Ultragenyx Pharmaceutical Inc. RARE announced positive data from an investigator-sponsored study on triheptanoin, which is being developed for the treatment of movement disorders associated with glucose transporter type-1 deficiency syndrome (Glut1 DS).
The data was presented at the annual meeting of the American Academy of Neurology. Based on this data, the company now intends to initiate a confirmatory randomized double-blind placebo-controlled study in the Glut1 DS movement disorder phenotype and will discuss details of the final study design with the FDA in the second half of 2015.
In the investigator-sponsored study, triheptanoin was evaluated in eight Glut1 DS patients aged between 7 and 47 years. Patients reported a statistically significant 90% reduction in paroxysmal manifestations including dystonic events during the triheptanoin treatment phase. Triheptanoin was found to be well tolerated.
Currently, there are no FDA-approved treatments specifically for Glut1 DS. According to the company press release, there are approximately 3,000 to 7,000 Glut1 DS patients in the U.S.
Ultragenyx is evaluating triheptanoin in a phase II study for the treatment of Glut1 DS patients who have failed the ketogenic diet and who continue to have breakthrough seizures. The study is being conducted in both the U.S. and the EU.
We note that the company is also evaluating triheptanoin in another phase II study for the treatment of long-chain fatty acid oxidation disorders with interim data expected in the second half of 2015.
Meanwhile, Ultragenyx has several other pipeline updates lined up. Phase II data on KRN23, being developed for the treatment of both X-linked hypophosphatemia and tumor-induced osteomalacia, should be out in late 2015 or early 2016. Another candidate, rhGUS, is currently in a phase III study for mucopolysaccharidosis 7 with data expected in 2016.
We expect investor focus to remain on further pipeline updates from the company.
Ultragenyx is a Zacks Rank #3 (Hold) stock. Some better-ranked stocks in the health care sector include CorMedix, Inc. CRMD, AVEO Pharmaceuticals, Inc. AVEO and Biogen BIIB. All three stocks sport a Zacks Rank #1 (Strong Buy).
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