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In The News

BioMarin’s Molecules Yield Miracles

Kendra Gottsleben turns 30 on this early November morning, the day I write her story. She stands only 3 feet, 3 inches tall, but her irrepressible spirit soars to the heavens.

Starting with symptoms at age 4, Kendra survived mucopolysaccaridosis type VI in part due to weekly hospital visits where she sits five hours for infusion of a $350,000- to $1.3 million-a-year biotech drug called Naglazyme from San Rafael-based BioMarin Pharmaceutical.

An only child, her tongue was swollen. Her hands curled inward. Her knees bent so she struggled to walk. She was tiny compared to kids her age. Her mother took her to an orthopedic doctor, who pronounced her fine. Her condition worsened.

Her mother, an elementary school principal, brought her to a geneticist who diagnosed MPS VI, also called Maroteau-Lamy syndrome. In severe cases, patients die by adolescence.

MPS VI, a rare genetic enzyme-deficiency disease, hits three to four children per million. Ten other MPS types inflict greater suffering and earlier mortality than VI; some damage the brain.

Often a drug’s human touch outshines its biotech marvel. Scientists who develop miracle molecules find themselves tugged into a deeper heartfelt mission – giving Kendra and others a taste of the health that makes many of us grateful. Naglazyme helps restore a missing enzyme in MPS VI patients that cleans out waste products in body cells.

Since 2005, BioMarin has been led by Jean-Jacques Bienaimé, a Frenchman who prefers that colleagues call him JJ rather than botch the nasal French sound. His last name means “beloved,” a fitting moniker for captain of a company that he saved. Though he considered medical school, Monsieur Bienaimé, who chuckled as he confessed squeamishness about blood, followed a knack for business and earned an MBA from Wharton. When he graduated, biotech was an infant. He worked at Genentech, later for the company now known as Sanofi-Aventis and then Sangstat Medical, acquired by Genzyme.

The year before he “joined,” BioMarin lost nearly $200 million. Its market capitalization was about $300 million with only $5 million revenue. Now market capitalization is $12.5 billion with $700 million revenue, though BioMarin’s net loss for the nine months ending Sept. 30 was $114 million; accumulated deficit was $780 million. There were 300 employees in 2005; now there are 1,500 with 1,000 in the Bay Area and, by next year, expected growth to a total of 1,750.

If the FDA nods OK to BioMarin’s pipelined drugs, revenue could gallop as the company transforms into racehorse. “Initially I had to get the horse started,” Monsieur Bienaimé said. “The horse was dying. We got it going again. It was pretty bad.”

“I downsized within three weeks of being CEO,” he said. “The company was low on cash, $10 million or $15 million in the bank, because of this bad acquisition the year before,” the $175 million purchase of the Medicis Pharmaceuticals pediatric business for its Orapred drug used to treat asthma and allergies. Orapred went generic, obliterating sales. He laid off 100 people. “I had to write off the entire investment,” Monsieur Bienaimé said. “Shareholders were pretty unhappy, with stock price at $4 a share. They had lost a lot of money and were irritated.” The stock recently traded at $85. A proxy fight – battle by shareholders for board control – helped bring him aboard.

Drug approval can take 10 to 15 years and cost $500 million up to $2 billion. BioMarin specializes in ultra-orphan drugs for diseases that affect few people, and aims for approval in five years.

The Orphan Drug Act of 1983 and Rare Diseases Act of 2002 provide market exclusivity for 7.5 years in the United States for companies that produce orphan drugs for pediatric illnesses affecting fewer than 200,000 Americans. Some 6,000 such illnesses affect a total of nearly 25 million Americans; hundreds of new diseases are discovered yearly. “It will be awhile before we hit the ceiling here,” he said.

BioMarin’s five approved drugs, mainly ultra-orphan drugs, comprise entries in a gargantuan potential market of diseases with fewer than 10,000 patients worldwide. Naglazyme, which Kendra takes, has 1,500 patients in developed countries excluding India and China, with about 200 in the United States. Vimizim’s population is 2,000 to 3,000 patients worldwide.

“When you get down to 500 patients,” he said, “it’s difficult to recover your investment.”

Small molecules such as Kuvan face earlier attacks from generic manufacturers. Complex designer molecules such as Aldurazyme and Naglazyme are tough to replicate with biosimilars. Exclusivity windows expired, but no competitors attacked either drug. “Our proteins are so complex with a three-dimensional structure,” Monsieur Bienaimé said. “If someone wants to copy it, it will be a different product. It’s very tough for them to make money.”

Another intriguing factor about BioMarin’s drug market niche: small patient populations mean close company contact with folks who become loyal. “If someone tries to switch them to their own generic biosimilar, we will know about it from the very, very early stage,” Monsieur Bienaimé said.

Major competitors include: Boston-based Genzyme, part of Sanofi; Shire Pharmaceuticals, based in Dublin, Ireland; and Alexion Pharmaceuticals, based in Connecticut.

“It’s a high-touch market,” he said, “a very close relationship with our patients. You hold the hand of the patients,” helping ensure payment for infusions. BioMarin employs an outside “reimbursement support” company in San Mateo to help patients obtain payment.

For a drug with a big market of $5 billion, another company could justify spending $100 million to $200 million to make, test and market a biosimilar drug.

BioMarin has unusual culture. “People like to make a big difference in a small patient population, helping very debilitated, suffering patients with limited life expectancy and terrible quality of life,” Monsieur Bienaimé said. His face softens. “Those patients are in need. You get hooked to it. You do.” Patients join the BioMarin family. “Big pharma has difficulty entering this market,” he said.

“When you sell a drug for $400,000 a year, it costs $65,000. There is no way we could charge $10,000. We would go bankrupt in like two months. We need the margins to fund the research.” The company aims to launch a new drug every 18 months.

With many drugs in BioMarin’s pipeline, “if we stop development of the drug, those patients will be dead,” he said. “There is nothing. We try to solve big problems – dwarfism. We are not going after a better painkiller.”

Kendra, affected by dwarfism, volunteered for clinical trials of BioMarin’s Naglazyme in Oakland, and stayed on it after FDA approval. Dakota Care insurance plus Medicaid pay for the medication, priced based on body weight.

When she told me it was her birthday, Kendra giggled in her pixie-like voice. She trounced the odds with biotech help, and spoke from a Sioux Falls hospital during weekly infusion. “My condition led my path,” Kendra said. “I accepted it. I would not be who I am if I did not have this. Some days I am down. I have had a lot of wonderful people come into my life because of what I have. I wouldn’t be who I am if I didn’t have it.”

Naglazyme brought some normalcy to Kendra’s life, which otherwise could have turned far worse. On weekends, “I go shopping,” she said, giggling again. “I visit with family and friends.”

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