BioMarin outlines its R&D game plan as Duchenne MD decision nears

BioMarin CEO Jean-Jacques Bienaimé

BioMarin's ($BMRN) executive team zeroed in on its late-stage R&D efforts for rare diseases on Monday after outlining a surge in Q2 revenue and a beat on analysts' expected losses for the quarter.

Front and center in the pipeline discussion that followed news of the company's 23-cents-per-share loss for the quarter is its run at an approval for the exon-skipping Duchenne muscular dystrophy drug drisapersen, picked up in its $840 million buyout of Prosensa. CEO Jean-Jacques Bienaimé noted that drisapersen will have to run the gamut of an advisory committee meeting before a formal decision comes from the FDA. But BioMarin is planning for a product launch in early 2016, which called for a predecision market review that notes that a large segment of the patient population will need to be tested to see exactly how many of them could benefit from the drug.

As it stands now, BioMarin expects that about 10,000 of the 75,000 DMD boys could benefit from the drug.

At the same time, the biotech has been angling to advance its closely-watched drug vosoritide (BMN 111), which is designed to correct dwarfism. BioMarin started dosing in the 30 microgram dose arm, the fourth cohort in their promising Phase II study. As R&D chief Hank Fuchs noted in the call, the 30 microgram arm is designed to test the drug's ability to spur "catch-up" growth among dwarves. And analysts have been closely following the drug's prospects after some promising results for correcting the genetic mutation behind dwarfism were reported earlier in the summer.

"We are preparing for discussions with health authorities on design and planning for our registration and ending studies, which we hope to begin in first half of 2016," said the CEO in a call with analysts.

BioMarin reported a few weeks ago that the 10 children with achondroplasia taking a 15 micrograms per kilogram per day dose of BMN-111 (or vosoritide) demonstrated a mean increase of 50% in what investigators termed their "annualized growth velocity compared to their annualized prior 6 month natural history baseline growth velocity."

Behind vosoritide BioMarin has been advancing an early-stage study for its gene therapy for hemophilia, a growing field in biotech as a number of competitors line up rival programs.

"We expect to enroll our first hemophilia A patient imminently," Fuchs noted. "We are very enthusiastic about this program, because of its potential to provide long-term therapeutic benefits to patients who currently require daily prophylactic treatment for their hemophilia.

The biotech also has a mid-stage program underway for other DMD candidates and a possible pivotal study for CLN2 disease--including Battens disease--which the company believes afflicts over 1,000 patients worldwide. BioMarin's focus on rare diseases has made it a frequently cited takeover target at a time that M&A deals have become red-hot in the industry.

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