BioMarin shares are up almost 30% since Monday's announcement that the phase III trial of GALNS for the treatment of MPS IV or Morquio syndrome met its primary endpoint of change in six-minute walk compared to placebo when dose weekly. Key secondary efficacy endpoints in the study were also met.
These GALNS results are definitely a huge positive and should help BioMarin get the drug approved easily. An FDA filing is expected in the first quarter of 2013, which means approval could come by the end of next year.
In late September, I said the GALNS study was the most important event in BioMarin's history because the MPS IV patient population is bigger than both the MPS I and MPS II populations treated by drugs the company already sells. GALNS has the potential to double BioMarin's revenue. I also predicted the study's success.Keep in mind that Biomarin still has a robust pipeline beyond GALNS. Peg-Pal for PKU will begin phase III trials in the first half of next year. BMN-701 will report phase 1b data early next year. A phase 2 trial for BMN-111 in treating dwarfism should also begin next year. Biomarin is the longest-tenured stock in my SharkBiotech portfolio. It's nice to see patience rewarded. On a conference call Tuesday, Lexicon management said partnering discussions for its diabetes drug LX4211 are moving forward well, discussions are robust and they expect to announce a partner before beginning phase III trials next year. As a reminder, diabetes is quickly becoming an epidemic in this country and Big Pharma is eager to find new ways of treating this disease . LX4211 is unique in that it targets two different transporters of glucose SLGT-1 and SLG-2, which makes it unique in the new class of SLGT inhibitors. The phase II data on LX4211 were outstanding and Lexicon should have no problem lining up a partner sooner rather than later Lexicon shares are 25% in the last few weeks but I believe the current stock price is attractive for adding ahead of a possible partnership. Rosenblum is long BioMarin and Lexicon.
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