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BioMarin (BMRN) Down on Phase III Data on Pegvaliase

BioMarin Pharmaceutical Inc.’s BMRN shares dipped 3.4% after the company announced results from the phase III study, PRISM-2, on pegvaliase for the treatment of patients with phenylketonuria (PKU).

The double-blind, placebo-controlled, randomized drug discontinuation trial (RDT) evaluated pegvaliase in comparison to matching placebo. Data from the study demonstrated that the treatment with pegvaliase led to an approximate 62% improvement in blood Phe, compared to placebo, thereby meeting the primary endpoint. The study, however, failed to achieve the secondary endpoint. Patients treated with pegvaliase failed to show any inattention or mood score benefit over the placebo arm over an 8-week period.

After a discussion with the FDA, BioMarin intends to submit a marketing application for pegvaliase by 2016 end.

According to information provided by the company, approximately 50,000 individuals in the developed countries are diagnosed with PKU.

We note that BioMarin currently has an approved drug, Kuvan, for this indication in its kitty. In 2015, the company generated Kuvan sales of $239.3 million, up 17.9%. Kuvan sales are expected in the range of $320–$350 million for 2016.

Our Take

Although this is not the first time that BioMarin has faced a pipeline setback, unfavorable data from the phase III study were highly disappointing. We remind investors that in Jan 2016, the company received a Complete Response Letter from the FDA for its New Drug Application for Kyndrisa for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping.

Going forward, we expect investor focus on further updates on the company’s pipeline.

BioMarin currently has a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector are AMAG Pharmaceuticals, Inc. AMAG, Emergent BioSolutions, Inc. EBS and Anika Therapeutics Inc. ANIK,  each sporting a Zacks Rank #1 (Strong Buy).

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