SAN DIEGO -- Biotechnology company
says partial test results of its blood substitute Hemopure unveiled Sunday show the experimental product to be effective and safe, but the data leave a number of questions unanswered.
Biopure is trying to prove that surgery patients using the oxygen-carrying Hemopure can safely avoid the need for red blood cell transfusions. The results, released Sunday in San Diego at the annual medical meeting of the Society of Critical Care Medicine, seem to bolster Biopure's case.
But don't count on approval from the Food and Drug Administration just yet. The results released Sunday came from just 35 patients, or 5%, of the 688 elective surgery patients enrolled in a pivotal late-stage test of Hemopure. The test results also were incomplete -- key facts were missing from the presentation, which are crucial to making any conclusions about the efficacy and safety of the product.
Shares of Biopure have fallen 41% since December, when the company said it was being forced to delay by up to six months the filing of an FDA approval application for Hemopure. The selloff intensified in mid-January after Biopure disclosed that several executives and insiders, most notably CEO Carl Rausch, were setting up preprogrammed trading plans to sell portions of their holdings. Biopure closed Friday at $10.29 per share -- a week in which the stock hit its 52-week intraday low of $8.75 per share.
On Sunday, Dr. Jonathan Jahr was at the Society of Critical Care meeting to give a poster presentation on results from 35 patients he enrolled as part of the Hemopure pivotal clinical trial. Jahr, an anesthesiologist, conducted his research on Hemopure in 1999 while on staff at the University of California, Davis, medical school. He has since moved to the University of California, Los Angeles.
Of the 35 orthopedic surgery patients enrolled by Jahr in the study, 19 received Hemopure and 16 received red blood cells. Results showed that nine Hemopure-treated patients, or 47%, avoided the need for red blood cells at 42 days after surgery -- surpassing the clinical goal of the study of 35%.
Patients tolerated Hemopure well from a safety standpoint, according to Jahr's findings. The blood pressure in Hemopure-treated patients was slightly higher than patients in the control group, but the effects were temporary, according to Jahr's findings.
Biopure has long been criticized for withholding the scientific proof to back up its claims about Hemopure, and while Jahr reported positive results Sunday, they won't do much to assuage critics because so much information is missing.
For starters, the clinical trial in which Jahr participated was completed in August 2000. Yet 18 months later, the only data Biopure has made available from this all-important study are from 5% of the patients, all enrolled at one clinical trial site. It's highly unusual for even large, complex clinical trials to take more than six months to analyze and report results.
Asked to explain the delay in releasing all the Hemopure data for review, Jahr says the trial was extremely complicated, which has made it difficult for Biopure to compile and analyze all the information.
Biopure spokesman Doug Sayles, reached by phone and asked why only 5% of the Hemopure data were presented Sunday, said the information from Jahr's patients was all that was ready in time for presentation at the Society of Critical Care meeting. "We'll be presenting more data in March at an anesthesiology meeting," he added. He declined to answer any other questions.
Jahr's findings also appear to be incomplete because they lack critical information about patients who were given red blood cells during the trial -- in other words, the control arm of the randomized, controlled study. These data are important because it's a straightforward way to directly compare the oxygen-carrying capacity of Hemopure vs. red blood cells.
The FDA is not likely to approve Hemopure unless Biopure can prove that the product is as effective and as safe as blood. The regulatory agency would need information about the control arm of the study to pass judgment, but so far, Biopure and Jahr are keeping those data to themselves.
Why? Jahr said that the trial's goal was to show the number of Hemopure-treated patients who avoided the need for red blood cells; therefore, those are the only results being reported.
And while Jahr says his study found Hemopure to be as safe as blood, there were 32 "adverse events" listed for Hemopure-treated patients, including most surprisingly, hypoxia, or oxygen deprivation. By comparison, the list of adverse events in the red blood cell group totaled 15, with nary a mention of hypoxia.
Asked to step away from the minutiae of the data and give his overall opinion on Hemopure, Jahr says he believes the product, if approved, will ultimately be used as an "oxygen bridge" -- meaning it will be given to oxygen-starved patients in situations where blood is either unavailable or can't be used. The most obvious situations are trauma helicopters or in ambulances, where blood, because it's perishable, is not used.
But the Hemopure study in which Jahr was involved doesn't involve trauma patients, only patients undergoing elective surgery. Do his data support this kind of trauma use?
"Absolutely not," he says. "For that kind of use, more testing would be required."