NDA for Macrilen™ for the Evaluation of Growth Hormone Deficiency in Adults Granted December 30, 2017 PDUFA Date
Aeterna Zentaris Inc. (NASDAQ: AEZS)(TSX: AEZS) (the “Company”) today announced that it has been notified by the U.S. Food and
Drug Administration (“FDA”), that the Company’s New Drug Application (“NDA”) seeking approval of Macrilen™ (macimorelin) for the
evaluation of growth hormone deficiency in adults (“AGHD”) has been accepted as a complete response to the FDA’s November 5, 2014
Complete Response Letter and granted a PDUFA date of December 30, 2017.
David A. Dodd, President and Chief Executive Officer of the Company stated, “We are pleased that the FDA has formally accepted
our resubmitted NDA and that it is under active review with an end-of-year PDUFA date. We remain confident that the FDA will
approve our NDA and, therefore, we are moving forward with our preparations to launch the product in the first quarter of
2018.”
The Company also announces that Mr. Kenneth Newport is no longer a member of the Board of Directors effective as of July 12,
2017.
About Macrilen TM (macimorelin)
Macimorelin, a ghrelin agonist, is an orally-active small molecule that stimulates the secretion of growth hormone. Macimorelin
has been granted orphan drug designation by the FDA for diagnosis of AGHD. The Company owns the worldwide rights to this patented
compound and has significant patent protection left. The Company’s U.S. composition of matter patent expires in 2022 and its U.S.
utility patent runs through 2027. The Company proposes, subject to FDA approval, to market macimorelin under the tradename
Macrilen™.
About 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 as well as an increase of cardiovascular risks.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty biopharmaceutical company engaged in developing and commercializing novel pharmaceutical
therapies. We are engaged in drug development activities and in the promotion of products for others. We recently completed Phase 3
studies of two internally developed compounds. The focus of our business development efforts is the acquisition of licenses to
products that are relevant to our therapeutic areas of focus. We also intend to license out certain commercial rights of internally
developed products to licensees in non-U.S. territories where such out-licensing would enable us to ensure development,
registration and launch of our product candidates. Our goal is to become a growth-oriented specialty biopharmaceutical company by
pursuing successful development and commercialization of our product portfolio, achieving successful commercial presence and
growth, while consistently delivering value to our shareholders, employees and the medical providers and patients who will benefit
from our products. For more information, visit www.aezsinc.com.
Forward-Looking Statements
This press release contains forward-looking statements made pursuant to the safe harbor provision of the U.S. Securities
Litigation Reform Act of 1995, which reflect our current expectations regarding future events. Forward-looking statements may
include, but are not limited to statements preceded by, followed by, or that include the words “expects,” “believes,” “intends,”
“anticipates,” and similar terms that relate to future events, performance, or our results. Forward-looking statements involve
known risks and uncertainties, many of which are discussed under the caption “Key Information – Risk Factors” in our most recent
Annual Report on Form 20-F filed with the relevant Canadian securities regulatory authorities in lieu of an annual information form
and with the U.S. Securities and Exchange Commission (“SEC”). Such statements include, but are not limited to, statements about the
progress of our research, development and clinical trials and the timing of, and prospects for, regulatory approval and
commercialization of our product candidates, the timing of expected results of our studies, anticipated results of these studies,
statements about the status of our efforts to establish a commercial operation and to obtain the right to promote or sell products
that we did not develop and estimates regarding our capital requirements and our needs for, and our ability to obtain, additional
financing. Known and unknown risks and uncertainties could cause our actual results to differ materially from those in
forward-looking statements. Such risks and uncertainties include, among others, the availability of funds and resources to pursue
our research and development 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 rejection or non-acceptance of any new drug application by one or more regulatory authorities
and, more generally, uncertainties related to the regulatory process (including whether or not the regulatory authorities will
definitively accept the Company’s conclusions regarding Macrilen™ and approve its registration following the Company’s
re-submission of an NDA for the product as described elsewhere in this press release), the ability of the Company to efficiently
commercialize one or more of its products or product candidates, the degree of market acceptance once our products are approved for
commercialization, our ability to take advantage of business opportunities in the pharmaceutical industry, our ability to protect
our intellectual property, and 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 securities commissions and the
SEC for additional information on risks and uncertainties. Given these uncertainties and risk factors, readers are cautioned not to
place undue reliance on these forward-looking statements. We disclaim any obligation to update any such factors or to publicly
announce 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 applicable law.
Aeterna Zentaris Inc.
Philip A. Theodore, 843-900-3211
Senior Vice President
ir@aezsinc.com
View source version on businesswire.com: http://www.businesswire.com/news/home/20170718006321/en/