Can Hemispherx's Ampligen See Happy Ending To Long Fatiguing Wait?


In less than a month, Hemispherx Biopharma Inc. (HEB) will face an FDA advisory panel that will review its lead compound Ampligen for the proposed treatment of chronic fatigue syndrome or CFS.

Ampligen, co-invented by Hemispherx's CEO William Carter, has been under development since the 1970s.

The new drug application for Ampligen as a treatment for chronic fatigue syndrome was filed by the company in October 2007 based on results from four well-controlled trials with over 1,200 trial subjects and 90,000 doses. But the NDA was deemed to be insufficient to permit a review and was sent back to the drawing board.

Addressing all the questions posed by the FDA concerning the Ampligen NDA, the company filed its response in January of 2008. Convinced by the company's reply, in July 2008, the regulatory agency accepted for review the new drug application for Ampligen to treat CFS and was expected to announce its decision by February 25, 2009.

Seeking additional time for a full review of the submission, the FDA postponed the decision date by 3 months to May 25, 2009. However, the decision was again deferred, and a complete response letter for Ampligen NDA was issued in November 2009, requesting additional confirmatory phase III study to support its approval.

Supported by supplemental clinical data analyses, in August of this year, Hemispherx submitted its response to the Complete Response Letter for Ampligen NDA. The resubmitted NDA has a decision date set for February 2, 2013.

Before the FDA makes its final decision, the Ampligen NDA is scheduled to be reviewed by an FDA panel of outside experts on December 20, 2012.

Chronic fatigue syndrome is a condition in which patients have persistent or relapsing fatigue. There is neither a known cause, nor an effective treatment for CFS.

Besides CFS, Ampligen has also been tested in clinical trials as a potential treatment of Hepatitis B, HIV and various cancers, earning it the sobriquet "a drug in search of a disease. Carter also had plans to treat tobacco with Ampligen and create a "healthy" cigarette.

The company has one FDA-approved product Alferon N Injection for refractory or recurring genital warts. Commercial sales of Alferon N Injection were halted in March 2008 when the company's finished goods inventory expired. In a recent SEC filing the company has revealed that the production of new Alferon drug product inventory will not commence until the capital improvements and related validation phases at its New Brunswick manufacturing facility are complete.

A quick look at Hemispherx's balance sheet...

In the third quarter ended September 30, 2012, the company's net loss widened to $6.42 million or $0.05 per share from $3.27 million or $0.02 per share in the year-ago quarter.

Revenue, which was solely derived from clinical trial programs of Ampligen, dropped 13% to $39 thousand in Q3, 2012 from $45 thousand in the comparable year-ago quarter. Note that the company had no Alferon N Injection Finished Good product to commercially sell, as of September 30, 2012.

Hemispherx ended Q3, 2012 with $35.4 million in cash.

Pacing the sidelines for nearly four decades, Ampligen has been a lightning rod for controversy. Will Ampligen be successful in passing regulatory muster at least this time? Stay tuned...

Shares of Hemispherx have thus far hit a 52-week low of $0.17 and a 52-week high of $1.10. The stock closed Friday's trading at $0.75, up 1.52% on a volume of over 551 thousand shares.

Related Reading

For a comprehensive look at Ampligen's early history, read Mindy Kitei's 1994 article from Philadelphia magazine, "The AIDS Drug No One Can Have".

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