As expected, BioMarin Pharmaceuticals' (NASDAQ:BMRN) first quarter earnings release was focused on two hot issues: sales of Vimizim and the approvability of its Duchenne muscular dystrophy drug, drisapersen.
Both discussions were quite positive.
Sales of Vimizim, which treats a genetic disease called Mucopolysaccharidosis type IVA, topped $50 million in the first quarter, substantially higher than the $37 million of Vimizim sales seen in the fourth quarter; year-over-year comparisons aren't very helpful since the drug didn't really launch until the second quarter of last year.
The better-than-expected result led management to increase 2015 guidance for Vimizim sales to between $200 million and $220 million, topping previous guidance for Vimizim sales between $170 million and $200 million.
Management also increased total 2015 revenue guidance, but only by $10 million on each end, which goes to show how important the added revenue from Vimizim really is. The disconnect -- Vimizim guidance was up $20 million to $30 million on either end -- has to do with headwinds associated with the stronger dollar.
While not on the hot-topic list, it's worth pointing out that sales of Kuvan, which treats another genetic disorder called phenylketonuria, increased 11% year over year. While it's nice to see double-digit growth, the drug is working off such a low base, that it's hard to get too excited about the increase. In a year on the market, Vimizim has achieved equivalent sales to Kuvan, which gained FDA approval over seven years ago.
On the drisapersen front, no news was good news for investors. BioMarin's meeting ahead of completing its marketing application earlier this week didn't bring any surprises; the company doesn't need to run any new clinical studies, which would obviously delay approval and BioMarin's plan for becoming profitable. Management expects the FDA will call an advisory committee meeting in the second half of the year to get the opinion of outside experts, but so did most investors, so that's not really news.
Until we see the documents the FDA sends to the advisory committee a couple of days ahead of the meeting, we're unlikely to get any additional insight into the approvability of drisapersen. The FDA's decision will likely come down to the agency's stringent desire to see a phase 3 trial that succeeded -- which BioMarin doesn't have -- weighed on the other hand with the unmet need and hints that the drug works, especially in younger patients.
Bonus future hot issue
BMN 270 is years away from generating revenue, since it hasn't been tested in humans yet, but it's worth highlighting because of the type of therapy and the potential curative nature of BMN 270, a gene therapy to treat hemophilia A. BioMarin is looking to start the first clinical trial in the next few months, which could result in data by the end of the year. Since the drug could be a cure -- and it's not like hemophilia just goes away on its own -- if the gene therapy works in the first few patients, it'll be a good sign for BMN 270 even if it takes a few more clinical trials to confirm the initial findings.