Sometimes results come back from a pivotal Phase III trial of a new drug and “You just go wow!” said Trimeris director of clinical research, Lynn Smiley, at a press conference Wednesday morning.
“This was one of those times,” she said of the data from Trimeris’ most recent Phase III trial of its lead anti-AIDS drug, T-20. Trimeris hopes to begin selling the drug next year, pending final federal approval.
Smiles were apparent all around as Trimeris executives reported on the company’s experience at 14th International AIDS Conference in Barcelona, Spain last week, where they presented the clinical trial data.
T-20 is a new class of anti-HIV drugs called fusion inhibitors. Fusion inhibitors stop the HIV virus from entering and infecting a cell. All 17 other drugs currently used to treat HIV work by attacking enzymes the virus needs to function after it has infected cells.
Data from the most recent Phase III trial of T-20 showed the drug both reduced viral load and bolstered immune systems in patients already failing to respond to other medications.
Trimeris Chief Executive and co-founder, Dani Bolognesi pointed out that T-20, like all other current AIDS drugs, must be used in combination with other anti-HIV treatments to prevent the virus from developing resistance too quickly. He also said it’s plain that a cure is not on the horizon. “We’ll have to think in entirely different ways if we want to talk cure,” he said.
One of the reasons the Trimeris fusion inhibitors are eagerly awaited in the medical community, however, is that many AIDS victims who started the life-extending cocktails of multiple drugs in the 1990s have developed resistance to many if not all the currently available drugs. They are quickly exhausting their treatment options.
Second drug in pipeline
T-20 was tested on just such patients and proved quite effective, lowering the virus from 100,000 per milliliter of blood to under 5,000 per ml, doing better than expected. It reduced viral loads 83 percent more than in control group patients receiving other treatments. It doubled immune response in those getting T-20.
Early clinical studies showed that patients develop resistance to T-20 if given alone. The wily AIDS virus mutates so readily that over time it develops resistance to all current treatments. About 87 percent of AIDS patients are already resistant to one or more treatments. There are about 900,000 people with HIV infections in the U.S., with 360,000 getting anti-viral drugs.
Bolognesi said Trimeris’ second drug, T-1249, another fusion inhibitor, works even with patients who develop resistance to T-20. T-1249 is two or three years behind T-20 in the development cycle, but should appear in time to give AIDS patients yet another defense against resistant HIV, he said.
Poised for success
Trimeris is poised to become the Triangle’s first real homegrown public biotechnology success story. Founded in 1993 based on research at Duke University, Trimeris went public in 1996. It expects to start selling T-20 next year, splitting profits with development partner Hoffman LaRoche. Roche will manufacture T-20 in Boulder, Colorado.
Bolognesi noted that the first anti-HIV drug was developed by Burroughs Welcome based on Duke University science in the 1980s. He quoted the late Dr. David Barry, president of Triangle Pharmaceuticals, another local company developing anti-HIV treatments: “The Research Triangle is the epicenter of developing drugs against this disease.”
Bolognesi said during the press conference that T-20 is a peptide, a much larger molecule than most drugs. “It’s ten times the size of existing drugs,” he said. This has advantages and disadvantages.
Bolognesi said its size means it can’t actually enter cells, so it has an exemplary safety record and can’t interfere with other anti-AIDS drugs. Patients receiving T-20 developed only mild irritation where it was injected, causing less than 3 percent to drop out of the Phase III trial – what he called a very low number.
On the other hand, “No one has manufactured these peptides in this quantity before,” Bolognesi said. Roche plans to make three metric tons of the T-20 peptide, three times as much as anyone has made to date.
Billion dollar market capitalization?
Manufacturing difficulties are likely to make the drug expensive, at least initially. Analysts have estimated T-20 may cost patients as much as $12,000 a year when many have trouble paying for treatments costing much less now.
One local biotech entrepreneur, however, tells Local Tech Wire, “Roche will get it down to half that in a year.” Even at $6,000 a year, the drug would be expensive.
Analyst estimates of how much Trimeris and Roche will make from T-20 — they split U.S. profits — range from $120 to $138 million the first year.
Trimeris stock traded at $42.81 midday Wednesday, up $1.94 or nearly 5 percent. Some stock watchers are hoping the company will become the region’s first billion-dollar market cap public biotech company. It had a market cap of $802 million Wednesday.