Aeterna Zentaris: Phase 3 Results for AEZS-130 in AGHD Presented at GRS and IGF Society Congress

 Aeterna Zentaris: Phase 3 Results for AEZS-130 in AGHD Presented at GRS and
                             IGF Society Congress

PR Newswire

QUEBEC CITY, Oct. 18, 2012

QUEBEC CITY, Oct. 18, 2012 /PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS)
(TSX:AEZ) (the "Company") today announced that Phase 3 results for its
ghrelin agonist, AEZS-130, show that it has promise as a safe and simple oral
diagnostic test for adult growth hormone deficiency (AGHD), with accuracy
comparable to available testing procedures. Results were presented earlier
today by George R. Merriam, MD, Director of the Clinical Study Unit at the VA
Puget Sound Health Care System, and Professor of Medicine at the University of
Washington, Seattle and Tacoma, WA, at the 6^th International Congress of the
Growth Hormone Research (GRS) and Insulin-like Growth Factor (IGF) Society,
currently being held in Munich, Germany.

Juergen Engel, PhD, President and CEO at Aeterna Zentaris, commented, "The
data presented earlier today by Dr. Merriam, extend those presented on this
same study last June at ENDO by Dr. JoseM. Garcia, MD, PhD, of the Baylor
College of Medicine and the Michael E. DeBakey VA Medical Center. Both confirm
AEZS-130's potential as possibly the first approved oral diagnostic test for
AGHD, with Dr. Merriam's data set showing the impact of Body Mass Index on
cut-off values. We are currently focusing our efforts on submitting a New Drug
Application for AEZS-130 in this indication during the first quarter of 2013."

The Study

This multicenter open-label study was originally designed as a cross-over
trial of AEZS-130 vs growth hormone-releasing hormone (GHRH)+L-Arginine (ARG)
in AGHD patients and in controls matched for Body Mass Index (BMI), estrogen
status, gender and age. After 43 AGHD patients and 10 controls had been
tested, GHRH became unavailable. The study was completed by testing 10 more
AGHD patients and 38 controls with AEZS-130 alone.

Of the 53 AGHD subjects enrolled, 52 received AEZS-130, and 50 who had
confirmed AGHD prior to study entry were included in this analysis, along with
48 controls. Two AGHD subjects could not be matched due to the combination of
young age, high BMI and estrogen use. The objective of this clinical trial was
to determine the efficacy and safety of AEZS-130 in the diagnosis of AGHD.


Mean peak growth hormone (GH) levels in AGHD patients and controls following
AEZS-130 administration were 2.36ng/mL (range 0.03-33) and 17.71ng/mL (range
10.5-94), respectively. The receiver operating characteristic (ROC) plot
analysis yielded an optimal GH cut-point of 2.7ng/mL, with 82% sensitivity,
92% specificity and a 13% misclassification rate. Obesity (BMI>30) was present
in 58% of cases and controls, and peak GH levels were inversely associated
with BMI in controls.

Adverse events (AE) were seen in 37% of AGHD patients and in 21% of controls
following AEZS-130. In contrast, 61% of AGHD subjects and 30% of controls
experienced AEs with L-ARG+GHRH. The most common AEs after AEZS-130 were
unpleasant taste (19.2%) and diarrhea (3.8%) for the AGHD patients and
unpleasant taste (4.2%) and diarrhea (4.2%) for the matched controls. AEs were
generally mild or moderate in severity.

Of the 50 subjects studied with both stimulation tests, 70% expressed a
preference for AEZS-130 over L-ARG+GHRH.

Summary and conclusions on the effect of BMI on optimal cut-off

Responses in normal subjects classified as obese, with BMI's above 30, were
significantly lower than in leaner subjects. Since GH deficiency can lead to
increased body fat, many of the patients also met criteria for obesity, and
therefore, a lower peak GH cutoff is more accurate in separating obese normals
from obese patients. Based upon these study results, a cut-off of 2.7 μg/L was
optimal for subjects with a BMI ≥ 30 and a cutoff of 6.8 μg/L for subjects
with a BMI of <30. Age had a much weaker effect on test performance and
gender made no difference. Thus GH stimulation with oral AEZS-130 may provide
a simple, rapid, safe, and well-tolerated diagnostic test for AGHD, with
accuracy comparable to that of the GHRH-ARG test.

The abstract used for the presentation, "Use of an orally-active ghrelin
mimetic, macimorelin (AEZS-130), as a safe, simple test for Adult Growth
Hormone Deficiency (AGHD): Effect of BMI on optimal cut-off ", can be viewed
by clicking on the following link.

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 an oral diagnostic test for AGHD. AEZS-130 has been granted
orphan drug designation by the FDA for use in this indication. Aeterna
Zentaris owns the worldwide rights to AEZS-130.

About Aeterna Zentaris

Aeterna Zentaris is an oncology and endocrinology drug development company
currently investigating treatments for various unmet medical needs. The
Company's pipeline encompasses compounds at all stages of development, from
drug discovery through to marketed products. For more information please visit

Forward-Looking Statements

This press release contains forward-looking statements made pursuant to the
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Forward-looking statements involve known and unknown risks and uncertainties
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uncertainties include, among others, the availability of funds and resources
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studies, the risk that safety and efficacy data from any of our Phase 3 trials
may not coincide with the data analyses from previously reported Phase 1
and/or Phase 2 clinical trials, 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.
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Investor Relations
Ginette Beaudet Vallières
Investor Relations Coordinator
(418) 652-8525 ext. 265

Media Relations
Paul Burroughs
Director of Communications
(418) 652-8525 ext. 406
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