Hyperion Therapeutics, Inc. (HPTX) recently presented multiple pipeline related data at the International Congress of Inborn Errors of Metabolism (:ICIEM) and the satellite symposium on urea cycle disorders (UCDs) in Spain.
The data was related to the company’s marketed product Ravicti. We remind investors that Hyperion acquired worldwide rights to Ravicti from Ucyclyd Pharma Inc., a subsidiary of Valeant Pharmaceuticals International, Inc. (VRX), last year for an upfront payment of $6 million. The future payments will be based on the achievement of regulatory milestones in indications other than UCD, sales milestones, and mid-to-high single-digit royalties on global net sales of Ravicti.
Ravicti received the U.S. Food and Drug Administration (:FDA) approval in Feb 2013 for use as a nitrogen-binding agent for chronic management in adult and pediatric UCD patients of more than two years of age, who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. In May 2013, Ravicti received orphan drug status in the U.S.
Hyperion emphasized on long-term data on ammonia control in Ravicti treated pediatrics suffering from UCD.
The long-term data was based on two studies on Ravicti comprising 49 UCD minor patients. The patients enrolled included 23 patients between the age of 2 to 5 years. Patients were treated for 12 months with Ravicti and the observed ammonia levels remained below the normal upper limit of 35 umol/L. Average ammonia levels ranged between 17.2 to 24.8 umol/L. Patients’ physical growth and development were also found to be normal.
Hyperion also presented a pooled analysis of data from two short-term studies which showed that Ravicti was non-inferior to its other marketed product Buphenyl. Buphenyl is approved by the FDA to treat UCD.
Hyperion currently carries a Zacks Rank #3 (Hold). Companies that look well-positioned include Actelion Ltd. (ALIOF) and Jazz Pharmaceuticals Public Limited Company (JAZZ) with a Zacks Rank #1 (Strong Buy) and a Zacks Rank #2 (Buy), respectively.
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