BioMarin Pharmaceutical, Inc. (BMRN)

BioMarin has developed and commercialized three products in ten years, a remarkable accomplishment in the biopharmaceutical industry, and a testament to the company’s passion and dedication to patients with serious, unmet medical needs.
BioMarin has three approved products on the market today:
•Kuvan® (sapropterin dihydrochloride) Tablets for PKU
•Naglazyme® (galsulfase) for MPS VI
•Aldurazyme® (laronidase) for MPS I The year 2008 was a very important milestone in BioMarin’s history. We achieved our first profitable full year, with total revenues up 144 percent from 2007 to nearly $300 million, and increased sales across all of our three products. We continue to build an exciting pipeline, with a clinical portfolio featuring PEG-PAL for PKU and GALNS for MPS IVA, and a preclinical lineup that provides hope to many other patients with a variety of other rare diseases.
The year also brought with it the U.S. launch of Kuvan and we have been encouraged by the progress made thus far in a market with unique complexities. We continue to look for new ways to enhance our commercial efforts in this area and we remain very optimistic about the long-term potential of the product.
Other successes throughout the year included several regulatory achievements such as the approval of Naglazyme in Japan and South Korea, and the recent approval of Kuvan secured in the European Union by our partner Merck Serono.
In 2008, BioMarin achieved important milestones that will fuel future growth opportunities in the orphan drug industry. Today, as a fully integrated mid-cap biopharmaceutical company with three products on the market, strong revenues and cash reserves of nearly $560 million, we are confident and well positioned for the long-term. In 2009, we will continue to advance our pipeline and actively pursue valuable business development opportunities to bring increased value to patients, shareholders and employees. Logical buyout candidate In the BIOTECH field,should reward shareholders Into the future... TS

i usually don't by based soley on a recommendation without researching it my self, but I did this one. probably a mistake?

Three approved drugs which are all classified as orphan drugs make this a slightly profitable company. Pipeline is a few years out so look for them to try to make a near term acquisition or be taken out themselves.

With the economy coming back and baby boomers getting older, this company should do very well.

Pharmaceutical companies should remain strong, even with a relatively high P/E I still like its chances in the next year.

Looking for a bounce off todays 52-week low...sometime in the next 12 months seems reasonable.

A solid biotech is beaten down. A 1.2 billion market cap company with revenue $300-400 million a year. Good management team. It should outperform the market.

A rising star following GENZ. Good deal with LJPC recently on lupus drug.

In the early stages of a big growth cycle.

30

Has a good position in the market for drugs that can help people.

I own it and I'm scared to sell. They're bound to keep coming up with new drugs.

P/E Ratio of 985.4 is over 47x industry.

May be over leveraged with 2.52 Debt/Equity and 11.1 Current.

Decent income indicates that this is likely a strong company that is simply overpriced at the moment.

Genzyme Jr
Possibly to be absorbed by Genzyme Sr.

A biotech that delivers on it's pipeline implies excellent research and mgmt.

684.75 P/E is absurd.

Continuation Wedge
score = 93

Biomarin targets diseases caused by genetic defects. Most of these are relatively rare, so there is not a large market, and this makes it less likely that a lot of competitors will get into the business. Also, the nature of the drug creation and testing is pretty technical and demanding in terms of the genetics, biochemistry and clinical support needed. Although this does not create a wide moat, it does create somewhat of a sustainable competitive advantage, as the research infrastructure and personal contacts developed for one genetic disorder will apply to a fair extent to other disorders.

On the other hand, since many of the diseases caused by genetic defects are quite severe and disabling, often leading to physical deformities, neurological malfunctions and mental retardation, the cost of caring for these individuals if they are not treated is quite high.

Thus, BMRN's products produce great value to society, by reducing the cost of lifetime care for otherwise developmentally disabled individual, or even allowing them to grow up to become tax-paying citizens. Its products can be priced accordingly. The affected individuals will need to remain on BMRN's products their entire life, barring advances in genetic therapy that will permanently correct the inborn errors in metabolism. Even when medical modification of our genetic make-up becomes possible, useful and safe, (still quite a few years off, imho) it will almost certainly be applied first to diabetes, cardiovascular disease, and other much more common conditions.

Thus, it seems quite likely that patients on BMRN products will take them for many years, leading to a predictable recurring revenue stream.

A further reason BMRN should outperform the S&P over the next 2 to 4 years (my rating) is that it rose from a low of under $4.00 a share (November 2004) to $40 last January. It is more than 35% off that high right now, apparently in large part due to slower than anticipated growth in sales of Kuvan, its newly approved drug for treating phenylketonuria (PKU), and likely also reflecting the general market malaise. BMRN has two other successful and growing drugs on the market, Aldurazyme and Naglazyme. The long-term story remains intact, or as there are no other drugs to treat PKU with which Biomarin needs to compete for market share. If doctors are a little slow to catch on to using Kuvan, it is only a minor bump in the road. Most of BMRN's products are the only option for the conditions they treat.

PKU results in high levels of phenylalanine (Phe), about which the FDA says, "In infants and children, high blood Phe levels over time can result in mental retardation, smaller brain size, delayed speech, and other neurologic problems. In adolescents and adults, high blood Phe levels can result in problems with concentration and attention." Treatment with a protein-restricted diet (no dairy products, meat, fish, eggs, beans, and nuts) is the only other option, and while this works, it is not exactly the best diet for a growing child.

All newborn babies in the US are tested for PKU, and about 1 in 15,000 has it. About half of people with PKU respond to Kuvan, so this is a potential market of 7,500 new patients per year in the US. Treatment costs about $55.000 a year. Biomarin had hoped to get 1,000 patients started on Kuvan in 2008, but won't make that goal. Nevertheless, with 7,500 newborn babies every year representing over half a million dollars in new patient business for Kuvan, BMRN only turning profitable this year, more drugs in the pipeline, and the stock off 35%, it seems very, very likely to beat the S&P over the next few years.

Steve Tackett-Nelson M.D.

Diplomate, American Board of Psychiatry and Neurology

(Long BMRN and soon to buy more)

THis company is in a good market because Pharmeceuticals will always be needed, but a PE ration of 659 is simply unsustainable. A PE of above 40 is high, but this companies stock is over 600 times more expensive than its earnings per share. That's bad.

selected this stock two years ago and still love its potential