Aeterna Zentaris Completes Patient Recruitment for Confirmatory Phase 3 Trial of Macrilen™
October 26 2016 - 8:00AM
Business Wire
Expects to File NDA in H1 of 2017, if
Warranted by Trial Results
Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the “Company”)
today announced it has successfully completed patient recruitment
for the confirmatory Phase 3 clinical trial of Macrilen™
(macimorelin) as a growth hormone stimulation test for the
evaluation of growth hormone deficiency in adults (“AGHD”). The
Company also confirmed that it expects to file a New Drug
Application for Macrilen™ with the United States Food and Drug
Administration (the “FDA”) during the first half of 2017, if the
results of the trial warrant doing so. Macrilen™ is the Company’s
proposed tradename for macimorelin. The proposed tradename is
subject to approval by the FDA.
Dr. Richard Sachse, the Company’s Chief Scientific Officer,
stated, “We are committed to the development of Macrilen™ because
of the medical need for such a convenient test in the absence of an
FDA-approved diagnostic test for AGHD. Patients who are suspected
of having AGHD are now very often evaluated by means of the insulin
tolerance test (“ITT”). While the ITT is the historical
gold-standard for the evaluation of AGHD, the procedure is
inconvenient for patients and physicians and contraindicated in
certain patients, such as patients with coronary heart disease or
seizure disorder, because it requires the patient to experience
hypoglycemia to obtain a result. In addition, it is not an
FDA-approved procedure. Furthermore, administration of the ITT is
expensive because the patient must be constantly monitored by a
physician for the 2- to 4-hour duration of the test and the test
must be administered in a setting where emergency equipment is
available and where the patient may be quickly hospitalized, if
necessary. With the completion of recruitment for our confirmatory
Phase 3 clinical trial of Macrilen™ for the evaluation of AGHD, we
are another step closer to being able to provide this important
product to clinicians and patients.”
The Company believes that, in the US alone, approximately 40,000
confirmatory tests for AGHD will be conducted each year after the
introduction of Macrilen™, if it is approved by the FDA, which
represents the target market for Macrilen™ at the time of its
anticipated commercialization. Furthermore, the Company believes
that Macrilen™, if it is approved, is likely to be rapidly adopted
by physicians as the preferred means of evaluating AGHD for the
following reasons:
- it is safer than the ITT because it
does not require the patient to become hypoglycemic and thus avoids
the symptoms and potential complications of hypoglycemia;
- Macrilen™ is administered orally, while
the ITT requires an intravenous infusion of insulin;
- the evaluation of AGHD using Macrilen™
is significantly less time consuming and labor intensive than the
ITT and, therefore, it is less expensive to conduct; and
- the evaluation can be conducted in the
physician’s office rather than in a hospital setting.
As a result, the Company believes that Macrilen™, if it is
approved, may be used for 40% to 50% of AGHD evaluations in the
U.S. during the first year after its introduction and that the
percentage could grow to as high as 85% within the first three
years of commercialization.
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 involves some 30 sites in the
United States and Europe. The study population consists of more
than 110 subjects (at least 55 ITT-positive and 55 ITT-negative)
with a medical history documenting risk factors for AGHD, and
includes a spectrum of subjects from those with a low risk of
having AGHD to those with a high risk of having the condition. The
primary objective is validation of a single oral dose of
macimorelin for the diagnosis of AGHD, using the ITT as a
comparator. 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 US and European approval, if
successful. For more details on the trial, please consult this
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 US, 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 are now conducting 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
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 provisions of the US Securities
Litigation Reform Act of 1995. 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 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 rejection or non-acceptance of any new drug application
by one or more regulatory authorities and, more generally,
uncertainties related to the regulatory process, 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, the ability of the
Company to take advantage of business opportunities in the
pharmaceutical industry, 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, except if
required to do so.
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Aeterna Zentaris Inc.Philip A. TheodoreSenior Vice
Presidentir@aezsinc.com843-900-3223
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