Biopure Says Hemopure Is Safe, but Holds On to Data

Biopure(BPUR Quote) said Monday that a statistical analysis of its human-blood substitute proves the experimental product is safe, but the biotech company refused to release any data to support its claim.

The company's announcement, in the form of a press release, comes two weeks after TheStreet.com uncovered mounting evidence that Hemopure, its oxygen-carrying red blood cell substitute, was unsafe and that Biopure executives appear to be scrambling for ways to portray the product in a more positive light.

On Monday, the company said that safety trials for Hemopure met its primary endpoint, which was to prove that "patients treated with Hemopure have outcomes no worse than patients treated with allogenic red blood cells per statistical methodology previously agreed to by the [Food and Drug Administration]."

Commenting on the results, Biopure CEO Carl Rausch said, in a statement, that "these results are a win for the company because they meet the study endpoints previously agreed to by the Food and Drug Administration and are supportive of our plan to file a marketing application later this year."

Shares of Biopure soared on the news, jumping $4.63, or 23%, to $24.53.

Today's announcement from Biopure does little to shed anything new on the safety of the company's blood substitute. While releasing the apparent results of a statistical analysis of Hemopure's safety, the company refused to release any real data that would bolster its claims. This would include, most significantly, the types and frequency of negative side effects.

The company also did not hold a telephone conference call with investors and analysts to discuss results, as is the custom for biotech companies with significant, market-moving news.

TheStreet.com has spoken to two doctors who have firsthand knowledge of patients experiencing temporary kidney failure after being administered Hemopure. TheStreet has also talked to other doctors who are aware of additional cases of kidney failure and problems with elevated blood pressure, after talking to doctors who treated these patients.

One of these doctors with first-hand knowledge of the product said today's announcement from Biopure does not change his view that Hemopure is unsafe and stands little chance of gaining FDA approval. He says the method of statistical analysis used by Biopure easily obscures the significance of major side effects with Hemopure, allowing the company to claim the product met its statistical safety goals.

But when the FDA takes the covers off the study results and looks at individual patients and their experiences using Hemopure, they will reach a different conclusion, the doctor said.

Biopure executives would not return phone calls seeking comment, but in the past they have steadfastly refused to acknowledge any side effects in Hemopure patients. Today, CEO Rausch, speaking to Reuters news service, acknowledged that some patients have suffered from temporary kidney failure and elevated blood pressure.

"What we can say is that the incidence of these events was low in both groups," Rausch told Reuters. "There is no statistical significance to these events that we know of."

Biopure is basing its results on a study of 693 patients, half receiving Hemopure and half receiving red blood cells. The study size, and the way it was designed, was preapproved by the FDA, the company says.

But in recent weeks, it's become clear that U.S. drug regulators are taking a harder look at blood substitutes. In the past two months, the FDA has ordered Hemosol (HMSL Quote), a Biopure competitor, to increase the number of patients in its blood substitute test from 700 patients to 900 patients in order to better gauge the product's safety. More recently, the FDA asked Hemosol to make even more changes to the design of its tests, which has forced the company to significantly delay development.

And just because a trial design was preapproved doesn't mean Biopure or any other company is in the clear with the FDA. In July, the FDA rejected an experimental asthma drug from Genentech and Novartis on safety concerns, even though the companies and regulators had previously agreed on a safety threshold. In essence, the FDA changed its mind and didn't tell the companies.

So Monday, Biopure tried to put the question of Hemopure's safety to rest. Instead, the company's announcement just raises more questions. The firm said further data would be made public at "appropriate medical forums," but as of two weeks ago, the company was not scheduled to appear at any medical or scientific conferences this fall. Biopure is scheduled to give a presentation at one investment banking conference in October.

With the company's reluctance to provide concrete proof of Hemopure's safety, the issue may not be resolved until the FDA conducts its own examination. If Biopure submits an approval application for submission later this year, investors may have to wait until well into next year before all the questions are answered.