Ever since 13-year-old Hanna McGary was born she has been on an extremely restricted diet; regular bread, pasta, meat, rice, beans and dairy products are all off-limits.

"It was hard going to birthday parties. She can't have cake or anything like that," said her mother, Marie Duellge of Santa Rosa.

Hanna suffers from phenylketonuria, a rare genetic disease that affects 1 in 15,000 newborn babies. The disease makes the body unable to tolerate anything more than a low-protein diet. If Hanna ate a typical diet, the result could be mental retardation, brain damage and seizures.

For years, adherence to a strict diet was the only treatment available to people like Hanna. But late in 2007, Novato-based BioMarin Pharmaceutical received approval from the Food and Drug Administration to begin selling Kuvan, the first specific drug therapy for the treatment of phenylketonuria. Hanna, who was part of the drug trial for Kuvan, has been taking the drug for two years now.

"Her diet is less restricted," Duellge said. "She can have a lot more protein every day."

BioMarin, one of only a handful of companies focused on the development and sales of orphan drugs such as Kuvan, saw its revenue jump 168 percent between 2007 and 2009 to $324.7 million. The company, the crown jewel of Marin's nascent biotech sector, is poised for more growth in 2010 with several potential new products in the pipeline.

Last month, the company celebrated an 80,000-square-foot internal expansion of its building at 46 Galli Drive. The $60 million project will triple BioMarin's manufacturing capacity, all of which is based in Novato, to 45,000 square feet of clean room space.

"We needed to expand our ability to manufacture proteins very substantially," said BioMarin CEO Jean-Jacques Bienaime.

It's difficult to pronounce the names of BioMarin's three drug products - Naglazyme, Aldurazyme, Kuvan - and even more difficult to explain the rare diseases that they treat. Bienaime said he doesn't bother trying when casual acquaintances inquire about his company.

"I just say that we're a company whose business strategy is to target rare, genetic medical disorders," Bienaime said. He adds that even though the number of people who suffer from these disorders is small, "We have a major impact on the quality of life or survival of those patients."

BioMarin is one of only a handful of companies focused on the orphan drug market. For years, there was little incentive for companies to develop drugs for a category of diseases that were so rare that the cost of developing drug treatments would exceed the possible payback. That all changed in 1983 when Congress passed the Orphan Drug Act. The legislation defined an orphan drug as a drug that treats a disease affecting fewer than 200,000 people in the United States. It gave orphan drug developers exclusive sales rights for seven years plus tax incentives for conducting clinical trials.

In addition to its three approved products, BioMarin is on track to launch a fourth one, Firdapse, on a country-by-country basis in the European Union later this month. And Biename said a potential product with even more potential is named GALNS.

"We have some early results in phase two trials that are very positive, so we believe the probability this product will make it to the market is pretty high," he said.

Bienaime said it's possible that GALNS could be approved for sale in the United States as early as 2013. If so, the new manufacturing capacity that BioMarin has just added will be put to good use.

"It will be our largest protein product," Bienaime said. He estimates that at its peak the product could produce yearly revenue of $600 million to $800 million.

Andrew Vaino, a stock analyst with Roth Capital Partners, issued a buy recommendation on BioMarin's stock in February.

"The company has three approved products and our take is that sales of those going forward are pretty predictable," Vaino said. "The additional bonus with these guys is they have three phase two clinical studies that are going to read out in the next two quarters, and we think if just one of those three hits it will also give the stock a pop."

Vaino said BioMarin has found its niche "developing enzyme therapies for ultra-orphan diseases," and demonstrated an ability to "find the patients to take their medicines."

Shiv Kapoor, an analyst with Morgan Joseph & Co. Inc., said BioMarin is a healthy company with a solid future. But Kapoor is not recommending the company's stock at its current price.

"I see some upside in the company but not enough to aggressively suggest people to buy," Kapoor said.

BioMarin lost about $500,000 in fiscal 2009 despite a 9.5 percent increase in revenue. Beiname is forecasting a small profit for 2010.

On Friday, the company's stock, which trades on NASDQ under the symbol BMRN, closed at $22.04 per share, up 37 cents - above its 52-week high of $21.70 per share. The stock went public in 1999 priced at $15 per share. The stock's 52-week low was $9.93 a share.

Because the diseases that BioMarin's drugs treat are so rare, BioMarin needs to market its products worldwide and charge high prices to achieve profitability. In addition to its 640 Marin employees, the company employs another 90 people in London, San Paulo, Brazil; Istanbul, Turkey; Hong Kong and Shanghai. Bienaime estimates the company will hire another 90 employees in Marin by the end of this year.

"These are very specialized jobs requiring technical skills and pay very competitively," Bienaime said.

BioMarin does some of its research and development work in its own labs in Novato. The company has eight buildings totaling 500,000 square feet of space in Marin. The company sometimes rents lab space at the Buck Center for Research on Aging in Novato. It also collaborates with universities around the world for additional research. In 2009, BioMarin boosted research and development spending by 23 percent to about $115 million.

Bienaime said the nation's economic crisis has slowed BioMarin's growth. "But it's not having a major impact on us because the products that we market are life-saving therapies," he said.

Bienaime said that while the economies of the United States and the European Union remain weak, other economies, in China and Brazil for example, are doing well. Relying heavily on foreign sales is a double-edged sword, however. Fluctuations in foreign currency rates cost BioMarin $7.7 million in the fourth quarter of 2009. The losses occurred despite the company's hedging strategies.

"We started hedging about three years ago to protect ourselves from wide variations in foreign exchange rates between the U.S. dollar and the euro," Bienaime said. "The good news is that in Latin America where we have a lot of Naglazyme sales, we sell mainly in U.S. dollars so we have no foreign exchange exposure there."

Beinaime said BioMarin could benefit if Congress passes a health reform package. Both versions of the plan, the House of Representatives' and the Senate's, give the producers of biologic drugs 12 years of protection from competition from generics. Biologic drugs are grown inside living cells. BioMarin uses hamster cells that have been engineered to make human proteins to produce Naglazyme and Aldurazyme.

BIOMARIN'S DRUGS

- Naglazyme: Treats a disorder that inhibits the metabolizing of lipids, sugar containing proteins and mucopolysaccharides. The disease affects mostly children and can cause developmental delay, movement disorders, seizures, dementia, deafness and blindness. Approximately 1,100 patients in developed countries worldwide have this disease.

- Kuvan: Treats phenylketonuria, an amino acid metabolizing disorder that can cause mental retardation, brain damage and seizures. Affects 1 in 15,000 births.

- Aldurazyme: Treats a progressive and debilitating life-threatening genetic disease for which no other drug treatment currently exists. The disease causes the build-up of carbohydrate residues in all tissues in the body. Symptoms include inhibited growth, delayed and regressed mental development, enlarged liver and spleen, joint deformities, impaired cardiovascular function and reduced pulmonary function. Affects 1 in 100,000 births.

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