Positive Data from BMRN's PKU-016 - Analyst Blog

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 (MKGAF), 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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