Full Press Release Details
Zentaris Provides Business Outlook and Outlines Pipeline Priorities
Company advancing diversified portfolio focused on areas of significant unmet medical need
Supported by solid financial position with cash to fund operations beyond 2023
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.
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.
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.
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.
and Clinical Programs Update:
Development Pipeline
Biologicals: Targeted, highly specific autoimmunity modifying therapeutics for the potential treatment of neuromyelitis optica
spectrum disorder ("NMOSD") and Parkinson's disease (PD)
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.
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.
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 na ve T cells.
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 -synuclein-specific T cells in AAV-A53T- -synuclein Parkinson's disease mice, which has recently
been published by University of Wuerzburg researchers.
| Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate. | ||
| Manufacturing process development for selected candidate. |
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. |
Clearance Parathyroid Hormone ("DC-PTH") Fusion Polypeptides: Potential treatment for primary hypoparathyroidism
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.
| 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. |
Therapeutic: Ghrelin agonist in development for the treatment of ALS (Lou Gehrig's disease)
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
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.
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).
| 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. |
Development Pipeline
Diagnostic: Ghrelin agonist in development for diagnostic use in childhood-onset growth hormone deficiency ("CGHD")
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.
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.
Development Pipeline
Vaccine Platform: Potential orally active, live-attenuated bacterial vaccine with application across multiple coronavirus types,
including COVID-19 (SARS-CoV-2) and Chlamydia
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.
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.
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.
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. |