Exhibit
99.1
Aeterna
Zentaris Announces the Start of Preclinical Development of Macimorelin at The University of Queensland as a Potential New Treatment Option
for Patients with Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig’s Disease)
-
Aeterna Zentaris granted exclusive option to negotiate a license of any resulting intellectual property and know-how developed by the
University related to the therapeutic use of macimorelin for the treatment of ALS
CHARLESTON,
S.C., May 17, 2021 — 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
Thomas
JTC
Team
T
(US): +1 (833) 475-8247
E:
aezs@jtcir.com
