For a company that is apparently leading the latest race to market a blood substitute, Biopure ( BPUR) doesn't look like much of a winner to investors these days. The Cambridge, Mass.-based company is the only one with a blood-substitute application pending before the Food and Drug Administration, yet its stock is down 85% since hitting a 52-week high Aug. 1. The company's chief executive, Thomas A. Moore, resigned in late February, after less than two years on the job. In late December, the Securities and Exchange Commission served Biopure with a Wells notice, revealing that the SEC staff had made a preliminary decision to recommend the agency bring a civil proceeding against the company.
The company's comments that a response could take 30 to 60 days evolved into comments that a response would be forthcoming in mid-June 2004. And now that projection is at risk. Last year "was certainly not the kind of year that we expected for Biopure," said Dr. Charles A. Sanders, the company's chairman, in a Feb. 24 letter to shareholders. Sanders recapped an assortment of disclosures that have dribbled out between October and February. Sanders said the FDA provided "an extensive list of questions," adding that Biopure is about halfway through a process in which 200,000 to 400,000 pages of data will be collected and tabulated for the FDA. He noted that in a January meeting between the FDA and Biopure, the agency asked "additional questions" about Hemopure's safety and efficacy "beyond those cited" in the regulator's letter last summer. "The FDA also indicated that new animal studies would need to be completed as part of our response," Sanders said. "Preparations to start three of these studies are under way, and a fourth study is under discussion." Because of these agency requests "and other information-gathering activities," Sanders added, Biopure may not be able to submit a formal written response to the FDA in June 2004 "as previously planned." A few weeks later, Biopure said it would conduct all four animal tests, adding that it may take the regulator six months to review the company's amended application for Hemopure. "It is currently unclear" whether the additional animal tests and the company's formal response "will be sufficient" to secure FDA approval, Biopure said in the March 16 filing of its quarterly 10-Q statement with the SEC. The agency might seek more information, or it might demand additional human clinical trials, Biopure said. "Therefore, the timetable for obtaining FDA approval for Hemopure remains uncertain."
Between the August announcement and the February stockholders' letter, investors also learned that the company in November withdrew a proposed clinical test plan for using Hemopure to treat hemorrhagic shock victims in a hospital setting. (Biopure's official application to the FDA is for Hemopure being used with acutely anemic patients undergoing elective orthopedic surgery.) Biopure had submitted the request in March 2003. The company recently revealed that the FDA had put a "clinical hold" on the test design "due to safety concerns" arising from the agency's review of late-stage clinical trials for Hemopure's use among orthopedic patients. Biopure said it responded to the FDA twice and that the agency reaffirmed its objections to the study design twice. But investors didn't find out about this issue until late December. Biopure didn't publicize the FDA's objections to the proposed study earlier "because the company does not consider correspondence with the agency about data interpretations in the development
of a study design to be material," Biopure said in its recent 10-Q statement.