Aeterna Zentaris: Phase 3 Results for AEZS-130 in AGHD Presented at
GRS and IGF Society Congress
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 6th
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.
Results
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
www.aezsinc.com.
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SOURCE AETERNA ZENTARIS INC.