Biomarin (BMRN) A Top Biotech Stock For Piper Jaffray Managing Director; MPS IV Drug Expected To Be Market-ready By 2013

67 WALL STREET, New York - November 28, 2011 - The Wall Street Transcript has just published its Biotechnology and Pharmaceuticals Report offering a timely review of the sector to serious investors and industry executives. This special feature contains expert industry commentary through in-depth interviews with public company CEOs, Equity Analysts and Money Managers. The full issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online.

Topics covered: Orphan Drug and Biologics Manufacturing - U.S. and European Drug Pricing - Biotechnology and Pharmaceutical Companies Valuation - Domestic Regulatory Uncertainty

Companies include: Eli Lilly (LLY); InterMune's (ITMN); Thoratec (THOR); Abbott (ABT); Acura Pharmaceuticals (ACUR); and many more.

In the following brief excerpt from the Biotech And Pharmaceutical Report, expert analysts discuss the outlook for the sector and for investors.

Ian Somaiya is a Managing Director and Senior Research Analyst at Piper Jaffray & Co. covering the biotechnology industry. Prior to joining Piper Jaffray in 2010, Mr. Somaiya worked in equity research at Thomas Weisel Partners Group. Mr. Somaiya was recognized as Best on the Street by The Wall Street Journal in 2006, 2007 and 2009 for his biotechnology coverage. Formerly, Mr. Somaiya was a Senior Analyst at Morgan Stanley with responsibilities for small- and mid-capitalization biotechnology companies. He spent a year with The Carson Group in its health care consulting practice prior to making his transition to the sell-side at Prudential Securities. Mr. Somaiya received a B.A. in biology/neuroscience from New York University.

TWST: What is driving the new wave of products?

Mr. Somaiya: A lot of it is timing. When you have a biotech industry with a large pipeline of drugs moving to mid-to-late stage development, and many of those products have delivered positive Phase III data and have presented to the FDA or will be in front of the FDA in a matter of months. It's just a case of that productivity has caught up and the question now is whether those products will live up to expectations. We've seen two drugs launched this year with high investor expectations that disappointed - Dendreon's (DNDN) Provenge and Human Genome Sciences' (HGSI) BENLYSTA.

We move into 2012 and with Biogen Idec (BIIB) launching BG-12, an oral drug for the treatment of M.S., we look at smaller companies like Incyte (INCY) and its launch of a drug for myelofibrosis, and these all represent the new product wave in biotech. The question now is how successful will they be in the marketplace.

TWST: So who are your favorite names in your coverage right now and why?

Mr. Somaiya: My favorite names right now are really focused on orphan diseases where I think we have a lot of insulation in terms of macro concerns. Orphan drugs are focused on smaller patient populations. They are priced higher, so one might think there is going to be more price sensitivity, because some of them are priced at $250,000 to $400,000 a year. But their benefit is near absolute, as long as the patient receives the drug earlier in the disease cascade. The two companies that I am really focused on right now are Alexion (ALXN) and Biomarin (BMRN). Alexion is a larger-cap name, probably about $12.5 billion to $13 billion market cap; Biomarin is a fraction of that. Alexion has a drug Soliris, which is approved for two different ultraorphan indications, but clinical data supports use in probably three or four new ones. Across these highly divergent diseases, the drug seems to offer benefit to a vast majority of patients and that's unique.

The stock had an amazing run over the past year, and probably has been one of the better performing stocks since the middle of last year. But we're also looking at a revenue stream that approaches $800 million this year that could generate peak sales of north of $5 billion. So we're still early in terms of its eventual opportunities. Biomarin is very different. They have a highly productive R And D platform. They have four drugs on the market today. Their pipeline is finally starting to materialize. They have a drug for a disease called MPS IV. The drug could produce positive Phase III data by the end of next year and be on the market by the end of 2013. And the company has done a great job of making sure the launch of the drug is successful. They have already identified over a thousand patients with MPS IV.

So when the product does get approval in the U.S. and the rest of the world, those patients that have been identified will become immediate candidates for the drug, and that should ensure a fairly robust launch. The company has two other drugs that I am excited about. One is for Pompe disease, which is a market currently served by Genzyme and Sanofi (SNY). But I think Biomarin has come up with a better enzyme, which could be given at lower doses or has better efficacy at the same dose as Genzyme's Myozyme.

And for the patient what that would mean is, instead of 10% of patients who are responding to Myozyme, there is a potential for up to 40%, 50% Pompe's patients responding to Biomarin's enzyme. So again, higher response rate, a greater degree of benefit potentially at the same dose - if not lower dose - and that's something I think would allow Biomarin to have another product in the market with an opportunity that could be $750 million to $1 billion at peak, just like the MPS IV products.

The Wall Street Transcript is a unique service for investors and industry researchers - providing fresh commentary and insight through verbatim interviews with CEOs and research analysts. This special issue is available by calling (212) 952-7433 or via The Wall Street Transcript Online.

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