Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS) (“Aeterna” or the
“Company”), a specialty biopharmaceutical company developing and
commercializing a diversified portfolio of pharmaceutical and
diagnostic products, today provided a corporate update and business
outlook.
“Over the course of 2021, we in-licensed six new
pre-clinical development programs, four potential therapeutics and
two potential vaccines, all of which were added to our development
pipeline based on their potential to represent significant
individual market opportunities,” commented Dr. Klaus Paulini,
Chief Executive Officer of Aeterna.
“We continue to make progress and are working to
advance all of our pre-clinical and clinical programs. With regards
to our Phase 3 DETECT clinical trial of macimorelin for diagnostic
use in childhood-onset growth hormone deficiency, we have
experienced unavoidable delays in site initiation and patient
enrollment due to rise of the Omicron variant in the COVID-19
pandemic. Our team is diligently working to get more clinical sites
up and running with the goal of building momentum and bringing this
study across the finish line while navigating as best as possible
through this challenge,” added Dr. Paulini.
“Our cash position remains an important strength
for Aeterna, which enables us to fully focus on our development and
strategic priorities. Our management team is committed to driving
our programs forward with the goal of transitioning our
pre-clinical assets towards in-human clinical studies. We believe
2022 will be an important year for the Company and a significant
step forward towards realizing the value of our pipeline and
unlocking value for our shareholders,” concluded Dr. Paulini.
Pre-Clinical and Clinical
Programs Update:
Therapeutics Development Pipeline
AIM Biologicals: Targeted,
highly specific autoimmunity modifying therapeutics for the
potential treatment of neuromyelitis optica spectrum disorder
(“NMOSD”) and Parkinson’s disease (PD)
Aeterna entered into an exclusive patent license
and research agreement with Julius-Maximilians-University of
Wuerzburg in January and September 2021 to develop AIM Biologicals
for the potential treatment of NMOSD and PD, respectively.
AIM Biologicals (Auto-Immunity Modifying
Biologicals) utilize a novel mechanism which is believed to
demonstrate that peptide antigens presented on immunosuppressive
MHC class I molecules can selectively and efficiently induce
antigen-specific tolerance. Based on this mechanism, the targeted
immunomodulating therapeutics are being designed as optimized
soluble molecules with the goal that they may be adapted to
selectively induce tolerance to various autoantigens.
During pregnancy, the maternal immune system
tolerates paternal antigens from the embryo but is still effective
to protect mother and embryo from foreign pathogens. Parts of the
natural mechanisms responsible for this feto-maternal immune
tolerance form the scientific basis for the concept of AIM
Biologicals. Pre-clinical studies conducted by the
Julius-Maximilians-University thus far indicate that tolerance
induction appears to be achieved via selective elimination of
antigen-specific immune effector cells and via induction of
antigen-specific regulatory T cells from naïve T cells.
The Company has entered into a Research
Agreement under which the Company has engaged the University of
Wuerzburg on a fee-for-service basis to conduct research activities
and pre-clinical development studies on the AIM Biologicals, the
results of which are covered within the scope of the license
agreements. Additionally, Prof. Dr. Joerg Wischhusen of the
University of Wuerzburg was engaged by the Company as a scientific
consultant to support development of this new class of potential
therapeutics. Michael Levy, MD, PhD, Research Director of the
Division of Neuroimmunology & Neuroinfectious Disease at
Massachusetts General Hospital ("MGH") was also retained as a
scientific consultant. Dr. Levy will provide scientific support,
animal model testing and advice to Aeterna Zentaris in the field of
inflammatory CNS disorders, autoimmune diseases of the nervous
system, and NMOSD.
For the development of AIM Biologicals as
potential PD therapeutics, Aeterna plans to utilize, among others,
an innovative animal model on neurodegeneration by
α-synuclein-specific T cells in AAV-A53T-α-synuclein Parkinson’s
disease mice, which has recently been published by University of
Wuerzburg researchers.
Next Steps –
NMOSD
- Conduct in-vitro and in-vivo
assessments to select an AIM Biologicals-based development
candidate.
- Manufacturing process development
for selected candidate.
Next Steps – Parkinson’s
Disease
- Design and produce antigen-specific
AIM Biologics molecules for the potential treatment of Parkinson’s
disease.
- Conduct in-vitro and in-vivo
assessments in relevant Parkinson’s disease models.
Delayed Clearance Parathyroid Hormone
(“DC-PTH”)
Fusion Polypeptides: Potential treatment for primary
hypoparathyroidism
In March 2021, Aeterna entered into an exclusive
patent and know-how license agreement and research agreement with
The University of Sheffield, United Kingdom, for the intellectual
property relating to DC-PTH fusion polypeptides with delayed
clearance for all human uses. In consultation with The University
of Sheffield, Aeterna has selected AEZS-150 as the lead candidate
in its DC-PTH program. AEZS-150 is being developed with the goal of
providing a potential new treatment option of primary
hypoparathyroidism in adults.
Next Steps
- Work with The University of
Sheffield to conduct in depth characterization of development
candidate (in-vitro and in-vivo).
- Develop manufacturing process.
- Formalize pre-clinical development
of AEZS-150 in preparation for a potential IND filing for
conducting the first in-human clinical study.
Macimorelin Therapeutic:
Ghrelin agonist in development for the treatment of ALS (Lou
Gehrig’s disease)
In January 2021, the Company entered into a
material transfer agreement with the University of Queensland,
Australia, to provide macimorelin for the conduct of pre-clinical
and subsequent clinical studies evaluating macimorelin as a
potential therapeutic for the treatment of ALS (Lou Gehrig’s
disease). The University of Queensland researchers have filed for
supportive grants and aim to conduct pre-clinical studies in
multiple pre-clinical models to demonstrate the therapeutic
potential of macimorelin to slow disease progression and
disease-specific pathology.
Macimorelin, a potent ghrelin agonist, is an
orally active small molecule that stimulates the secretion of
growth hormone from the pituitary gland. Acting via this mechanism,
which was established during the development as a diagnostic test
for growth hormone deficiency, it is believed that macimorelin may
slow the progression of certain neurodegenerative diseases like
ALS.
Apart from already available pre-clinical and
clinical data on macimorelin for the development as a diagnostic,
Aeterna may utilize the established supply chain to support this
development. Alternative formulations are currently also under
development, as a further option in addition to the existing oral
solution already approved for the diagnostic use in adult growth
hormone deficiency (AGHD).
Next Steps
- Work with The University of
Queensland to conduct proof-of-concept studies with macimorelin in
disease-specific animal models.
- Assess alternative
formulations.
- Formalize pre-clinical development
plan.
Diagnostics Development Pipeline
Macimorelin
Diagnostic: Ghrelin agonist in development for
diagnostic use in childhood-onset growth hormone deficiency
(“CGHD”)
Aeterna is currently conducting its pivotal
Phase 3 safety and efficacy study AEZS-130-P02 (the “DETECT-trial")
evaluating macimorelin for the diagnosis of CGHD. Children and
adolescents from two to less than 18 years of age with suspected
growth hormone deficiency are to be included. The study is expected
to include approximately 100 subjects in Europe and North America,
with at least 40 subjects in pre-pubertal and 40 subjects in
pubertal status. Macimorelin growth hormone stimulation test
(“GHST”) will be performed twice for repeatability data and two
standard GHSTs will be used as controls: arginine (i.v.) and
clonidine (p.o.). On April 22, 2021, the U.S. FDA Investigational
New Drug Application associated with this clinical trial became
active. The first clinical sites in the U.S. and in Europe are open
for patient recruitment. In Europe, national clinical trial
approval procedures and site initiation activities are ongoing. At
this point in time, we are closely monitoring delays in site
activation and enrollment due to the ongoing COVID-19 pandemic, to
mitigate potential impact on estimated trial completion dates.
The Company continues to advance its ongoing
business development discussions to secure commercialization
partners for macimorelin in additional markets. In addition to its
previously established agreements, Aeterna recently entered into a
license agreement with NK Meditech Ltd., for the development and
commercialization of macimorelin in the Republic of Korea, and a
distribution agreement with Er-Kim Pharmaceuticals Bulgaria EOOD
for the commercialization of macimorelin in Turkey and some Balkan
countries.
Vaccine Development Pipeline
Salmonella-Based
Vaccine Platform: Potential
orally active, live-attenuated bacterial vaccine with application
across multiple coronavirus types, including COVID-19 (SARS-CoV-2)
and Chlamydia
In February 2021, Aeterna entered into an
exclusive option agreement with the University of Wuerzburg to
evaluate a pre-clinical, potential COVID-19 vaccine developed at
the University of Wuerzburg. In March 2021, the Company exercised
its option and entered into a license agreement where the Company
was granted an exclusive, world-wide, license to certain patent
applications and know-how owned by the University of Wuerzburg to
research and develop, manufacture, and sell a potential COVID-19
vaccine. The Company’s vaccine platform is currently undergoing
pre-clinical studies for the prevention of coronavirus diseases,
including COVID-19 (SARS-CoV-2) with the planned start of clinical
development targeted for H1 2023.
In September 2021, the Company exercised its
option under the agreement with the University of Wuerzburg on a
then undisclosed field, now known to be Chlamydia. Chlamydia
trachomatis is a sexually transmitted bacterium infecting over 130
million subjects annually. Asymptomatic disease can spread to the
reproductive tract eventually inducing infertility, miscarriage, or
ectopic pregnancy, which is a life-threatening condition. Ocular
infections can lead to inclusion conjunctivitis or trachoma, which
is the primary source of visual impairment or infectious
blindness.
Additionally, the Company has entered into a
Research Agreement under which the Company has engaged the
University of Wuerzburg on a fee-for-service basis to conduct
supplementary research activities and pre-clinical development
studies on the potential vaccines, the results of which are covered
within the scope of the license agreements. Additionally, Prof. Dr.
Thomas Rudel of the University of Wuerzburg was engaged by the
Company in September 2021 as a scientific consultant to support
development of the salmonella-based vaccine platform for the
coronavirus and Chlamydia vaccines.
Next Steps –
Coronavirus
Vaccine
- Evaluate
administration route, dose and immunization scheme.
- In-vivo
immunology experiments with antigen variant candidates in relevant
mice models.
- Conduct virus
challenge experiments in immunized transgenic animals.
- Start
manufacturing process assessment / development.
- Conduct
pre-clinical safety and toxicology assessment.
Next Steps –
Chlamydia Vaccine
- Design and
prepare candidate vaccine strains.
- Evaluate
administration route, dose and immunization scheme.
- In-vivo
immunology experiments with candidate strains in relevant mouse
models.
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 (Macrilen™), 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), an area of significant unmet
need, in collaboration with Novo Nordisk.
Aeterna Zentaris is dedicated to the development
of therapeutic assets and has recently taken steps to establish a
growing pre-clinical pipeline to potentially address unmet medical
needs across a number of indications, including neuromyelitis
optica spectrum disorder (NMOSD), Parkinson's disease (PD),
hypoparathyroidism and amyotrophic lateral sclerosis (ALS; Lou
Gehrig's disease). Additionally, the Company is developing an oral
prophylactic bacterial vaccine against SARS-CoV-2 (COVID-19) and
Chlamydia.
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: Aeterna’s expectations with respect to the DETECT-trial
(including regarding the enrollment of subjects in the
DETECT-trial, the application of the Macimorelin growth hormone
stimulation tests and the completion of the DETECT-trial);
Aeterna’s expectations regarding conducting pre-clinical research
to identify and characterize an AIM Biologicals-based development
candidate for the treatment of NMOSD as well as Parkinson’s
disease, and developing a manufacturing process for a selected
candidates; Aeterna’s expectations regarding conducting assessments
in relevant Parkinson’s disease models; The University of
Queensland undertaking a subsequent investigator initiated clinical
trial evaluating macimorelin as a potential therapeutic for the
treatment of ALS and Aeterna formulating a pre-clinical development
plan for same; the commencement of Aeterna’s formal pre-clinical
development of AEZS-150 in preparation for a potential IND filing
for conducting the first in-human clinical study of AEZS-150;
Aeterna’s plans to perform challenge experiments, select a
development candidate, start clinical development and establish a
manufacturing process for the orally active COVID-19 (SARS-CoV-2)
and Chlamydia live-attenuated bacterial vaccine.
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 the DETECT-trial 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 the DETECT-trial and
the result of the DETECT-trial may not support receipt of
regulatory approval in CGHD; the coronavirus vaccine platform
technology (and any vaccine candidates using that technology)
licensed from the University of Wuerzburg has never been tested in
humans and so further pre-clinical or clinical studies of that
technology and any vaccine developed using that technology may not
be effective as a vaccine against COVID-19 (SARS-CoV-2) or any
other coronavirus disease; the timeline to develop a vaccine may be
longer than expected; such technology or vaccines may not be
capable of being used orally, may not have the same characteristics
as vaccines previously approved using the Salmonella Typhi Ty21a
carrier strain; 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 and the amended license agreement
(collectively the Novo Amended License Agreement); 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; and our ability to continue to list
our common shares on the NASDAQ. 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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