Positive Data from BMRN's PKU-016

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BioMarin Pharmaceutical Inc. (BMRN) recently announced positive results from the PKU-016 ASCEND study. This is the largest randomized controlled study evaluating the safety and therapeutic effects of Kuvan (sapropterin dihydrochloride) on neuropsychiatric symptoms in subjects with phenylketonuria (:PKU).

We note that Kuvan is already approved for the treatment of patients suffering from PKU. BioMarin has a partnership with Merck Serono, a division of Merck KGaA (MKGAY), for the development and commercialization of Kuvan since 2005.

PKU-016 is a randomized, placebo-controlled, 13-week outcomes study in patients who were treated with Kuvan. The study evaluated medically important symptoms similar to attention deficit hyperactivity disorder (:ADHD) in patients suffering from PKU and whose blood levels of phenylalanine (Phe) were reduced by Kuvan.

The study results showed that Kuvan improved the attention deficit hyperactivity rating scale (ADHD-RS) driven by a statistically significant change in the inattention component of the score. BioMarin now plans to submit data from the study to the US Food and Drug Administration (:FDA) to include this in the Kuvan label.

BioMarin reported Kuvan net product revenues of $36.4 million in the third quarter of 2012, up 19.3% year over year. The company expects Kuvan net product revenue in the range of $130–$140 million for the full year 2012.

BioMarin also mentioned in its press release that the results from the study will help the company to design the phase III PEG-PAL (poly ethylene glycol-phenylalanine ammonia lyase) program. The candidate is being developed for the treatment of severe PKU. In Sep 2012, BioMarin reported positive preliminary results from an open-label, multi-center, phase II program of PEG-PAL.

We remind investors that BioMarin will report its fourth quarter and full year results on Feb 21. We expect investor focus to stay on the company’s 2012 results.

BioMarin currently carries a Zacks Rank #3 (Hold). However other bio pharma stocks such as Alkermes plc (ALKS) and Alexion Pharmaceuticals Inc. (ALXN) carry a Zacks Rank #2 (Buy).

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