BioMarin Pharmaceutical (BMRN) Price Target Raised to $145 at RBC

RBC Capital maintained an Outperform rating on BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and raised its price target to $145.00 (from $125.00). Yesterday BioMarin reported positive results of a Phase 2 proof-of-concept and dose finding study of BMN 111 (vosoritide) in children with achondroplasia, the most common form of human dwarfism. Analyst Michael J. Yee said data looks good.

"... stat sig 50% improvement in growth velocity, dose response, no safety issues, injections well tolerated, no symptomatic hypotension; all pts rolling into high dose 15ug/kg cohort extension study – so we’re looking forward to even more data early next year and update on FDA discussions for pivotal study. New 4th cohort to test for even more efficacy as they prepare for Phase III. Questions: Is FDA ok with the therapeutic window? Is it a durable effect over time and will pts grow in proportionality? How confident is the IP post 2022 composition of matter and will they easily be able to license 3rd party methods of use patents that they may need?," said Yee.

"BMN-111 (vosoritide) could become BMRN's biggest drug with peak $1-2B sales. Based on prevalence of ~57k across US and EU, and assuming 25% are <15yrs and eligible for drug, if we assume $300k pricing this yields as high as $2B sales based on varying penetration. On 100% prob. of success, this could be theoretically worth $38/share but currently we model only 65% POS and $25/share," continued the analyst.

Yee added, "From a trading perspective, we expect stock to continue to move higher this year on growing Street confidence in dwarfism program that will advance to Phase III and isn’t fully included in consensus estimates yet - and most importantly – Street anticipation of potential FDA approval in DMD by year-end 2015 (the big event of the year)."

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 $123.60 yesterday.

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