Specialist Sees BioMarin's (BMRN) Pegvaliase As Approvable - PiperJaffray

Piper Jaffray analyst, Joshua Schimmer, hosted a call with a PKU specialist to gauge prospects for BioMarin (NASDAQ: BMRN) Pegvaliase after P3 results. He has a high degree of confidence in FDA approval and plans to offer the drug broadly to his patients. In his practice, ~50-60 patients out of a total 200 with PKU are on Kuvan, but only a small number have been able to fully normalize diet and discontinue protein supplements. He believes Pegvaliase has uniquely potent effects on Phe levels with the potential to eliminate diet restrictions/supplements and is not perturbed by the lack of statistically significant neurocog benefit in P3 given the short trial duration. Depth of market penetration will depend on patients' willingness to undergo daily injections and get through early side effects, but trial experience has been good, suggesting a healthy outlook for the drug.

The specialist was comfortable with Pegvaliase' profile, noting its high potency, well beyond that of Kuvan for a majority of patients (there is a minority of patients who fail to respond, perhaps due to neutralizing antibodies). The safety profile in his view is acceptable; while some have trouble with injection site reactions, this can often be dealt with by rotating injection sites. Early rash and joint discomfort often fades within a few weeks. Discontinuation rates in clinical trials has been low and patients haven't complained much about side effects.

No change to Overweight rating or $107 PT.

For an analyst ratings summary and ratings history on BioMarin Pharmaceutical Inc. click here. For more ratings news on BioMarin Pharmaceutical Inc. click here.

Shares of BioMarin Pharmaceutical Inc. closed at $80.87 yesterday.

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