All amounts are in US dollars
QUÉBEC CITY, April 13, 2015
/PRNewswire/ - Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ)
(the "Company") today announced plans to conduct a new,
confirmatory Phase 3 clinical study to demonstrate the efficacy of
Macrilen™ (macimorelin), a novel orally-active ghrelin agonist for
use in evaluating adult growth hormone deficiency ("AGHD"), as well
as a dedicated thorough QT study to evaluate the effect of
Macrilen™ on myocardial repolarization. This decision follows a
positive and helpful meeting with the U.S. Food and Drug
Administration ("FDA") regarding its New Drug Application for
Macrilen™. The Company requested the meeting to gain clarity on the
approval deficiencies described in the Complete Response Letter
("CRL") the Company received on November 6, 2014.
Following receipt of the CRL, the Company convened a panel of US
and EU endocrinology experts to advise it regarding the options for
Macrilen™. The panel advised the Company to continue to seek
approval for the compound because of their confidence in its
efficacy and because there currently is no FDA-approved diagnostic
test for AGHD. Dr. Richard Sachse,
Chief Scientific Officer and Chief Medical Officer of the Company
stated, "I am very grateful for the advice of our advisory
committee and for their confidence in Macrilen™. Their
recommendations were very helpful in formulating our proposal to
the FDA. Also, as we've continued to evaluate the available
information on Macrilen™, we continue to believe further trials
will confirm the efficacy and safety of this product. Our intent is
to successfully develop this product, providing a much needed
improved and FDA-approved method for endocrinologists to evaluate
and diagnose AGHD."
During an end-of-review meeting with the FDA on March 6, 2015, the Company and the FDA agreed on
the general design of the confirmatory study as well as evaluation
criteria. The study will be conducted as a two-way crossover with
the insulin tolerance test as the benchmark comparator. The study
population will consist of patients with a medical history
documenting risk factors for AGHD and will include a spectrum of
patients from those with a low risk of having AGHD to those with a
high risk of having the condition. The Company will submit a
proposed final protocol to the FDA for approval prior to commencing
the confirmatory study.
David A. Dodd, Chairman and Chief
Executive Officer of the Company commented on the future
development of MacrilenTM: "We are committed to moving
forward rapidly with the development and trials of
MacrilenTM. We believe that completion of the
confirmatory Phase 3 study and the QT study will take about 18
months and will require a combined expenditure of between
$5 million and $6 million. We have
the resources necessary to bring this product to market and intend
to do so as rapidly as possible pending regulatory approvals. I
want to thank my team for their extraordinary efforts in dealing
with the Complete Response Letter and in quickly finding a path
forward for MacrilenTM."
The Company's goal is to conduct a Phase 3 study that will
satisfy the registration requirements of the European Medicines
Agency ("EMA") as well as the FDA. The Company expects to receive
comments from the EMA regarding the study design during a
Scientific Advice Meeting in early May.
About MacrilenTM (macimorelin)
Macimorelin, a ghrelin agonist, is a novel orally-active small
molecule that stimulates the secretion of growth hormone.
Macimorelin, under the trade name MacrilenTM, has been
granted orphan drug designation by the FDA for diagnosis of
AGHD. The Company owns the worldwide rights to this novel
patented compound.
About Adult Growth Hormone Deficiency (AGHD)
AGHD affects approximately 75,000 adults across the U.S.,
Canada and Europe. Growth hormone not only plays an
important role in growth from childhood to adulthood, but also
helps promote a hormonally-balanced health status. AGHD mostly
results from damage to the pituitary gland. It is usually
characterized by a reduction in bone mineral density, lean body
mass, exercise capacity, and overall quality of life.
About Aeterna Zentaris
Aeterna Zentaris is a specialty biopharmaceutical company
engaged in developing and commercializing novel treatments in
oncology, endocrinology and women's health. For more information,
visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made
pursuant to the safe harbor provisions of the US 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 and clinical trials, 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 efficiently commercialize one
or more of its products or product candidates, the ability of the
Company to take advantage of business opportunities in the
pharmaceutical industry, uncertainties related to the regulatory
process, the ability to protect our intellectual property, the
potential of liability arising from shareholder lawsuits and
general changes in economic conditions. Investors should consult
the Company's quarterly and annual filings with the Canadian and US
securities commissions for additional information on risks and
uncertainties relating to forward-looking statements. Investors are
cautioned not to place undue reliance 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.