AtheroGenics Inc.'s experimental heart drug missed the primary goal of a pivotal late-stage trial but cut death, heart attack and stroke and dramatically reduced the risk patients would develop diabetes, researchers said Tuesday.
The anti-oxidant, anti-inflammatory drug, the first of its kind, reduced the risk of developing diabetes by 64 percent and demonstrated a small but statistically significant reduction in blood sugar after 12 months, researchers said.
Data from the 6,144-patient study were presented at the American College of Cardiology scientific meeting in New Orleans and gave a brief boost to AtheroGenics shares.
Earlier this month the stock fell 60 percent when the company said the drug, known as AGI-1067, had missed its composite primary goal of reducing cardiac death, heart attack, stroke, need for revascularization procedures and unstable angina, or chest pain, by 20 percent.
Because the drug failed to succeed in its primary goal but showed promise on secondary measures, doctors and researchers called for further large studies, which could significantly delay the medicine's time to approval.
"We don't think this was a chance finding. We just need to confirm it in a large clinical trial," Dr Jean-Claude Tardif, the study's co-lead investigator, said at a news conference.
"I think this drug has a chance to change cardiovascular medicine," he added.
The impressive diabetes results may come as a surprise to investors who have abandoned AtheroGenics or who have been betting the drug will fail.
Its shares climbed as much as 26 percent in early Tuesday trading before surrendering those gains.
PART OF THE GOAL
When the last two measures in the primary composite goal were excluded, the drug reduced death, heart attack and stroke by a statistically significant 19 percent over a placebo, researchers said.
While those were considered secondary goals, or end points, they are likely to receive attention from heart doctors and investors in the volatile biotechnology stock.
"The death, heart attack and stroke end point is actually more important, because those are the worst events," said Dr. Christopher Cannon, a cardiologist at Brigham and Women's Hospital in Boston.
"We probably need some type of confirmatory trial before one could adopt that therapy as an approach, but it certainly keeps it alive as a strategy," he said.
Needham analyst Mark Monane agreed that further testing will probably be required.
"There were definitely some encouraging findings," Monane said. "But trials are generally judged on the primary end point, and they missed on that."
AstraZeneca Plc, seeking to rebuild its depleted developmental drug pipeline, has agreed to pay up to $1 billion for exclusive rights to the drug. But the London-based drugmaker can still opt out of the deal.
"We are working through the statistical analysis at the moment, and when we've got through that, then we will start the 45-day period of deciding whether we will continue with the compound," an AstraZeneca spokeswoman said.
Heart patients in the study received either 300 milligrams of AGI-1067 or a placebo on top of a host of standard-of-care medicines they were already taking, such as aspirin, cholesterol-lowering statins, blood thinners and/or diabetes medicines.
The drug had an undesirable impact on blood fats, raising bad LDL cholesterol by about 12 percent and lowering good HDL cholesterol by roughly the same amount. There were also some potentially troubling safety signals with a trend toward more heart failure in those taking the drug.
But the death and heart attack outcomes and diabetes effect may be enough to keep it on track to eventual approval.
"In diabetes there was a profound effect, and that needs to be followed," said Dr. Marc Pfeffer, a co-principal investigator of the study. "I'm pretty bullish that we need to go to the next step."
AtheroGenics shares were unchanged at $3.58 in afternoon trading on Nasdaq after climbing as high as $4.50 earlier. (Additional reporting by Ben Hirschler in London)
