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Drug Companies Research Rare Diseases in North Carolina

Posted on: Friday, 15 April 2005, 21:00 CDT

Apr. 15--The Triangle has always been a hot spot for unprofitable drug research and development -- but that's not necessarily a bad thing.

Indeed, start-ups, as young companies are called, are the best hope for people in need of drugs that treat rare diseases.

Large pharmaceutical companies such as GlaxoSmithKline and Pfizer tend to focus on potential blockbusters, drugs that generate more than $1 billion in annual sales, and ignore experimental drugs for diseases affecting a small number of patients.

That's why companies such as Metabolon, Icagen and Parion Sciences are needed.

Metabolon, a biotech company in Durham, is conducting research into Lou Gehrig's disease, which it says affects about 30,000 people in the United States.

Parion Sciences, a Durham drug development company, focuses on treatments for cystic fibrosis. The company says about 35,000 Americans suffer from the disease.

And Icagen, another Durham drug development company, is working on a drug for sickle cell disease, which it says affects about 100,000 in the United States.

Few if any drugs are on the market to treat these diseases. Still, a corner on the market doesn't mean huge revenues. But that's not a problem for the start-ups.

"Small companies are not so hungry for billion-dollar revenue," said Kay Wagoner, Icagen's chief executive. "For them, $50 million or $100 million in revenue is good."

The National Institutes of Health estimate that about 20 million Americans are affected by about 6,000 rare diseases.

Government incentives exist to research rare diseases, which are defined by the Orphan Drug Act as those affecting fewer than 200,000 Americans. Treatments, also known as orphan drugs, can be developed with the help of grants from the institute or nonprofit support groups. Also, orphan drugs tend to pass regulatory review faster, and once they're approved, they are protected from competition for several years.

Still, only 26 orphan drugs came to market in the past three years, institute records show.

Being able to justify expenses for research and development remains key.

The sickle cell treatment is Icagen's most advanced drug. Outside the United States, the drug targets more than 4 million Africans suffering from the disease. The company, which employs 75, has six other experimental drugs in its development pipeline.

Parion, which employs 10, is eyeing the $20 billion worldwide market for a chronic bronchitis drug, said Ross Johnson, the company's chief executive. Cystic fibrosis and chronic bronchitis have similar symptoms.

Metabolon, which employs 22, is financing its research of Lou Gehrig's disease with institute grants. So far the company has received three, each worth $100,000 to $200,000.

John Ryals, chief executive of Metabolon, expects to attract about $2 million worth of grants for the work.

"We'll take this as far as we can," Ryals said. Then he hopes to sell the rights to use the information.

So far, Metabolon has raised about $9 million in venture capital. The money supports research in other neurodegenerative diseases, specifically Alzheimer's and Parkinson's.

"We couldn't justify putting our own money into this," Ryals said about the Lou Gehrig's research. "It's not a large enough population."

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Copyright (c) 2005, The News & Observer, Raleigh, N.C.

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GSK, PFE,

Source: The News & Observer

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