Aeterna Zentaris Announces Favorable Top-Line Results of Completed Phase 3 Study on AEZS-130 as First Oral Diagnostic Test for Adult Growth Hormone Deficiency
Company will file New Drug Application
QUÃƒ“°BEC CITY, Aug. 30, 2011 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the “Company”) today
announced favorable top-line results of its completed Phase 3 study
with AEZS-130 as the first oral diagnostic test for Adult Growth
Hormone Deficiency (AGHD). The results show that AEZS-130 reached its
primary endpoint demonstrating >90% area-under-the-curve (AUC) of the
Receiver Operating Characteristic (ROC) curve, which determines the
level of specificity and sensitivity of the product. The Company is
currently proceeding with further 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.
The parameters of the study, as defined below under Study Design, were
achieved as agreed to with FDA under our Special Protocol Assessment
(SPA). Importantly, the primary efficacy parameters show that the study
achieved both specificity and sensitivity at a level of 90% or
greater. In addition, 8 of the 10 newly enrolled AGHD patients were
correctly classified by a pre-specified peak GH threshold level. The
use of AEZS-130 was shown to be safe and well tolerated overall
throughout the completion of this trial.
“We are pleased with the results obtained and we therefore expect to
meet with the FDA and work out the content of a submission for an NDA.
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
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(Ã‚®) + Arginine (ARG) standard test. Geref Diagnostic(Ã‚®) was subsequently withdrawn from the market, worldwide, in 2008; the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.
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 Ã‚µg/kg
(max. 100 Ã‚µg) dose of GHRH (Geref Diagnostic(Ã‚®), Serono) with 30 g of ARG (Ar-Gine(Ã‚®), 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 to be enrolled to match the AGHD patients from the original cohort; -- Further, an additional 20 subjects were to be 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.
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, which normally only occurs during
sleep, 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.
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.
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.