on its mucopolysaccharidosis 7 (MPS 7) candidate, rhGUS (recombinant human beta-glucuronidase, UX003), at the 10th Annual World Lysosomal Disease Network Symposium. Investors reacted positively to the news with the stock soaring 11.6% on the news.
Data from a case study of an advanced multi-system MPS 7 patient (12 years old) with respiratory insufficiency, who is being treated with rhGUS will be presented. The preliminary data suggests that rhGUS helps in reducing lysosomal storage (based on reduced excretion of urinary glycosaminoglycans), as well as the size of the enlarged liver and spleen. Plus, an improvement in pulmonary function was noted. Additionally, no infusion-related side effects were found during the first 14 weeks of treatment.
We remind investors that in Dec 2013, Ultragenyx had initiated an open-label phase I/II study to evaluate the safety and efficacy of rhGUS (administered every alternate week through intravenous infusion) in five MPS 7 patients aged between 5 and 30 years. Interim data from the phase I/II study is expected this year. The initial primary analysis phase of 12 weeks will be followed by dose-exploration and long-term extension.
The U.S. Food and Drug Administration (FDA) has granted an emergency Investigational New Drug (eIND) for rhGUS. MPS 7 represents an underserved market with no approved therapies being available.
Ultragenyx is a biopharmaceutical company. Other biopharma stocks that are worth considering include Alexion Pharmaceuticals, Inc. (ALXN), Emergent BioSolutions, Inc. (EBS) and Actelion Ltd. (ALIOF). All three sport a Zacks Rank #1 (Strong Buy).
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