BioMarin's (BMRN) Batten 'Surprisingly Looks Good', Say RBC Capital

RBC Capital maintained an Outperform rating on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) with a price target of $110. Earlier BioMarin reported interim results from its Phase 1/2 pivotal study for BMN 190 to treat of patients with late infantile CLN2 disease, a form of Batten disease. Analyst Michael Yee thinks there will be an FDA filing on the drug.

"Bottom line is we fully believe that BMRN will file this to the FDA at the end of the study -- and it has a reasonably good chance of approval perhaps in 2016+ -- our discussions with Batten's experts and market analysis is that this is for a "subsegment" of Batten's and would be a $200M+ drug or so with premium Orphan pricing....and we think the stock could get to $100 or higher on this," said Yee.

"This positive surprise showing that patients are "stable" when they should be rapidly declining (compared to natural history) means that patients may be experiencing a drug effect since the company says it's well validated and proven that they should be declining. The analysis was done using a control match with their natural history counterpart," continued the analyst.

"What's also interesting to us is that this could be at the "low" dose....as these are the first round of patients and the lowest dose who've been on the longest.....so we think a stabilization could support approval and/or the medium/high doses are as good or better," he added.

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 $93.78 yesterday.

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