Aeterna Zentaris Intends to File NDA with Respect to Macrilen™ in Third Quarter of 2017
March 30 2017 - 8:00AM
Business Wire
Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (the “Company”)
today announced that, following its meeting with the U.S. Food and
Drug Administration (the “FDA” or the “Agency”) on March 29, 2017,
the Company intends to file a new drug application (“NDA”) seeking
approval of Macrilen™ (macimorelin) for the evaluation of growth
hormone deficiency in adults (“AGHD”).
Commenting on the meeting, Dr. Richard Sachse, the Company’s
Chief Scientific Officer, stated, “During our meeting with the FDA,
the Agency stated that the clinical studies we performed with
respect to Macrilen™ address the prior deficiencies mentioned in
the November 2014 complete response letter. This conclusion paves
the way for our re-submission of an NDA for Macrilen™, which we
expect to file in the third quarter of this year. While indicating
that our conclusions regarding the performance of Macrilen™ are
review issues subject to an examination of the complete data set,
the Agency indicated that the summary data we submitted prior to
the meeting appear to support the propositions we advanced. Most
importantly, the FDA specified the additional statistical analysis
of existing data that would be required to further support our
conclusions. We expect that we can provide those data in a
compelling fashion and demonstrate that Macrilen™ is a robust,
repeatable test, demonstrating adequate sensitivity and specificity
and that the performance of the product would be improved by
utilizing a more appropriate cut-off point.”
David A. Dodd, President and Chief Executive Officer of the
Company stated, “We were encouraged by our dialogue with the FDA
regarding Macrilen™. The Agency provided very helpful guidance to
us that should help us in the registration process. Of course, the
FDA will thoroughly review all data we provide with our NDA and
make a decision regarding the approval of the product after doing
so. Although there can be no assurance of approval of any NDA, we
believe that we are now one important step closer to the
commercialization of Macrilen™ in the U.S., providing a much needed
new option and alternative to the ITT.”
About the Study
The confirmatory Phase 3 clinical study of Macrilen™, entitled
Confirmatory validation of oral macimorelin as a growth hormone
(GH) stimulation test (ST) for the diagnosis of adult growth
hormone deficiency (AGHD) in comparison with the insulin tolerance
test (ITT), was designed as a two-way crossover study with the ITT
as the benchmark comparator and involved some 26 sites in the U.S.
and Europe. The trial involved 157 subjects, of whom 140 completed
two evaluable tests for AGHD using both Macrilen™ and the ITT.
Thirty-four of the patients were evaluated using Macrilen™ a second
time to measure the repeatability of the result obtained using
Macrilen™ as the evaluation method. The study population consisted
of 115 patients who were suspected of having AGHD as a result of
the presence of one or more symptoms or risk factors. This segment
of the population included a range of patients from those
considered at low risk of having AGHD to those considered at high
risk. The study population also included 25 healthy subjects, who
had no risk of having AGHD. Under the study protocol, the
evaluation of AGHD with Macrilen™ will be considered successful, if
the lower bound of the two-sided 95% confidence interval (or lower
bound of the one-sided 97.5% confidence interval) for the primary
efficacy variables is 75% or higher for “percent negative
agreement”, and 70% or higher for the “percent positive agreement”.
Based on meetings with the FDA as well as the European Medicines
Agency (“EMA”) and subsequent written scientific advice, the
Company believes that the study meets the FDA’s and the EMA’s
study-design expectations allowing U.S. and European approval if
other conditions are met. Dr. Jose M. Garcia, MD, PhD, an Associate
Professor of Medicine at the Puget Sound VA Hospital and the
University of Washington in Seattle, was the principal investigator
of the confirmatory Phase 3 clinical trial. More details about the
trial are available at the following link:
https://www.clinicaltrials.gov/ct2/show/NCT02558829?term=macimorelin&rank=1.
About MacrilenTM (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 treatments in
oncology, endocrinology and women’s health. 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 comprehensive
review of the Phase 3 study data 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.
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version on businesswire.com: http://www.businesswire.com/news/home/20170330005118/en/
Aeterna Zentaris Inc.Philip A. Theodore, 843-900-3223Senior Vice
Presidentir@aezsinc.com
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