BioMarin Pharmaceutical Inc. (BMRN)

[ghmm]

We should be do for news in a couple areas. Besides the initiation of the sickle cell trial, I think that the Kuvan NDA gets filed this month (my estimate) and listening to the recent presentations JJ indicated that an update on the BH4 Cardio Vascular program would occur in a few weeks/about a month which should be about mid month as well.

While the update on the cardio vascular could be negative (they could elect to drop it or even altar the PAD trial) there were some vague encouraging remarks that they had some interesting findings in subsets (though retrospective subset analysis is not one of my favorite findings).

With BH4 in sickle cell disease, PAH and Erectile dysfunction we have three completely different indications that each could be a rather significant market.

In thinking about the potential for Kuvan I think it becomes a much bigger drug for a reason some analysts aren't noting. The company keeps emphasizing the IQ benefit and that this is not a lifestyle drug. I thinking pricing ends up being in the neighborhood of what Tracleer costs, in the 35-40K range. One way they could do this with less of a hurdle is price the low dose (5mg) at around 7500/yr for a "normal" weight individual. One of the analysts pointed out that physicians they talked to planned on usage at the high end of the range (20mg). Then factor in that PKU patients are probably a bit heavier then average because of their diet/getting enough nutrients and the price could easily be in the 30K/yr annual range.