Exhibit
99.1
Aeterna
Zentaris Provides Business Outlook and Outlines Pipeline Priorities
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Company advancing diversified portfolio focused on areas of significant unmet medical need
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Supported by solid financial position with cash to fund operations beyond 2023
TORONTO,
ONTARIO, January 26, 2022 – 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
Thomas
JTC
Team
T
(US): +1 (833) 475-8247
E:
aezs@jtcir.com
