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Hemispherx Biopharma, Inc.
FDA Advisory Committee Makes Recommendations
on Ampligen for Chronic Fatigue Syndrome
Philadelphia, PA, December 21, 2012: Hemispherx
Biopharma, Inc. (NYSE MKT: HEB) (the "Company" or "Hemispherx") today announced the outcome of the meeting
of the Arthritis Advisory Committee (the "AAC") of the U.S. Food and Drug Administration (the "FDA") on
the New Drug Application ("NDA") for Ampligen for Chronic Fatigue Syndrome ("CFS").
On the question: "Considering the
totality of the data, is there substantial evidence of efficacy for Ampligen for the treatment of patients with chronic fatigue
syndrome (CFS)?," the AAC voted 9 no, 4 yes and 1 AAC member left the room and did not vote.
On the question: "Has the safety
of Ampligen been adequately assessed and characterized for the treatment of chronic fatigue syndrome (CFS)?," the AAC voted
9 no, 4 yes and 1 AAC member left the room and did not vote.
On the question: "Is the safety profile
of Ampligen adequate for approval for the treatment of CFS?," the AAC voted 8 yes, 5 no and 1 non-vote.
On the question: "Based on the information
included in the briefing materials and presentations, has the applicant provided sufficient efficacy and safety data to support
marketing of Ampligen for the treatment of CFS?," the AAC voted 8 no, 5 yes and 1 non-vote.
Hemispherx is committed to the Ampligen
clinical development program and to bringing Ampligen to market to ease the suffering of those seriously afflicted with CFS.
The Company will also continue to work closely with the FDA to support the review of this investigational compound. Hemispherx
will seek to do everything necessary to confirm in a scientifically rigorous manner that Ampligen is a safe and effective
The AAC based its voting on a review of
data from the Ampligen clinical development program included as part of the Company's NDA submission. This submission
included data on nine studies conducted in patients with CFS, including two pivotal studies and seven supportive studies. The trials
were designed to evaluate safety, tolerability and efficacy in the approximately 845 patients (589 unique subjects suffering from
severely debilitating CFS) who received Ampligen . The majority of the AAC members expressed the view that an additional controlled
clinical trial of Ampligen should be conducted prior to the FDA granting approval.
The Prescription Drug User Fee Act ("PDUFA")
review goal for the FDA to complete its review of the NDA is February 2, 2013. The FDA uses committees, like the AAC, to obtain
independent expert advice on scientific, technical, and policy matters to assist in its mission to protect and promote public health.
The FDA is not bound by the AAC's recommendation, but will consider their recommendation in its review.
DISCLOSURE NOTICE: The information in this
press release includes certain "forward-looking" statements (explained below), including statements about the remaining
steps to potentially gain FDA approval of the Ampligen NDA for the treatment of Chronic Fatigue Syndrome. The final results
of these and other ongoing activities could vary materially from Hemispherx's expectations and could adversely affect the
chances for approval of the Ampligen NDA. These activities and the ultimate outcomes are subject to a variety of risks and
uncertainties, including but not limited to risks that (i) the FDA may follow the AAC's recommendation and decide not to
approve the Ampligen NDA; (ii) the FDA may ask for additional data, information or studies to be completed or provided prior
to approval; (iii) the FDA may require additional work related to the commercial manufacturing process to be completed prior to
approval or may, in the course of the inspection of manufacturing facilities, identify issues to be resolved; (iv) the FDA may
determine that the complete response submitted by Hemispherx is not "complete," potentially requiring the Company to
conduct additional activities before it can re-file, if at all, the complete response; and (v) until completion of the FDA review
of the Ampligen NDA and final approval of the product and prescribing information, if any, the specific patient population
for which Ampligen may be indicated will not be known. Any failure to satisfy the FDA's requirements could significantly
delay, or preclude outright, approval of the Ampligen NDA.
About Hemispherx Biopharma
Hemispherx Biopharma, Inc. is an advanced
specialty pharmaceutical company engaged in the manufacture and clinical development of new drug entities for treatment of seriously
debilitating disorders. Hemispherx's flagship products include Alferon N Injection (FDA approved for a category of sexually
transmitted diseases) and the experimental therapeutics Ampligen and Alferon LDO. Because both Ampligen and Alferon
LDO are experimental in nature, they are not designated safe and effective by a regulatory authority for general use and are legally
available only through clinical trials with the referenced disorders. Ampligen is an experimental RNA nucleic acid being developed
for globally important debilitating diseases and disorders of the immune system including Chronic Fatigue Syndrome. Hemispherx's
platform technology includes components for potential treatment of various severely debilitating and life threatening diseases.
Hemispherx has patents comprising its core intellectual property estate and a fully commercialized product (Alferon N Injection ).
The Company wholly owns and exclusively operates a GMP certified manufacturing facility in the United States for commercial products.
For more information please visit www.hemispherx.net.
Forward-Looking Statements
To the extent that statements in this press
release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe harbor provisions
of the Private Securities Litigation Reform Act of 1995. Words such as "believes," "plans," "anticipates,"
and similar expressions are intended to identify forward-looking statements. These statements are based on the company's
current beliefs and expectations and represent the Company's judgment as of the date of this release. The inclusion of forward-looking
statements should not be regarded as a representation by Hemispherx that any of its plans will be achieved. These forward-looking
statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties,
many of which are beyond Hemispherx's control, which could cause actual results to differ materially from those contemplated
in these forward-looking statements. Examples of such risks and uncertainties include those set forth in the Disclosure Notice,
above, as well as the risks described in Hemispherx's filings with the Securities and Exchange Commission, including the
most recent reports on Forms 10-K, 10-Q and 8-K. You are cautioned not to place undue reliance on these forward-looking statements,
which speak only as of the date hereof, and Hemispherx undertakes no obligation to update or revise the information contained in
this press release, whether as a result of new information, future events or circumstances or otherwise revise or update this release
to reflect events or circumstances after the date hereof.