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BioMarin Pharmaceuticals (NASDAQ:BMRN) reported second-quarter earnings last week. While the biotech's current drugs are still growing at a nice pace, it's BioMarin's pipeline, including two drugs that have already read out positive phase 3 data, that has the potential to accelerate growth in the coming years.

BioMarin Pharmaceutical results: The raw numbers


Q2 2016 Actuals

Q2 2015 Actuals

Growth (YOY)


$300 million

$250 million


(Loss) from Operations

($573 million)

($64 million)


(Loss) per share




Data source: Company press release. YOY = year over year.

What happened with BioMarin Pharmaceutical this quarter?

  • The large loss came from a charge for the impairment of intangible assets associated with the discontinuance of its Duchenne muscular dystrophy drug Kyndrisa. On an adjusted basis, net income was $16.1 million for the quarter.
  • Sales of Vimizim nearly doubled year over year, but that was partially due to the timing of large orders from Brazil and the Middle East. The company estimates that $15 million of the $107 million of Vimizim sales in the second quarter were for future demand.
  • Naglazyme experienced the opposite effect, with sales down 29% year over year because of a large order from Brazil in the year-ago quarter.
  • Kuvan sales increased 50%, but much of that was from the reacquisition of the international rights to Kuvan from Merck Serono. Nevertheless, North American sales were up a healthy 13% year over year.
  • BioMarin filed U.S. and EU marketing applications for Brineura, its drug for CLN2, late-infantile form of Batten disease.
  • Last month, the company reported positive proof-of-concept data for its gene therapy program with BMN 270 for hemophilia A and plans to move into a phase 2b study.

What management had to say

With limited but very promising data for BMN270, Hank Fuchs, Biomarin's chief medical officer, noted that the company might see if a higher dose would work better while not causing more side effects, "We're considering whether a second dose level would be a wise thing to incorporate into that phase 2B trial."

While the large buying patterns of some countries cause lumpy sales, BioMarin has factored that into guidance. "Due to the unevenness of order timing, we recommend that you follow full-year guidance as the best measure of anticipated performance for Vimizim," said Jeff Ajer, Biomarin's chief commercial officer.

Looking forward

Management increased 2016 guidance for sales of Vimizim and Kuvan and also bumped overall revenue guidance to a range of $1.1 billion to $1.15 billion, an increase over the previous guidance, which topped out at $1.1 billion.

In addition to designing the aforementioned phase 2b study for BMN270, BioMarin also plans to start a phase 3 trial for vosoritide that treats achondroplasia, the most common form of dwarfism, by the end of the year.

After getting positive phase 3 data for pegvaliase for PKU -- the same disease Kuvan treats -- BioMarin is working on its FDA marketing application. But it's a large data package, in part because pegvaliase is a nonhuman biologic, so it will take until the fourth quarter, or possibly the first quarter of next year to file the application.

Finally, looking to next year, BioMarin will start the year off with a decision on the marketing application for Brineura on or before January 27, if the FDA sticks to its goal. European regulators haven't set a goal yet, but their decision shouldn't come too long after that.

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