Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (the “Company” or
“Aeterna Zentaris”), a specialty biopharmaceutical company
developing and commercializing a diversified portfolio of
pharmaceutical and diagnostic products, today announced the
commencement of the previously announced preclinical program to
qualify macimorelin for clinical development as a potential
treatment option for amyotrophic lateral sclerosis (ALS; Lou
Gehrig's disease). The preclinical development program is part of
the recently executed Material Transfer Agreement (MTA) with The
University of Queensland (“The University”).
The University researchers, led by Frederik
Steyn, MSc, PhD, will conduct preclinical studies in
disease-related in-vitro and in-vivo models of ALS to demonstrate
the therapeutic potential of macimorelin on disease progression and
ALS-specific pathology. Recently, The University researchers have
filed funding applications to dedicated organizations in Australia
to finance parts of the abbreviated preclinical development program
and to conduct a subsequent investigator-initiated clinical trial
to evaluate the safety, tolerability and efficacy of macimorelin as
a potential new treatment option for ALS patients.
Dr. Klaus Paulini, Chief Executive Officer of
Aeterna Zentaris, commented, “The start of the preclinical phase
represents a significant milestone in the development of a
potential new treatment option for ALS to help patients with this
terrible, incurable disease and to improve their quality of life.
We look forward to supporting Dr. Steyn’s research team in
addressing this significant unmet medical need.”
In the event of positive preclinical results,
the Company has the exclusive right to acquire a license from The
University on the therapeutic use of macimorelin for the treatment
of ALS.
Aeterna Zentaris’ lead product, macimorelin, is
the only United States Food and Drug Administration (“FDA”)
approved oral drug indicated for the diagnosis of adult growth
hormone deficiency (AGHD) and is currently marketed in the United
States under the tradename Macrilen™, by Novo Nordisk. It is also
licensed to Consilient Health Ltd. for the European Economic Area
and United Kingdom, and Megapharm Ltd. for Israel and the
Palestinian Authority. Aeterna Zentaris is currently developing
macimorelin for the diagnosis of childhood-onset growth hormone
deficiency (CGHD), an area of significant unmet need, in
collaboration with Novo Nordisk.
Ghrelin is a natural hormone stimulating release
of growth hormone. Its level is often reduced in ALS patients and
stimulation of GH excretion improves survival in ALS mouse models.
Ghrelin and ghrelin mimetics have already demonstrated in
preclinical models stimulation of growth factors to protect motor
neurons and enhance muscle building, stimulate appetite, slow
metabolism to promote positive energy balance and dampen
neuroinflammatory processes. Dr. Steyn’s research team at The
University have confirmed these positive effects of ghrelin
mimetics in several experiments. Ghrelin itself is not qualified as
medicine because of rapid clearance from circulation. However,
macimorelin is an orally active ghrelin agonist and could mimic the
biological effect of the hormone via mechanisms similar to
ghrelin.
About ALS
Amyotrophic lateral sclerosis (ALS) is the most
common motor neuron disease (MND). Most patients with ALS present
with random, asymmetric symptoms, consisting of cramps, weakness,
and muscle atrophy of the hands (most commonly) or feet. Weakness
progresses to the forearms, shoulders, and lower limbs.
Fasciculations, spasticity, hyperactive deep tendon reflexes,
extensor plantar reflexes, clumsiness, stiffness of movement,
weight loss, fatigue, and difficulty controlling facial expression
and tongue movements soon follow. Death is usually caused by
failure of the respiratory muscles; 50% of patients die within 3
years of onset, 20% live 5 years, and 10% live 10 years. Survival
for > 30 years is rare.
The mainstay of care for patients with
amyotrophic lateral sclerosis is timely intervention to manage
symptoms.
About Macimorelin Macimorelin,
a ghrelin agonist, is an orally active small molecule that
stimulates the secretion of growth hormone from the pituitary
gland. Stimulated growth hormone levels are measured in blood
samples after oral administration of macimorelin for the assessment
of growth hormone deficiency (GHD). Macimorelin is the only U.S.
FDA and European Commission approved oral test indicated for the
diagnosis of AGHD. Macimorelin is currently marketed in the United
States under the tradename Macrilen™, through a license agreement
with Novo Nordisk. Through a license agreement with Consilient
Health Ltd, macimorelin will be commercialized in Europe and the
United Kingdom upon receipt of pricing and reimbursement approvals
in the particular country. Additionally, the Company has entered
into a distribution agreement with MegaPharm Ltd. under which
MegaPharm will seek regulatory approval and then commercialize
macimorelin in Israel and the Palestinian Authority. Additionally,
the Company is leveraging the clinical success and compelling
safety profile of macimorelin to develop it for the diagnosis of
childhood-onset growth hormone deficiency (CGHD), an area of
significant unmet need, in collaboration with Novo Nordisk.
About Aeterna Zentaris Inc.
Aeterna Zentaris is a specialty
biopharmaceutical company developing and commercializing a
diversified portfolio of pharmaceutical and diagnostic products
focused on areas of significant unmet medical need. The Company's
lead product, 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). The Company is
leveraging the clinical success and compelling safety profile of
macimorelin to develop it for the diagnosis of childhood-onset
growth hormone deficiency (CGHD) in collaboration with Novo
Nordisk.
Aeterna Zentaris is dedicated to the development
of therapeutic assets and has recently taken steps to establish a
growing pipeline to address unmet medical needs across a number of
indications, including neuromyelitis optica spectrum disorder
(NMOSD), hypoparathyroidism and ALS (Lou Gehrig’s disease).
Additionally, the Company is developing an oral prophylactic
bacterial vaccine against SARS-CoV-2, the virus that causes
COVID-19.
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 statements that may
constitute forward-looking statements within the meaning of U.S.
and Canadian securities legislation and regulations and such
statements are made pursuant to the safe-harbor provision of the
U.S. Securities Litigation Reform Act of 1995. Forward-looking
statements are frequently, but not always, identified by words such
as “expects,” “anticipates,” “believes,” “intends,” “potential,”
“possible,” and similar expressions. Such statements, based as they
are on current expectations of management, inherently involve
numerous risks, uncertainties and assumptions, known and unknown,
many of which are beyond our control. Forward-looking statements in
this press release include, but are not limited to, those relating
to: the Company’s exclusive right to acquire a license from The
University on the therapeutic use of macimorelin for the treatment
of ALS; the development of macimorelin for the diagnosis of CGHD in
collaboration with Novo Nordisk; the ability of macimorelin to
mimic the biological effect of the hormone via mechanisms similar
to ghrelin; Aeterna’s expectation that, upon receipt of pricing and
reimbursement approvals, macimorelin will be marketed in Europe and
the United Kingdom;
Forward-looking statements involve known and
unknown risks and uncertainties, and other factors which may cause
the actual results, performance or achievements stated herein to be
materially different from any future results, performance or
achievements expressed or implied by the forward-looking
information. Such risks and uncertainties include, among others,
our reliance on the success of the pediatric clinical trial in the
European Union and U.S. for Macrilen™ (macimorelin); the
commencement of Study P02 may be delayed or we may not obtain
regulatory approval to initiate that study, we may be unable to
enroll the expected number of subjects in Study P02 and the result
of Study P02 may not support receipt of regulatory approval in
CGHD, we may be delayed or unsuccessful in obtaining pricing and
reimbursement approvals in Europe and the UK to market macimorelin;
results from ongoing or planned pre-clinical studies of macimorelin
by The University of Queensland or for our other products under
development may not be successful or may not support advancing the
product to human clinical trials; our ability to raise capital and
obtain financing to continue our currently planned operations; 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 Nordisk; the global instability due to the
global pandemic of COVID-19, and its unknown potential effect on
our planned operations; our ability to enter into out-licensing,
development, manufacturing, marketing and distribution agreements
with other pharmaceutical companies and keep such agreements in
effect. Investors should consult our quarterly and annual filings
with the Canadian and U.S. securities commissions for additional
information on risks and uncertainties, including those risks
discussed in our Annual Report on Form 40-F and annual information
form, under the caption "Risk Factors". Given the 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.
No securities regulatory authority has either
approved or disapproved of the contents of this news release. The
Toronto Stock Exchange accepts no responsibility for the adequacy
or accuracy of this release.
Investor Contact:Jenene ThomasJTC TeamT (US):
+1 (833) 475-8247E: aezs@jtcir.com
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