Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the
“Company”), a specialty biopharmaceutical company commercializing
and developing therapeutics and diagnostic tests, today provided a
corporate update.
“Over the course of the last year since my
appointment as CEO, a tremendous amount has been achieved. With the
set intention to first assess and determine how best to support our
current development partnership and how to maximize its success,
followed by pursuing the expansion of macimorelin into pediatric
use, I believe we are now well-positioned to advance Aeterna into
its next phase of growth,” commented Dr. Klaus Paulini, Chief
Executive Officer of Aeterna. “A catalyst that has significantly
powered the Company with the momentum we have today was
successfully raising a total of $23.5 million since the beginning
of this year. By strategically and opportunistically securing this
capital, we believe we have both a solid foundation to support
optionality moving forward and equipped the Company with necessary
resources to enhance our pipeline in a meaningful way.”
“Our team is first and foremost focused on
continuing our efforts to bring the expanded use of macimorelin
over the finish line for the diagnosis of childhood-onset growth
hormone deficiency (“CGHD”), an area of significant unmet need and,
if approved, has the potential to significantly increase the
available patient population for macimorelin. We are actively
preparing, and hope to be able to commence, our pivotal Phase 3
safety and efficacy study AEZS-130-P02 (“Study P02”) in the first
quarter of 2021,” added Dr. Paulini.
Macimorelin Clinical Program
Update
The Company’s lead product, macimorelin, is the
only United States Food and Drug Administration (“FDA”) approved
oral drug indicated for the diagnosis of AGHD and is currently
marketed in the United States (“U.S.”) under the tradename
Macrilen™, by Novo Nordisk. Aeterna is currently developing
macimorelin for the diagnosis of CGHD, an area of significant unmet
need, in collaboration with Novo Nordisk.
Results from the AEZS-130-P01 (“Study P01”), the
first of two studies as agreed with the European Medicines Agency
(“EMA”) in the Company’s Pediatric Investigation Plan (“PIP”) for
macimorelin, were announced in April 2020. The positive
dose-finding results from Study P01 provide the clinical framework
for Study P02 and establish a dose that could both be safely
administered to pediatric patients and cause a clear rise in growth
hormone concentration in subjects ultimately diagnosed as not
having GHD.
Study P02 is planned as open-label, single dose,
multicenter and multinational study expected to enroll ~100
subjects worldwide, with at least 40 pre-pubertal and 40 pubertal
subjects and a minimum of 25 subjects expected to be enrolled in
the USA. Enrolled subjects in the study will be children and
adolescents from 2 years of age to less than 18 years of age with
suspected GHD. Enrolled subjects in Study P02 will have macimorelin
GHST performed twice (for repeatability data) in comparison to two
standard GHSTs as controls, arginine (i.v.) and clonidine (p.o.).
The study design is suitable to support a claim for potential
stand-alone testing, if successful.
Aeterna has also begun exploring the potential
therapeutic use of macimorelin in various other indications. The
Company has ongoing plans to evaluate the development of
alternative formulations or administration routes with the goal of
ensuring sufficient bioavailability and expects to be providing
updates on its progress as results become available over the course
of the next several months.
In addition to advancing its pediatric program,
Aeterna is intensifying its business development activities with
the goal of securing marketing partners for macimorelin for the
diagnosis of GHD in Europe and other key markets.
Pipeline Expansion
Opportunities
Aeterna Zentaris intends to balance risks and
secure growth opportunities by re-establishing a diversified, yet
focused, development pipeline to which the Company can best
leverage its expertise and experience. The Company is focused on
opportunistically utilizing its well-established, extensive network
with universities in Europe and the US. This strong network of
strategic collaborations with universities, provides, what the
Company believes will be, vital access to innovative development
candidates in different indications, with a focus on rare or orphan
indications and potential for pediatric use.
Dr. Paulini concluded, “Aeterna Zentaris was
established with strong expertise in research and development,
which I believe is an incredible asset to the Company and one that
we plan to leverage moving forward. Apart from investigating
opportunities for new therapeutic usages of macimorelin, we are
assessing whether development candidates from our previous programs
may be re-purposed for alternative indications based on prior key
findings from data already available to us. With our capabilities
and specific know-how, we believe we are well-positioned as we
explore these opportunities and consider potential co-development
strategies that could be synergistic for the Company moving
forward.”
About Aeterna Zentaris Inc.
Aeterna Zentaris Inc. is a specialty
biopharmaceutical company commercializing and developing
therapeutics and diagnostic tests. The Company’s lead product,
Macrilen™ (macimorelin), is the first and only U.S. FDA and
European Commission approved oral test indicated for the diagnosis
of adult growth hormone deficiency (AGHD). Macrilen™ is currently
marketed in the United States through a license agreement with Novo
Nordisk and Aeterna Zentaris receives double-digit royalties on
sales. Aeterna Zentaris owns all rights to macimorelin outside of
the U.S. and Canada.
Aeterna Zentaris is also leveraging the clinical
success and compelling safety profile of macimorelin to develop it
for the diagnosis of child-onset growth hormone deficiency (CGHD),
an area of significant unmet need.
The Company is actively pursuing business
development opportunities for the commercialization of macimorelin
in Europe and the rest of the world, in addition to other
non-strategic assets to monetize their value. For more information,
please visit www.zentaris.com and connect with the Company on
Twitter, LinkedIn and Facebook.
Forward-Looking Statements
This press release contains forward-looking
statements (as defined by applicable securities legislation) 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
include those relating to the Company obtaining approval of
macimorelin for CGHD and the resulting potential to significantly
increase the available patient population for macimorelin, the
Company’s ability to to secure marketing partners for macimorelin
for GHD in Europe and elsewhere, the commencement of the CGHD Study
P02, the ability of the Company to identify and develop therapeutic
uses for macimorelin in new indications and the ability of the
Company to expand its pipeline of products, and may include, but
are not limited to statements preceded by, followed by, or that
include the words "will," "expects," "believes," "intends,"
"would," "could," "may," "anticipates," and similar terms that
relate to future events, performance, or our results.
Forward-looking statements involve known and unknown risks and
uncertainties, including those discussed in this press release and
in our Annual Report on Form 20-F, under the caption "Key
Information - Risk Factors" filed with the relevant Canadian
securities regulatory authorities in lieu of an annual information
form and with the U.S. Securities and Exchange Commission. 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, our ability to raise
capital and obtain financing to continue our currently planned
operations, our ability to continue to list our Common Shares on
the NASDAQ, our now heavy dependence on the success of Macrilen™
(macimorelin) and related out-licensing arrangements and the
continued availability of funds and resources to successfully
commercialize the product, including our heavy reliance on the
success of the License Agreement with Novo, the global instability
due to the global pandemic of COVID-19, and its unknown potential
effect on our planned operations, including studies, our ability to
enter into out-licensing, development, manufacturing, marketing and
distribution agreements with other pharmaceutical companies and
keep such agreements in effect, our reliance on third parties for
the manufacturing and commercialization of Macrilen™ (macimorelin),
potential disputes with third parties, leading to delays in or
termination of the manufacturing, development, out-licensing or
commercialization of our product candidates, or resulting in
significant litigation or arbitration, uncertainties related to the
regulatory process, unforeseen global instability, including the
instability due to the global pandemic of the novel coronavirus,
our ability to efficiently commercialize or out-license Macrilen™
(macimorelin), our reliance on the success of the pediatric
clinical trial in the European Union (“E.U.”) and U.S. for
Macrilen™ (macimorelin), the degree of market acceptance of
Macrilen™ (macimorelin), our ability to obtain necessary approvals
from the relevant regulatory authorities to enable us to use the
desired brand names for our product, our ability to successfully
negotiate pricing and reimbursement in key markets in the E.U. for
Macrilen™ (macimorelin), any evaluation of potential strategic
alternatives to maximize potential future growth and shareholder
value may not result in any such alternative being pursued, and
even if pursued, may not result in the anticipated benefits, 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 our quarterly and annual filings with the Canadian
and U.S. securities commissions 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.
Investor Contact:
Jenene Thomas JTC Team T (US): +1 (833) 475-8247 E:
aezs@jtcir.com
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