Hemispherx Chronic Fatigue Drug Scrutinized Ahead of Panel Review

by Brett Chase, Minyanville staff writer

Shares of drug developer Hemispherx BioPharma HEB are falling Tuesday after a government report questioned whether the company’s treatment for chronic fatigue syndrome works. The stock fell by almost half after previously rising more than 200% this year.

Philadelphia-based Hemispherx is trying to win US approval for its drug Ampligen. In a report, government reviewers not only question whether the drug is effective, but they also point out that the company was warned earlier this year that its drug research data may not support approval.

“It would be unusual for this type of data to provide adequate evidence of efficacy. However, the adequacy of the data will ultimately be a review issue,” Food and Drug Administration officials told company representatives in June. That’s according to this report released today.

Hemispherx faces a panel of expert advisers to the FDA on Thursday. The advisers will hear arguments from the company on why the experimental drug Ampligen should be approved for sale in the US. The FDA will consider the advisers’ recommendations as it decides whether to approve the medicine. The agency is expected to decide by February 2.

In a review of the drug posted online, staff for the FDA questioned a pair of studies of patients conducted to show Ampligen’s effectiveness. “Signals of efficacy are inconsistent between the two trials,” staff reviewers wrote.

Throughout the report, the descriptions for the study were more blunt. A methodology used in one trial was “ill-defined and invalid.”  “Limited conclusions” about safety and effectiveness can be drawn from data, FDA staffers said. (See the meeting briefing documents here.)

FDA staff reviewers raised a number of issues related to the research of the drug, including study design and execution, patient selection for the trials, and analysis of the data. What’s more, the company is relying on research conducted before the FDA first rejected the drug in 2009. There are also questions about the safety of the drug, including potential liver damage. As these FDA staff reviews go, the report was scathing.

Hemispherx officials declined to comment, citing the upcoming FDA panel. “It is not appropriate for companies to speak to the press prior to an advisory committee,” a spokeswoman says.

Shares of the company fell 45% to $.36 midday Tuesday. Public since 1996, the stock traded above $17 per share in early 2000. It traded as high as $1 in September.

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