Last updated on April 18, 2014 at 1:21 EDT

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

October 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. Jose M. 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

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 mg/L was optimal for subjects with a BMI >= 30
and a cutoff of 6.8 mg/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

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 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. For
example, because the market price of the Company’s Common Shares will
also be based on the Company’s financial and operational results, its
financial position, including its capital availabilities and liquidity
resources, the development of its product pipeline, market conditions,
the market perception of its business and other factors, which are
unrelated to the number of shares outstanding, there can be no
assurance that the market price of the Common Shares will in fact
increase following the Consolidation or will not decrease in the
future, or that the minimum closing bid price of the Common Shares will
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assurance that the Consolidation alone will guarantee the continued
listing of the Common Shares on The NASDAQ Global Market or that the
Common Shares will not be delisted due to a failure to meet other
NASDAQ continued listing requirements. Other 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 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. 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
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Source: PR Newswire