BioMarin Pharmaceutical Inc. on Target for Its 15% Revenue Growth Goal

It was a solid third quarter for BioMarin Pharmaceutical (BMRN -1.60%) as the biotech keeps plugging along, increasing sales of its orphan drugs while making progress on its pipeline drug candidates.

BioMarin results: The raw numbers

Data source: BioMarin.

What happened with BioMarin this quarter?

• Sales growth came from Kuvan, which increased sales by 16% year over year, and Vimizim, which was up 12% year over year. The revenue line also got a $35 million boost from a one-time payment from Sarepta Therapeutics (SRPT -0.80%) related to the settlement over the companies' exon-skipping patent litigation.
• Sales of Naglazyme fell 7% year over year, but that's thanks to lumpy sales. The number of commercial patients increased 7% year over year, and sales are up 8% looking at the first nine months of the year; both numbers are better indicators of demand.
• Recently approved Brineura had sales of $3.1 million, which is nothing to get excited about, but management warned that the launch would be slow. The company is making progress with reimbursement with sales in five countries already.

Image source: Getty Images.

What management had to say 

Chairman and CEO Jean-Jacques Bienaime laid out short- and long-term plans for revenue growth, including valrox, its gene therapy for hemophilia, and vosoritide, which treats achondroplasia, the most common form of disproportionate short stature:

In the nearer term, we anticipate that our current commercial products, plus pegvaliase, will drive our top line revenues to increase at a 15% compounded rate through 2020, with operating expenses increasing at a lower level. After that, valrox and vosoritide, which are both billion-dollar opportunities, can drive accelerated growth.

Sales in Brazil have caused BioMarin's revenue to be lumpy because the country buys drugs in large quantites that last multiple quarters, and the timing of the purchases can be unpredictable -- an order expected in the third quarter will actually occur in the fourth quarter, for instance -- but Jeffrey Ajer, BioMarin's chief commercial officer, said it should become less of an issue in the future as BioMarin continues to grow:

Over the last 5 years, sales concentration in Brazil relative to our global sales has declined by 50%, from 15% of sales to 7% of sales, and we expect this trend to continue as we expand the global reach of our commercial portfolio. Consequently, while Brazil will always be an important market, we expect revenue from Brazil will continue to be a smaller percentage of our total revenues going forward.

Looking forward

Management reaffirmed its revenue guidance to fall between $1.29 billion and $1.32 billion for the full year, which is a 15.5% to 18.2% increase over 2016 revenues, in line with its goal of revenue increases of 15%.

Looking to next year, the FDA should rule on the marketing application for pegvaliase in the first half of 2018, and BioMarin plans to file an application with EU regulators in the first quarter of 2018.

Further down the road, management gave some information on its pipeline, including for BMN 270 -- which now goes by valrox, short for valoctocogene roxaparvovec -- its gene therapy for hemophilia. Valrox is about to enter two independent phase 3 trials at two different doses. BioMarin expects to complete enrollment by the end of 2018 and have data by the end of 2019. Well before then -- as in, next month -- investors will get to see updated data from the phase 2 trial for valrox at the American Society of Hematology meeting, which will hopefully induce confidence that the phase 3 trials will be successful.

And even further back in the pipeline, BioMarin announced a new drug candidate, BMN 290, which should enter clinical trials next year to treat Friedreich's Ataxia, a neurologic and cardiac disorder.