Healthy Skepticism Library item: 7211

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Publication type: news

Japsen B.
Blood substitute `a disaster' in trial: Northfield still plans to seek FDA approval
Chicago Tribune 2006 Dec 20
http://www.chicagotribune.com/business/chi-0612200304dec20,0,7607612.story?coll=chi-business-hed

Abstract:

In a potential blow to the future of an Evanston-based biotech company, a clinical trial showed Tuesday that its key product, an experimental blood substitute, performed worse than standard treatments in patients who suffered traumatic injuries.

In a trial of 712 patients, Northfield Laboratories Inc. reported 13.2 percent of the 349 who had been given Northfield’s product died, compared with deaths of 9.6 percent in the control group of 363 who received either saline solution in the ambulance or blood in the hospital.

“No matter how they slice it, it’s a disaster,” said Martin Shkreli, a hedge-fund manager at New York-based Elea Capital, which invests in health-care companies and sold its Northfield shares earlier this year. “If you look at the absolute number of deaths, it kills more patients than saline does.”

In the trial, 46 in the group receiving the blood substitute Polyheme died, while 35 in the control group died, according to Northfield Laboratories.

Northfield has been working for more than 20 years on a potentially lucrative blood replacement that could save lives in trauma situations by carrying oxygen through the body, which the saline used during emergencies now cannot do. A blood substitute also would be easier to transport, especially to battlefields and accident scenes, and does not require the sometimes-complicated matching process real blood does.

Northfield’s product did not meet a predetermined goal worked out with the Food and Drug Administration more than two years ago.

Dr. Steven Gould, Northfield’s chief executive and chairman, said Tuesday that Polyheme the would have had to have performed “3.8 percent better” than the current standard treatment to show “superiority.”

Before Northfield made its announcement Tuesday afternoon, its shares fell more than 20 percent in Nasdaq trading. The stock continued to get pummeled by investors in after-hours trading, falling more than 50 percent, or $5.87 a share, to $5.65.

Gould cautioned that the study’s data needed to be “reanalyzed,” noting that Polyheme worked better in a smaller patient population of the trial that was free of “discrepancies” and “protocol violations.” He said he considered the latest results to be “preliminary.”

The last-stage trial of Polyheme drew controversy because it involved patients at accident scenes who could not consent to participating.

Gould said Northfield would move forward and planned to submit its product and the study data to the FDA, believing Polyheme still had benefits to patients and could win approval.

“Ultimately, it is their call,” Gould said of the FDA. “We think we are in the ballpark.”

Gould said the results were better from the portion of the patients that was free of protocol violations, a number he considered a “sizeable sample.”

“It is more than 80 percent of the study,” Gould said. “We can give our interpretation, and everybody is free to come to their own.”

A complicated trial

Gould hopes the FDA will review Northfield’s data carefully, considering that the trial was complicated and involved a complex group of patients who had suffered traumatic injuries that were treated by emergency workers under intense pressure at accident scenes or while being transported by ambulance.

“This was a logistically complex study with many variables and a high incidence of protocol violations,” Gould said. “We believe that there is an unmet medical need for a hemogloblin-based, oxygen-carrying red-blood-cell substitute, and that Polyheme is that product.”

No consent an issue

Progress has been slowed in developing a blood substitute.

Recently, an FDA panel decided against endorsing a clinical trial of Biopure Corp.‘s Hemopure blood substitute, the only other major contender in the market, because it was going to use the same disputed method of testing without the patient’s consent as Northfield did. The panel’s vote came despite pressure from the U.S. Navy, which had agreed to conduct the tests for Biopure on civilian trauma patients.

Industry analysts say the controversy over blood-substitute development makes Northfield’s attempt to gain approval more challenging.

“Clearly, I think the news is very negative for them,” said Eugene Trogan, a biotechnology industry analyst with Morgan Joseph & Co.

What’s more, Northfield has met with increasing criticism from consumer groups, influential members of Congress and medical ethicists, who said participants in the company’s trial were not given adequate notice or enough information about the Polyheme no-consent trial. Such criticism makes company observers wonder about the product’s future.

Further putting it in doubt is the perception that the FDA will make more rigorous reviews of drugs, particularly in the wake of the painkiller Vioxx, which was pulled from the market in 2004 after a study revealed it increased the risk of heart attack and stroke.

“If the FDA would even consider this, they would violate their mandate to protect public safety,” said Elea Capital’s Shkreli. “It would be shocking to see the FDA even consider this.”