BioMarin Pharmaceutical Inc. Got It Done in the Second Quarter

BioMarin Pharmaceutical (BMRN -9.90%) turned in a second-quarter report on Wednesday that showed it continuing to grow sales of its current drugs, getting new drugs approved, and producing promising clinical trial data on its pipeline candidates. There isn't much else investors in biotechs can ask for.

BioMarin Pharmaceutical results: The raw numbers

Data source: BioMarin Pharmaceutical.

What happened this quarter?

• Sales of its top-selling drug Vimizim came in at $103.2 million, which was down slightly year over year, but that was only due to buying patterns. The number of commercial patients grew 24% year over year.
• Sales of Kuvan were hot on Vimizim's heels at $102 million, thanks to 13% year-over-year growth.
• Sales of Naglazyme, which is in its 12th year on the market, continue to tread forward. Commercial patient counts rose 7% year over year.
• BioMarin received approval for its newest orphan drug Brineura in both the U.S. and EU.
• The company submitted a marketing application to the FDA for pegvaliase in the U.S. The EU submission is expected by the end of the year.
• In July, BioMarin presented promising data for BMN 270, a gene therapy treatment for hemophilia A. As part of the earnings report, the company reported further data on the mid-range dose, which doesn't have as much protein expression to compensate for the missing protein in patients with hemophilia, but appears to have less variability of expression. BioMarin's plan is to run two separate phase 3 studies, one at each dosage.
• Also in July, BioMarin received a $35 million one-time payment from Sarepta Therapeutics (SRPT 2.53%) to settle a lawsuit over patents on exon skipping.

Image source: Getty Images.

What management had to say 

Henry Fuchs, BioMarin's president of worldwide research and development, explained why the company decided to run two separate phase 3 trials for BMN 270 rather than running one trial with patients split between the high 6e13 dose and the mid-range 4e13 dose:

You have to remember that there are a very active strong group of clinicians and patient advocates that are very keen to see the 6e13 group get enrolled and finished and through registration. ... If that information turns out to be what guides enrollment, then there's a very real possibility that 6e13 will finish much faster than 4e13. So we didn't want to slow down the application if 4e13 was going to end up going slower. That's why we chose to do two studies.

Chief Commercial Officer Jeffrey Ajer touched on how the launch of recently approved Brineura, which treats CLN2 disease, is going:

Due to the complexity of coordinating institutions' preparedness for port surgery and biweekly intraventricular infusions, one of our key launch metrics is the number of institutions ready to treat and service CLN2 patients. Recall, our global target for the first year during commercial launch is 50 target institutions, so we are well on our way. I'm pleased to share that as of today, 26 target institutions are treatment-ready. This means they are interested and able to serve CLN2 patients properly resourced and in-service.

Looking forward

Biomarin updated its 2017 guidance to take the aforementioned payment from Sarepta into account, but otherwise left product sales revenue guidance intact. Management still thinks it'll be able to produce a non-GAAP income of $30 million to $70 million for the year, although the company will still lose money on a GAAP basis this year.

Looking further ahead, investors should be focused on how quickly BioMarin can ramp up sales for Brineura. Pegvaliase, assuming it's approved, should be easier to market since it treats the same disease as Kuvan.

BMN 270 offers the biggest upside potential, but BioMarin is going to have to act fast; this week, competitor Spark Therapeutics released promising phase 1/2 data for its own gene therapy to treat hemophilia A.