Last updated on April 23, 2014 at 15:23 EDT

Aeterna Zentaris: FDA Grants IND to Investigator at Baylor College of Medicine for Phase 2A Trial with AEZS-130 in Cancer Cachexia

November 28, 2011

QUÓ°BEC CITY, Nov. 28, 2011 /PRNewswire/ – Aeterna Zentaris Inc. (NASDAQ:
AEZS) (TSX: AEZ) (the “Company”) today announced that the U.S. Food and
Drug Administration (FDA) has granted Jose M. Garcia, MD, PhD,
Assistant Professor, Division of Diabetes Endocrinology and Metabolism,
Departments of Medicine and Molecular and Cell Biology, Baylor College
of Medicine and Michael I. DeBakey Veterans Administration Medical
Center, in Houston Texas, an Investigational New Drug (IND) approval
for the initiation of a Phase 2A trial to assess the safety and
efficacy of repeated doses of the Company’s ghrelin agonist, AEZS-130
(macimorelin), in patients with cancer cachexia. Cachexia, which is
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.

Juergen Engel, PhD, President and CEO of Aeterna Zentaris stated, “This
trial is further proof of the versatile potential of AEZS-130. Not only
are we developing AEZS-130 as a supportive treatment in cancer care,
but we are also developing it as the first oral diagnostic test for
Adult Growth Hormone Deficiency for which we have already initiated the
preparation for an NDA filing in the United States.”

Jose M. Garcia, MD, PhD, the principal investigator added, “Weight loss
and decreased appetite (cachexia and anorexia) are devastating
complications of cancer. However, treatments for this condition are
lacking. Ghrelin agonists have the potential of benefiting these
patients by virtue of their anabolic and appetite-stimulating
properties. The current proposal will explore the safety and efficacy
of the novel ghrelin agonist, AEZS-130, in this setting. If effective,
this could allow subjects to receive more effective doses of
chemotherapy and to have a better functional status. The results
generated by the proposed experiments will set the bases 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. The study will be conducted
under a cooperative research and development agreement (CRADA) with the
Michael I. DeBakey Veterans Administration Medical Center which will be
funding the study. 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 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, change of appetite, muscle
strength and energy expenditure.

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. It can be used in both
endocrinology and in oncology indications. In endocrinology, 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. 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.

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: PR Newswire