In 1991, Martine Rothblatt got some jarring news from doctors: Her 8-year-old daughter, Jenesis, suffered from primary pulmonary hypertension, a rare lung disease that is difficult to treat and can be fatal.
A bibliophile, Rothblatt plowed into everything she could read on the subject. "I was really scared by what I read," she recalled.
Only about 5,000 cases of the disorder a year are diagnosed in the United States and Europe. In these patients, the blood pressure in the pulmonary artery connecting the heart to the lungs becomes abnormally high. It is marked by a shortness of breath, fatigue and fainting spells. Parents and patients hope that the standard therapy, a drug called Flolan, will work. Otherwise, they must seek a heart-lung transplant.
Rothblatt didn't like the outlook for Jenesis, or the tedious treatment regimen, which included wearing a backpack-size container filled with Flolan and a surgically implanted catheter.
So the satellite communications executive, no stranger to a big challenge, began a quest to find a better treatment.
The result of that eight-year effort: The 45-year-old woman now heads United Therapeutics Corp., which she founded in 1996.
Despite long odds, Silver Spring-based United Therapeutics has emerged as a favorite of biotechnology industry analysts and investors on Wall Street. Since June, when the company went public, its stock has more than doubled, closing Friday at $28.875.
"When you first hear about an executive forming a company because she's got a sick daughter, you tend to be cautious about what's really going on," said Robert J. Toth, senior biotechnology analyst at Prudential Vector Healthcare in San Francisco.
"But Martine hasn't let emotion get ahead of running a solid company. I would argue she's an extremely savvy business manager who's been making some very smart decisions," Toth said.
The chief reason for investor interest: Rothblatt has acquired rights to a small portfolio of promising drugs that big pharmaceutical concerns had either shelved, axed, or won't market in the United States.
The leading candidate is UT-15. Toth believes that the drug, which is in late-stage clinical trials, could be on the U.S. market as early as 2001.
He estimates that the drug could generate $75 million in revenue that year, and potentially $2 billion annually if it wins approval in Europe and the United States. The drug could make the company profitable, despite the relatively small number of people afflicted with primary and secondary pulmonary hypertension: about 40,000 in both markets.
Although its cost is expected to be about $50,000 a year per patient, it's still cheaper than the $100,000 a year Flolan costs, the reason health insurers are expected to embrace it.
The drug is designed to be delivered under the skin through a beeper-size pump patients could wear on their belts, eliminating the risk of infection and the need for a catheter. The company has a deal with MiniMed Inc. of Sylmar, Calif., maker of a miniature insulin pump, to market the drug and pump if approved.
Rothblatt also gets kudos from Wall Street for drafting top talent.
Since launching United Therapeutics with $30 million raised from investors, she has shed all other responsibilities.
"This," said Rothblatt, "is my mission in life."
The Los Angeles native, who holds MBA and law degrees from the University of California at Los Angeles, is no stranger to the rigors involved in starting a company. She has helped found or run at least three satellite communications companies. They include publicly held PanAmSat Inc., a Greenwich, Conn., satellite TV and communications company.
But a drug company brings its own set of obstacles.
For one, the time and cost of developing even a single drug are crushing.
The Pharmaceutical Manufacturer's Association of America estimates that it takes seven to 10 years and more than $300 million to develop a drug and get it to market. There is always high risk that the drug will fail.
Rothblatt said she was undeterred by the statistics. She believed that she could expedite getting a promising pulmonary hypertension drug tested and approved.
"I've spent my whole life having people tell me something would take 10 years, and getting it done in three or four," said Rothblatt, who used her graduate thesis at UCLA as a springboard for co-founding PanAmSat.
After her daughter's illness was diagnosed, Rothblatt compiled a list of the world's top pulmonary hypertension experts and founded the Pulmonary Hypertension Cure Foundation in 1994.
An epiphany of sorts came when a friend gave her the film "Lorenzo's Oil," which tells the powerful story of a couple's quest to find a treatment for their son's degenerative brain disease.
Rothblatt said the Oscar-nominated movie had an enormous impact, persuading her to dedicate her time and energy to getting a better drug developed.
"I knew I had to get smarter about this," said Rothblatt.
A name that cropped up often as Rothblatt plowed through professional journals and research was that of Dr. Robyn Barst, director of the Children's Pulmonary Center at Columbia-Presbyterian Medical Center in New York. Rothblatt took her daughter to be evaluated by Barst.
During one meeting, Barst told Rothblatt about a promising drug the then-Burroughs Wellcome & Co. had stopped work on after concluding that its potential market was too small.
"She said the best thing I could do would be to get the rights to that drug, and get it developed," recalled Rothblatt.
On Barst's advice, Rothblatt called James W. Crow, the scientist who had been project manager of the team working on the drug, known at Burroughs as BW15AU.
In 1995, Crow was among the legions of career Burroughs employees forced to leave when it was acquired by Glaxo & Co.
"Burroughs was the kind of company you married in, retired in and died in. I never thought for a minute I'd work anywhere else. I was really in shock when I left," said Crow.
Rothblatt called Crow to tell him about her daughter and that she was thinking of starting a company to develop better treatments for pulmonary hypertension. Crow thought little of it until February 1996, when Rothblatt asked him to come to Washington for a meeting. Crow said he would do so in May. There was a pause on the phone.
"She said, 'Well, it'll have to be sooner than that. I've just FedExed [airplane] tickets to you so we can meet tomorrow,' " Crow recalled.
The next day, Crow found himself at lunch in a large private room at the posh Four Seasons Hotel in Washington. Rothblatt asked him to sketch out what he would do if given the resources to develop a new hypertension drug.
Crow asked Rothblatt if she had any idea of the costs and risks involved. "She didn't even bat an eye when I told her," he said.
Within weeks the two had a deal and Crow his first task: tracking down BW15AU and acquiring the rights to it.
He told Rothblatt that licensing the drug could take three years. Rothblatt said that was too slow. She wanted it in a matter of months.
"I had nothing to lose and everything to gain. This seemed like an exciting opportunity to do something I'd never done before," said Crow.
After working his old Burroughs and Glaxo contacts, Crow advised Rothblatt that Glaxo Wellcome would license marketing rights and the patent.
Since then, United Therapeutics has broadened its drug pipeline to include three other drugs licensed from drug companies, including Berapost, a treatment for early-stage pulmonary hypertension. Analysts expect Berapost to hit the U.S. market by 2004 and potentially generate $225 million a year in sales.
Meanwhile, Jenesis, now 16, looks forward to the day she can sport a minipump instead of her bulky Flolan pack.
Crow believes that he's figured out what's behind Rothblatt's success with getting the improbable accomplished.
"I've watched Martine with great interest," said the scientist. "She has this technique where you are the center of the universe when she's talking to you. That translates into a very appealing relationship."
Said Rothblatt, "I fundamentally ask myself if I can succeed. It's scary each time starting a company. There are a hundred ways to fail and just a few to succeed. Each time I just felt it was my calling to do it."