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A mother's race for a cure

One entrepreneur plunged into biotech to save her daughter's life.

By Maggie Overfelt, FSB writer

(FSB Magazine) -- It took a few years before Martine Rothblatt got used to describing her daughter's chronic lung disease as a lucrative market opportunity. "I choked every time I said it - it sounded so immoral," says Rothblatt, 52. But when she realized that the fastest track to a cure was to launch a biotech firm and then take it public, Rothblatt started United Therapeutics (unither.com).

That was in 1996, and today she is convinced that capitalism is the answer. "I no longer have any doubt about the benefit of using profit motivation to develop cures," she says. "It's the language I need to build the company," she says. "It's the language that Wall Street understands."

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United Therapeutics founder Martine Rothblatt at her office in Silver Spring, Md.

After four consecutive years of growing annual revenues by about 40 percent, Rothblatt has proved to be a deft linguist. United Therapeutics (Charts), No. 22 on the FSB 100, reported $159 million in revenues last year. The company, based in Silver Spring, Md.,makes and sells Remodulin, a drug that treats pulmonary hypertension (PPH), abnormally high blood pressure in the arteries that supply the lungs. The rare, incurable, and often fatal ailment causes shortness of breath, fainting spells and fatigue. Most treatment options, ranging from daily pills to intravenous medicines, are fully reimbursable by insurance companies.

Sitting beneath bright paintings of DNA strands bursting from planets and stars, commissioned by Rothblatt for her company's headquarters, she describes watching her daughter, Jenesis, battle PPH when she was diagnosed as a little girl. Jenesis survived on a mix of pills, but doctors warned that if her condition worsened, she would have to take Flolan, a GlaxoSmithKline (Charts) drug that stays in the bloodstream for only three minutes. It must be continually administered via a catheter threaded directly into the groin or neck. Unstable at room temperature, Flolan requires patients to carry an ice pack 24 hours a day to ensure that it stays cool.

"The treatment seemed worse than the disease," recalls Rothblatt, stroking one of the three mohawked Labradoodles that roam her offices. "My vision became an inhaled version of the medicine." Her first step was to develop a drug that lasted longer than Flolan.

Rothblatt had no background in biotech: She had started her career as a corporate lawyer and gone on to help launch three successful satellite-communication companies, including Sirius Satellite Radio (Charts). In 1995, Rothblatt sold some of her telecom stock and used the proceeds to fund nonprofit PPH research. But after spending $2 million over two years, she saw little progress and grew discouraged.

She started researching the disease herself and found more than just a potential treatment - she found a business opportunity. The National Institutes of Health estimates that more than 200,000 suffer from PPH worldwide. Each patient pays as much as $150,000 a year for treatment - a $7.5 billion market in the U.S. alone.

After selling her vision of an inhaled PPH therapy to angel investors, Rothblatt tracked down and recruited two scientists who had developed a promising PPH drug at Burroughs Wellcome before it merged with Glaxo in 1995. It took Rothblatt seven months to license the compound, which Glaxo had shelved in favor of Flolan. Rothblatt founded United Therapeutics in 1996 and took the company public three years later.

Reaching her goal of developing an inhaled drug would take years of clinical trials, and Rothblatt had to find a way to keep investors happy in the meantime. "Something I learned from my satellite companies - you have to develop iterations of your products over time, producing real revenues incrementally," she says.

In 2002 the FDA approved the company's first version of Remodulin, the compound she licensed from Glaxo. Remodulin, which patients inject under their skin, lasts as long as three hours in a patient's bloodstream and doesn't have to be cooled. The drug, say some pulmonologists, is easier and safer to administer than Flolan. Neither a spokesman for Glaxo nor one for the drug's U.S. distributor would comment on the ease and safety of the use of Flolan.

United Therapeutics in 2004 introduced the drug in an IV form that is less painful to inject than the original version. Later this year the company hopes to complete clinical trials on the inhaled and tablet forms of Remodulin, which are expected to hit the market in late 2008 and 2009, respectively. But Rothblatt faces a lot more competition today. At least four companies now offer PPH drugs. One, Ventavis, is an inhaled medication produced by Actelion (actelion. com), a San Francisco biotech firm.

Ironically, the young girl who inspired Rothblatt to found United Therapeutics has not benefited from the company's work. Jenesis Rothblatt is 22 today and still takes pills for her PPH (her condition never became severe enough to require Flolan or Remodulin).

Rothblatt, a transsexual, pursues many interests outside biotech. She has published four books, including "The Apartheid of Sex: A Manifesto on the Freedom of Gender" (Rivers Oram Press, 1996) and "Two Stars for Peace" (iUniverse, 2003), in which she argues that the U.S. could bring peace to the Middle East by granting U.S. statehood to Israel and Palestine. She also sits on the boards of several organizations that study the use of technology to extend human life.

Yet her primary goal is to grow United Therapeutics by finding treatments for other chronic illnesses neglected by big pharmaceutical companies. The firm's first drug outside the pulmonary market, OvaRex, promises to help keep ovarian cancer in remission (it is in the last phase of clinical trials). Says Rothblatt: "In an industry dominated by dinosaurs, we're going to concentrate on carving out our own little niches, helping the chronically sick live an easier life." Top of page

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Market indexes are shown in real time, except for the DJIA, which is delayed by two minutes. All times are ET. Disclaimer Morningstar: © 2014 Morningstar, Inc. All Rights Reserved. Disclaimer The Dow Jones IndexesSM are proprietary to and distributed by Dow Jones & Company, Inc. and have been licensed for use. All content of the Dow Jones IndexesSM © 2014 is proprietary to Dow Jones & Company, Inc. Chicago Mercantile Association. The market data is the property of Chicago Mercantile Exchange Inc. and its licensors. All rights reserved. FactSet Research Systems Inc. 2014. All rights reserved. Most stock quote data provided by BATS.