Controversy surrounds failed test drug results

Independent researcher faces off with major drug companies over timeliness of failed experimental heart drug findings.

By Aaron Smith, staff writer

CHICAGO ( -- Unexpected controversy at the American Heart Association conference over test results of a failed experimental drug pits an independent researcher against a massive drug company and its biotech partner.

The experimental heart disease drug, a joint venture between Procter & Gamble and Alexion Pharmaceuticals failed to improve the survivability of heart attack patients, announced the lead researcher of the company funded study, Dr. Paul Armstrong, a cardiologist at University Hospital of Alberta in Edmonton Canada, on Tuesday.


But representatives from the companies that financed the study - P&G (Charts) and the biotech Alexion Pharmaceuticals (Charts) - downplayed Dr. Armstrong's announcement as old news, noting that they announced this study months ago and promptly gave up on the drug.

Alexion's stock price dropped 3 percent in morning trading on the news. "We were scratching our heads on what was going on with the stock," said Eric Schmidt, analyst for Cohen & Co., who owns shares of Alexion stock. "Pexelizumab is a zero for the company. But on Wall Street, we've all written that off long ago. [The companies] terminated that drug a long time ago."

Separately, Alexion announced Tuesday that the Food and Drug Administration accepted the application for its experimental drug Soliris for the treatment of a genetic blood disorder, and the agency granted it "fast track" review status. That means that the FDA intends to make a decision within six months, compared to the normal review process, which for most drugs takes more than a year.

Soliris is also being reviewed by European regulators, who also granted Soliris an accelerated review process, said Alexion.

Soliris is being reviewed as the first specific treatment for a disorder where the body's own immune system destroys red blood cells, said Alexion. According to the National Institutes of Health, the disorder is inherited, often resulting in fatal blood clots.

The disorder affects up to 10,000 people in North American and Europe, and the FDA has given the experimental drug "orphan drug" status, said the biotech.

The late-stage study involved 5,745 patients who had suffered heart attacks and undergone angioplasty to reopen their blocked arteries, with the intention of helping them survive the first 30 days after the procedure. But those who took pexelizumab, the drug in question, showed almost identical mortality results as those who took a placebo, with about 4 percent of patients dying during that 30-day period in both groups, said Dr. Armstrong, who made his announcement at the annual conference of the AHA.

"The result was not what we anticipated," said Dr. Armstrong, medical professor and cardiovascular research director at the University of Alberta. "This was a striking and important surprise for us."

The mortality results between those patients who took the experimental drug and those who did not were "almost dead even at 30 days," said Dr. Armstrong, with 3.92 percent of the placebo patients dying in that time period, compared to a 4.06 percent mortality rate among pexelizumab patients.

Tom Millikin, spokesman for P&G, and Tom Dubin and Scott Rollins, senior vice presidents for Alexion, told that the study results are old news, having been announced on June 28, when Alexion reported, as required by the Securities and Exchange Commission, that pexelizumab had failed the study.

Millikin emailed a press release from the P&G partner Alexion dated June 28, 2006. In that press release, Alexion said that "preliminary" results in the late-stage study of more than 5,700 patients showed that pexelizumab "did not achieve a statistically significant reduction in mortality at 30 days" following the angioplasty procedure.

"Of most relevance to sponsors and regulators is the primary clinical endpoint, which was announced in June," said Millikin to

But Dr. Armstrong told that the exact mortality results - of 3.92 percent for placebo versus 4.06 percent for pexelizumab - had not been reported prior to Tuesday morning.

"The results are brand new," said Dr. Armstrong. He said that the companies reported on June 28 that the trial had failed, but the exact results were not known until later.

Dr. Armstrong's statement was reiterated by Dr. Timothy Gardner, chairman of the AHA committee that runs the scientific sessions, the forum where clinical data is reported.

"All [the companies] did was report that their clinical trial failed to meet its primary endpoint," said Dr. Gardner, referring to the endpoint that is used to gauge whether an experiment is successful. "They did not disclose any information about the trial."

Dr. Armstrong and two co-authors in the study received research grants from P&G and Alexion. But Dr. Armstrong is considered an independent investigator, so he is not beholden to these companies. Clinical trials of experimental drugs are typically conducted by independent investigators, to achieve objective results.

Getting to the hospital quickly

A completely separate study that had nothing to do with the pexelizumab drug announced at the AHA showed that balloon angioplasty, a surgical procedure to reopen clogged arteries following a heart attack, followed by the use of stents, tubes used to prop open those arteries, failed to reduce major heart complications for patients who wait at least three days before undergoing the procedure.

Dr. Judith Hochman, the lead researcher, said the study of more than 2,000 heart attack patients demonstrated the importance of getting to a hospital within 12 hours after a heart attack to reopen arteries. She said some patients benefited from the procedure up to 36 hours following a heart attack.

"Get to the hospital early, because early opening of blocked arteries saves lives," said Dr. Hochman, director of the Cardiovascular Clinical Research Center at the New York University School of Medicine. She described the study results as "unexpected."

"It was logical than an open artery would be better, but logic doesn't always dominate, because the human body is a complicated organism," said Dr. Hochman, who co-authored the study with 17 other researchers.

This study used stents and other materials donated by Cordis Corp., Johnson & Johnson (Charts), Eli Lilly & Co. (Charts), Guidant Corp. (Charts), Medtronic, Merck & Co. (Charts), Boston Scientific, Millennium Pharmaceuticals, Inc. and Schering-Plough Corp. The study focused on the timeliness of treatment, rather than the quality of the stents.

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