Wednesday, December 2, 2009

FDA did not approve Ampligen

Source: The Street
Date:   1 december 2009
Author: Adam Feuerstein
Ref:    Hemispherx Press release,

Hemispherx's Ampligen dealt FDA blow

PHILADELPHIA (TheStreet) -- Hemispherx BioPharma(HEB Quote) said late
Tuesday night that the Food and Drug Administration refused to approve
the experimental drug Ampligen for use in patients with chronic
fatigue syndrome.

The regulatory agency's rejection of Ampligen is a staggering blow to
Hemispherx, which has pursued the drug's development in a dizzying
array of diseases for more than 20 years with no success.

  From Wall Street, the bad news for Hemispherx was a decisive victory
for bearish investors and company critics (including myself, on a
personal note) who've long contended that Ampligen would never be

Hemipsherx shares plunged 43% to 68 cents in Tuesday's after-hours
trading session.

The FDA's complete response letter to Hemispherx -- summarized in
the company's Tuesday night press release -- essentially instructs
Hemispherx to start Ampligen's clinical trial program from scratch.

The agency's medical reviewers concluded that the two clinical
studies of Ampligen submitted by Hemispherx "did not provide credible
evidence of efficacy," according to the company.

In order to reconsider Ampligen for review, FDA instructed
Hemispherx to conduct at least one additional clinical study in
chronic fatigue syndrome. The study needs to test different doses of
Ampligen for a minimum of six months, including at least 300 patients
on Ampligen dose regimens intended for marketing, according to
Hemispherx's summation of the FDA's letter.

Hemispherx could find meeting the FDA's demands exceedingly difficult
and expensive. If the FDA requires 300 patients treated with Ampligen
in a new pivotal study, for example, Hemispherx would probably have
to enroll 450 total patients at a minimum (assuming two patients
treated with Ampligen for every one patient treated with a placebo to
act as a control.)

By comparison, the Ampligen phase III study submitted and rejected by
FDA enrolled just 230 patients total and took six years to complete.

But the FDA is asking for even more from Hemispherx, including tests
of Ampligen in rodents to rule out the risk of cancer and a safety
study in humans to ensure that Ampligen doesn't cause dangerous
changes to a patient's heart rhythm. Ampligen's manufacturing problems,
previously flagged by FDA inspectors, also remain unresolved, the
company said Tuesday.

Hemispherx tried in vain to put the best face on the Ampligen rejection.

"Management is pleased to have received specific advice on the remaining
issues and is looking forward to making a thorough but expedited
response its top priority, and plans to take all appropriate steps to
seek approval and commercialization of Ampligen," the company said, in
its statement.

Conspicuously absent were any direct quotes or comments from Hemispherx
CEO Bill Carter. In the past, Carter has excoriated critics of the
company for not understanding the science behind Ampligen and calling
into question the drug's efficacy. At other times, he's suggested that
staffing shortages at the FDA were the cause for a delay in Ampligen's
approval and insisted that 80% of drugs that reach the FDA are approved
(implying somehow that this boded well for Ampligen's approval.)

Ampligen in its current form has been around since the late 1980s,
touted for its antiviral and immune system-boosting properties. At
various times, Hemispherx has promoted and tested Ampligen as a treatment
for smallpox, HIV, ebola, avian flu and most recently, swine flu, or the
H1N1 virus.

The company's biggest push has been in chronic fatigue syndrome, a
poorly understood disease with no known cause that starts with flu-like
symptoms and progresses to chronic weakness and fatigue (hence the
disease's moniker.)

Hemispherx submitted Ampligen to the FDA for approval in October 2007.
An approval decision date of Feb. 25, 2009, was extended to May 25,
2009. But that date came and went with no word from the FDA, although
Hemispherx claimed the agency requested "one or two more weeks" to
make a decision. That short 14-day delay eventually stretched for seven
months, culminating in Tuesday's announcement.

(c) 2009 The Street

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