BioMarin Surging on Impressive Phase II Dwarfism Data

BioMarin Surging on Impressive Phase II Dwarfism Data
June 18, 2015
By Mark Terry, BioSpace.com Breaking News Staff

San Rafael, Calif.-based BioMarin Pharmaceutical Inc. announced yesterday that BMN-111 (vosoritide) for the treatment of achondroplasia, the most common form of human dwarfism, had positive results.

The drug has received Orphan designation in the U.S. and Europe. In the Phase II trial, 26 children participated. Ten children in Cohort 3 were treated with 15 micrograms per kilogram daily and showed a mean increase of 50 percent in their annualized growth velocity. No serious side effects were observed.

“We are very encouraged to have observed evidence of activity with vosoritide in children participating in our Phase II study,” said Wolfgang Dummer, vice president of clinical development at BioMarin in a statement. “In children receiving the highest dose of 15 micrograms per kilogram daily, we observed a 50 percent increase in mean annualized growth velocity compared to their own natural history control growth velocity. This increase in growth velocity, if maintained, could allow children with achondroplasia to resume a normalized growth rate.”

In a note to clients, analyst Raluca Pancratov of SunTrust. Robinson Humphrey indicated that if the drug were approved, it could pull in about $700 million worldwide. Achondroplasia affects about 1 in 15,000 to 40,000 newborns.

BioMarin stock took a jump on the news. Shares were trading for $123.72 on Wednesday and is currently selling for $136.50. The company stock has been on a steady and significant rise for the last year. Shares traded for $55.78 on July 17, 2014, rose to $67.27 on Oct. 14, 2014, and hit $129.14 on Mar.30, 2015.

RBC Capital maintained its rating for BioMarin of “Outperform” and increased its target price to $145.00 from $125.00. Analyst Michael Yee, of RBC Capital, wrote in a note, “BMN-111 (vosoritide) could become BMRN’s biggest drug with peak $1-2 billion sales. Based on prevalence of about 57,000 (patients) across the U.S. and the European Union, and assuming 25 percent are older than 15 years of age and eligible for the drug, if we assume $300,000 pricing this yields as high as $2 billion in sales based on varying penetration.”

Yee also predicted the stock would continue higher this year on the basis of the company’s continuation into Phase III trials and possible FDA approvals later in the year.

Jeffreys Group gave the company stock a target price of $156.00, up from $135.00 and gave it a “Buy” rating. Barclays raised price target from $150 to $156 and gave it an “overweight” rating. Analyst at JPMorgan Chase & Co also gave it an “overweight” rating and set a target price of $151.00, up from $140.00.

In its April 30, 2015 quarterly earnings report BioMarin announced first quarter total revenue of $203.3 million, an increase of 34 percent from the same period last year. “We begin 2015 prepared to execute on a number of transformational milestones,” said Jean-Jacques Bienaimé, chief executive officer of BioMarin in a statement. “Having completed the submission of the NDA for approval of drisapersen in the U.S., we will turn our attention to filing for approval in Europe this summer.”

In addition to the BMN-11 results, the company expects Phase I/II study results on cerliponase alfa for CLN2 disorder, a late infantile form of Batten Disease later in the year. It is also focusing on legacy products such as Kuvan, Neglazyme and the most recent launch of Vimizim, a treatment for a rare genetic disorder, Morquio A (MPS IVA).


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