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Hemispherx Biopharma, Inc.
Hemispherx Biopharma Receives Complete
Response Letter from FDA on
Ampligen New Drug Application
for Chronic Fatigue Syndrome
Hemispherx will seek an End-of-Review
Conference with the FDA
Philadelphia, PA - February 4,
2013 - Hemispherx Biopharma, Inc. (NYSE MKT: HEB) (the "Company" or "Hemispherx"), announced
that it received a Complete Response Letter from the US Food and Drug Administration ("FDA") declining to approve its
new drug application ("NDA") for Ampligen for Chronic Fatigue Syndrome ("CFS").
The FDA said Hemispherx should conduct at least one additional clinical trial, complete various
nonclinical studies and perform a number of data analyses.
In its Complete Response Letter ("CRL"),
the FDA set forth the reasons for this action and provided recommendations to address certain of the outstanding issues. The Agency
stated that the submitted data do not provide substantial evidence of efficacy of Ampligen for the treatment of
CFS and that the data do not provide sufficient information to determine whether the product is safe for use in CFS due to the
limited size of the safety database and multiple discrepancies within the submitted data.
In the two pivotal clinical studies
that form the basis of approval for Ampligen , Hemispherx believes that the primary efficacy endpoints were met
and that they showed a statistically significant improvement (i.e., with a p-value of 0.05 or less). The FDA and Hemispherx do
agree that in clinical study AMP-502, the primary endpoint was met (p=0.02). In clinical study AMP-516, the FDA's analysis
resulted in a p-value of 0.10, while Hemispherx's calculation resulted in a p-value of <0.05, and yet both analyses indicate
that those patients on Ampligen improved over those on placebo. With regard to safety, Hemispherx has provided
data from the 845 subjects who have received Ampligen , including 589 subjects suffering from severe CFS and over
200 CFS patients who have received Ampligen for at least one year or longer. The Company believes that these data
are sufficient to determine the safety profile of Ampligen . At the December 20, 2012 FDA Advisory Committee meeting,
8 of the 13 Advisory Committee members voted yes on the question of "Is the safety profile of Ampligen adequate
for approval for the treatment of CFS?"
Hemispherx plans to request an end-of-review
conference with the FDA as a precursor to submitting a formal appeal to the Office of New Drugs in the FDA's Center for Drug
Evaluation and Research regarding the Agency's decision. The purpose of the conference is to review all of the issues raised in
the Agency's CRL as well as to discuss the corroborating data and experiences of clinicians and patients who have seen the
benefits of Ampligen therapy.
Hemispherx has become aware that a prominent
CFS advocate and long-time CFS sufferer, who has been on Ampligen since 1999 through a treatment IND, began a hunger
strike on January 30, 2013 to seek FDA approval of Ampligen . Hemispherx understands the frustration that there
is still no FDA-approved treatment for CFS and the concern that patients may lose access to Ampligen therapy. Out
of concern for the health of the CFS community, Hemispherx has asked any hunger strikes be discontinued and that patients join
in a collaborative effort between the FDA, Hemispherx, CFS clinicians and patient advocates to find a solution to this significant
In the past, the FDA has shown great willingness
to work with stakeholders to find solutions for serious and life-threatening illnesses. Dr. Margaret
Hamburg, Commissioner of the FDA has previously stated that, "FDA has an important
role to play in shaping the future of medical breakthroughs by bringing stakeholders together to identify and overcome challenges."
Hemispherx hopes that the FDA will view the Ampligen end-of-review conference as an opportunity to involve patient
advocacy, clinicians and researchers in a concentrated effort to do something for these patients over the near-term, including
further evaluation of how new legislation, such as the recently enacted "FDASIA" statute, may have a role in finding
a solution. The views of one internationally recognized researcher/clinician, Dr. Nancy Klimas, who has over 20 years' experience
evaluating and treating CFS patients, can be found at http://www.sciencedaily.com/releases/2013/01/130124183448.htm
NOTICE: The information in this press release and the article referenced therein includes certain "forward-looking"
statements (explained below), including statements about the remaining steps, including the aforementioned end-of-review conference
and appeals process, which the FDA may require and Hemispherx may take in further seeking 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. Any failure to satisfy the FDA's requirements could significantly delay, or preclude outright, approval of the Ampligen
About Hemispherx Biopharma
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
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, including its intent to pursue the end-of-review conference and appeals process.
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 and Hemispherx's beliefs that the Ampligen
NDA may be covered by the new provisions of the FDASIA statute, which are subject to FDA interpretation and implementation, or
that such provisions, if applicable, will be helpful with regard to obtaining FDA approval of the Ampligen
NDA. 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.