Company expects to file New Drug Application
QUÉBEC CITY, July 26, 2011
/PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") today announced completion of its Phase 3 study
with AEZS-130 as the first oral diagnostic test for Adult Growth
Hormone Deficiency (AGHD). The Company is currently proceeding with
detailed analyses of the data and preparing for a pre-New Drug
Application (NDA) meeting with the U.S. Food and Drug
Administration (FDA) in the upcoming months, which would be
followed by the filing of a NDA for the registration of AEZS-130 in
the United States.
"We wish to thank all those involved in this project for their
utmost dedication. Preliminary results are encouraging and we
therefore expect to submit a NDA to the FDA. We believe that
AEZS-130 could become the first approved oral test for the
diagnosis of AGHD, providing patients with a potentially safer,
accurate and more convenient alternative to the current injectable
tests", stated Juergen Engel, Ph.D.,
President and CEO at Aeterna Zentaris.
Study History
The study titled, "A Multi-Center Study Investigating a New,
Oral Growth Hormone Secretagogue (AEZS-130, formerly ARD-07) as a
Growth Hormone (GH) Stimulation Test in Terms of Safety and
Efficacy", was originally initiated to compare the performance
of AEZS-130 against the then-available diagnostic growth
hormone-releasing hormone (GHRH) Geref Diagnostic® +
Arginine (ARG) standard test. Geref Diagnostic® was
subsequently withdrawn from the market, worldwide, in 2008; the
trial's sponsor, Ardana Biosciences (Ardana), discontinued the
study for financial reasons before it was completed. In 2009,
Aeterna Zentaris entered into an agreement with administrators of
Ardana and regained the rights to AEZS-130, and with the FDA,
established the best way forward to complete this Phase 3 study and
continue to utilize the data already obtained, in light of the loss
of the original comparator. A Special Protocol Assessment (SPA)
granted by the FDA, resulted in a modification of the original
study, without altering the basic study design so that the
completed portion of the study and the new part of the study would
provide one, complete, Phase 3 study.
Study Design
The first part of the study conducted by Ardana was a two-way
crossover study involving 42 patients with confirmed AGHD or
multiple pituitary hormone deficiencies and a low insulin-like
growth factor-I. A control group of 10 subjects without AGHD were
matched to patients for age, gender, body mass index and (for
females) estrogen status.
Each patient received two dosing regimens in random order, while
fasting, at least 1 week apart. One regimen consisted of a 1 µg/kg
(max. 100 µg) dose of GHRH (Geref Diagnostic®, Serono)
with 30 g of ARG (Ar-Gine®, Pfizer) administered
intravenously over 30 minutes; the other regimen was a dose of 0.5
mg/kg body weight of AEZS-130 given in an oral solution of 0.5
mg/ml.
As a result of the SPA reached with the FDA in order to complete
the trial, the second part of the study contained the following
revisions/additions to the first protocol:
- An additional 30 normal control subjects were enrolled to match
the AGHD patients from the original cohort;
- Further, an additional 20 subjects were enrolled - 10 AGHD
patients and 10 matched normal control subjects;
- The above brought the database to 100 subjects;
- All subjects received a dose of 0.5 mg/kg body weight of
AEZS-130;
- As a secondary endpoint, the protocol required that at least 8
of the 10 newly enrolled AGHD patients be correctly classified by a
pre-specified peak GH threshold level.
About AEZS-130
AEZS-130, a ghrelin agonist, is a novel orally active small
molecule that stimulates the secretion of growth hormone. The
Company has completed a Phase 3 trial for use as a simple oral
diagnostic test for adult growth hormone deficiency (AGHD).
AEZS-130 works by stimulating a patient's growth hormone secretion
after which a healthcare provider will measure how well the body
responds to that stimulation based on the patient's growth hormone
levels over a period of time. Low growth hormone levels, despite
giving an effective stimulating agent, confirm a diagnosis of AGHD.
AEZS-130 has been granted orphan-drug designation by the FDA for
use as a diagnostic test for growth hormone deficiency. Aeterna
Zentaris owns the worldwide rights to AEZS-130 which, if approved,
would become the first orally administered diagnostic test for
AGHD.
About AGHD
AGHD affects 35,000 adult Americans, with 6,000 new adult
patients diagnosed each year. Growth hormone not only plays an
important role in growth from childhood to adulthood, but helps
promote good health throughout life. AGHD is usually characterized
by low energy levels, decreased strength and exercise tolerance,
increased weight or difficulty losing weight, emotional changes,
anxiety and impaired sleep. Available diagnostic tests for AGHD are
complex and can produce significant side effects.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a late-stage oncology drug development
company currently investigating potential treatments for various
cancers including colorectal, multiple myeloma, endometrial,
ovarian, prostate and bladder cancer. The Company's innovative
approach of "personalized medicine" means tailoring treatments to a
patient's specific condition and to unmet medical needs. Aeterna
Zentaris' deep pipeline is drawn from its proprietary discovery
unit providing the Company with constant and long-term access to
state-of-the-art therapeutic options. For more information please
visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made
pursuant to the safe harbour provisions of the U.S. Securities
Litigation Reform Act of 1995. Forward-looking statements involve
known and unknown risks and uncertainties that could cause the
Company's actual results to differ materially from those in the
forward-looking statements. Such risks and uncertainties include,
among others, the availability of funds and resources to pursue
R&D projects, the successful and timely completion of clinical
studies, the ability of the Company to take advantage of business
opportunities in the pharmaceutical industry, uncertainties related
to the regulatory process and general changes in economic
conditions. Investors should consult the Company's quarterly and
annual filings with the Canadian and U.S. securities commissions
for additional information on risks and uncertainties relating to
forward-looking statements. Investors are cautioned not to rely on
these forward-looking statements. The Company does not undertake to
update these forward-looking statements. We disclaim any obligation
to update any such factors or to publicly announce the result of
any revisions to any of the forward-looking statements contained
herein to reflect future results, events or developments, unless
required to do so by a governmental authority or by applicable
law.
SOURCE AETERNA ZENTARIS INC.