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From soil in Haiti, hopes for antibiotic

Local biotech firm gets $70m in venture funds as 'superbug' fears rise

With doctors increasingly worried about "superbugs," deadly infections that can defeat most known drugs, a small Cambridge biotechnology firm has won $70 million in venture capital money to develop a powerful antibiotic from a microbe discovered in Haitian dirt.

Reflecting the renewed importance of antibiotics, once spurned as a low-profit sideline in the drug industry, the venture investment is the biggest in a New England biotech company in nearly a year, and one of the largest nationwide.

"We're in a very dire situation in the antibiotic business, where nobody really knows where the next good antibiotics are going to come from," said Eric Gordon, a venture capitalist and former antibiotic researcher who helped finance the Cambridge company, Targanta Therapeutics Inc.

Targanta's drug is still experimental, but it joins a series of new efforts to attack strains of bacteria that kill thousands of people each year.

The past several years have seen a handful of small biotechnology companies emerge to develop new intravenous antibiotics, including Cubist Pharmaceuticals Inc. of Lexington. Its drug, Cubicin, won approval in 2003, and the company sells about $200 million worth of the treatment annually. Two other drug firms have applied for approval to sell similar antibiotics.

Targanta Therapeutics hopes to be next in line. But the path to develop its drug, oritavancin , has been anything but a straight line from its roots in Haiti.

The drug was discovered not by Targanta in Cambridge, but by scientists at Eli Lilly & Co. in Indiana during the 1990s.

At the time, Lilly's department of antibiotic researchers was one of the most respected in the country. In analyzing bacteria-rich samples of tropical soil, the researchers noticed one type of bacteria in a sample from Haiti appeared to be extremely effective at fending off attacks from rival bacteria.

"You've got the Darwinian battle going on under the ground in Haiti," said Targanta chief executive Mark Leuchtenberger . "Somehow, this became the don't-mess-with-me microbe."

Following a standard path in antibiotic research, Lilly researchers isolated the microbe's bacteria-killing chemical and tweaked it repeatedly, finally creating a new drug more effective against bacteria and less toxic to patients.

Instead of developing the drug further, however, Eli Lilly exited the antibiotics business. Because antibiotics are prescribed for only a matter of days, they promise smaller long-term sales than many other pharmaceuticals. Lilly's leaders decided to focus their research on mental-health drugs, such as its blockbuster, Prozac, and sold its prospective new antibiotics piecemeal to smaller firms.

One of the substances, daptomycin , was licensed to Cubist in 1997 and eventually became Cubicin. Another drug, oritavancin, was licensed in 2001 to a California firm called InterMune Inc., which sank millions of dollars into human trials, but ultimately decided to sell the rights to oritavancin rather than invest the money needed to bring it to market. A year ago, the drug changed hands again, this time going to Targanta, a 10-year-old research company founded in Montreal .

"The smaller companies are doing what the bigger companies should have been doing, which is bringing these things to market," said Gordon.

Superbugs have become an especially dangerous and expensive problem for hospitals in the United States as more patients each year develop bacterial infections that resist not only penicillin and other common pills, but newer and stronger antibiotic treatments. One family of bacteria alone causes 90,000 serious infections annually, about 17,000 of them fatal.

"These are rather scary organisms, and as of the moment we're not even sure how they're spread," said Dr. Robert Moellering , an infectious-disease specialist at Beth Israel Deaconess Medical Center, who is a scientific advisor to Targanta.

The prevalence of new bacterial strains, especially within hospitals, means patients admitted for medical treatment can sometimes contract an unrelated infection that leaves them fighting for life. The most common superbug, a drug-resistant strain of staphylococcus , can cause a range of conditions from painful skin abscesses to deep infections of the bones or heart valves. Treatment can take weeks, and even then may not be effective.

Hospitals spend about $800 million a year on drugs to treat such infections, usually with potent antibiotics delivered through an intravenous line. For years, doctors have reserved one antibiotic, vancomycin , as a "last line of defense," but researchers are reporting that some new bacteria can survive vancomycin treatment.

Targanta's drug has been tested on about 1,500 patients with serious skin infections, and has shown encouraging results. The company sought new investors to help fund its application for federal approval and to begin testing the drug against a broader range of diseases,

"There are a lot of so-so drugs that people have dragged out of the closet, but we recognize this one as something that was medically important and would fill a need that's growing," said Gordon.

His firm, Skyline Ventures of California, joined with several other investors in the $70 million financing round.

To bring oritavancin to market, Targanta hired biotechnology veteran Leuchtenberger as chief executive in September and moved its headquarters to Cambridge. Only five employees work there, with the rest in Indiana and Montreal.

In a sign of how quickly fortunes turn in biotechnology, the Indiana office is staffed with a number of former Lilly scientists who worked together during the Midwestern company's days as an antibiotic powerhouse.

Meanwhile, Leuchtenberger didn't have far to look for office space in Cambridge. His previous company, Therion Biologics, suffered a clinical setback and filed for bankruptcy last year. The Therion office was still empty, so Leuchtenberger simply moved back into his old suite. Earlier this week he hired a former Therion colleague to be his finance chief.

"It's unusual to be sitting in the same office," Leuchtenberger said of his return, "but sitting with the same people? That's pretty common."

Stephen Heuser can be reached at sheuser@globe.com.

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