Trial conducted through a Cooperative Research and
Development Agreement
QUÉBEC CITY, March 8, 2012
/PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") today announced that the Michael E. DeBakey
Veterans Affairs Medical Center, in Houston, Texas, has initiated a Phase 2A trial
assessing the safety and efficacy of repeated doses of the
Company's ghrelin agonist, AEZS-130 (macimorelin), in patients with
cancer cachexia. The study is conducted under a Cooperative
Research and Development Agreement (CRADA) between Aeterna Zentaris
and the Michael E. DeBakey Veterans Affairs Medical Center which is
funding the study. Cachexia, characterized by diminished appetite
and food intake in cancer patients, is defined as an involuntary
weight loss of at least 5% of the pre-illness body weight over the
previous 6 months. Jose M.
Garcia, MD, PhD, Assistant Professor, Division of Diabetes
Endocrinology and Metabolism, Departments of Medicine, Molecular
and Cell Biology, the Dan L. Duncan Cancer Center and the
Huffington Center on Aging at Baylor College
of Medicine and Michael E. DeBakey Veterans Affairs
Medical Center, will act as the Principal Investigator of this
trial.
Juergen Engel, PhD, President and
CEO of Aeterna Zentaris stated, "Reaching agreement on this CRADA
with the Michael E. DeBakey VA Medical Center was an important step
which paved the way for the initiation of this trial. Furthermore,
the study with AEZS-130 in cancer cachexia opens another important
pathway for the development of this drug in the clinic."
Jose M. Garcia, MD, PhD, added,
"I am pleased that we reached agreement on the conduct of this
research cooperatively and that we can now initiate this study. The
results generated by the proposed experiments will set the basis
for future trials in this and other wasting conditions that also
are very prevalent including chronic obstructive pulmonary disease
(COPD), renal failure or chronic heart failure (CHF)-induced
cachexia."
The Study
This is a double-blind, randomized, placebo-controlled Phase 2A
trial to test the effects of different doses of the ghrelin
agonist, AEZS-130, in 18 to 26 patients with cancer-cachexia.
AEZS-130 will be provided by Aeterna Zentaris. The study will
involve 3 sequential groups receiving differing doses of AEZS-130.
Each dose group will have 6 patients who will receive AEZS-130 and
2-4 patients who will receive placebo. After analysis of safety and
efficacy at each dose level vs. placebo, a decision will be taken
either to decrease or increase the dose. If there are major safety
issues, the study will be stopped. For this study, adequate
efficacy will be defined as a ≥0.8 kg of body weight gain or a ≥50
ng/mL increase in plasma IGF-1 levels. The primary objective of the
study is to evaluate the safety and efficacy of repeated oral
administration of AEZS-130 at different doses daily for 1 week in
view of developing a treatment for cachexia. The following
parameters will be recorded to assess efficacy during the study:
change of body weight, change of IGF-1 plasma levels, and change of
quality of life score (Anderson Symptom Assessment Scale, FACIT-F).
Other secondary objectives will include food intake, and changes in
the following: appetite, muscle strength, energy expenditure,
reward from food and functional brain connectivity.
About Cancer Cachexia
Over 1,500,000 new diagnoses of cancer are made in the U.S.
every year. In most cases, this condition can have a significant
impact on the individuals' survival and quality of life. Cachexia,
a complex metabolic syndrome associated with underlying illness, is
common in patients with cancer. These patients have diminished
appetite, food intake and insulin sensitivity which contribute to a
decrease in functional performance, takes a heavy toll on patients'
quality of life and is associated with poor survival.
Despite the significant burden that cachexia and other
cancer-related symptoms such as anorexia and poor functional status
represent to cancer patients, these symptoms often remain
undiagnosed and untreated. Moreover, there are currently no
approved treatments for cancer-related cachexia and off-label
treatments used for this condition such as appetite stimulants
(e.g., megestrol acetate) are largely ineffective and are
associated with potentially serious side effects (i.e., adrenal
insufficiency, hypogonadism, deep venous thrombosis, etc.).
About AEZS-130
AEZS-130, a ghrelin agonist, is a novel orally-active small
molecule that stimulates the secretion of growth hormone by binding
the ghrelin receptor (GHSR-1a). It can be used in both oncology and
endocrinology indications. In oncology, an IND has been granted for
a Phase 2A trial with AEZS-130 in cancer induced cachexia, a
disease which leads to significant weight loss and diminished
functional performance. Since ghrelin agonists such as AEZS-130
have been shown to stimulate food intake and increase body weight
in rats and mice, AEZS-130 could lead to better quality of life for
patients with cancer-induced cachexia. Ghrelin agonists have been
in clinical trials for over a decade and have demonstrated good
safety and efficacy profiles.
In endocrinology, the Company has completed a Phase 3 trial for
use as a simple oral diagnostic test for adult growth hormone
deficiency (AGHD) and expects to file an NDA in 2012. 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.
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 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 developments, unless
required to do so by a governmental authority or by applicable
law.
SOURCE AETERNA ZENTARIS INC.