Full Press Release Details
Zentaris Reports First Quarter 2022 Financial Results and Provides Business Outlook
Continued advancement across diversified development pipeline
Company ended the quarter with $63.6 million in cash, expected to fund operations through 2023
ONTARIO, May 11, 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 reported its financial and operating results for the first quarter ended March 31, 2022. The Company also provided an update on
its pre-clinical and clinical development programs.
are beginning to see tangible progress related to the development programs we in-licensed throughout 2021. In particular, we are very
pleased to report data with our pre-clinical program for AIM biologicals, which have recently been accepted for presentation at two scientific
conferences. We remain focused advancing our pipeline, executing on our strategic priorities and unlocking the full potential of our
pipeline and value for all stakeholders," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna.
| Presented results from pre-clinical studies of Aeterna's AIM Biologicals (Autoimmunity Modifying Biologicals) for the potential treatment of Parkinson's Disease ("PD") at IMMUNOLOGY2022 , the Annual Event of the American Association of Immunologists, held May 6-10, 2022. | ||
| Secured new European patent providing intellectual property protection of macimorelin in 27 countries within the European Union as well as additional European non-EU countries, such as the UK and Turkey, for macimorelin (Ghryvelin ; Macrilen ) for use to diagnose growth hormone deficiency (GHD) in adults. | ||
| Announced that results from pre-clinical studies of Aeterna's AIM Biologicals for the potential treatment of neuromyelitis optica spectrum disorder ("NMOSD") were accepted for presentation at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens, Greece. |
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)
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. With AIM Biologicals, the Company aims to restore the tolerance against such proteins to treat autoimmune diseases.
studies conducted by the University of Wuerzburg, Germany 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.
AIM Biologicals thus have the potential to become highly specific and effective yet not personalized treatments of NMOSD. Data from the
pre-clinical studies will be presented at the 13th International Congress on Autoimmunity to be held June 10-13, 2022 in Athens,
the treatment of NMOSD, it is believed that the AIM Biologicals will present a specific antigen derived from the water channel protein
aquaporin-4 (AQP4) loaded to soluble immunoregulatory HLA-G protein to selectively induce immunological tolerance in the central nervous
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. Additionally, the Company recently presented pre-clinical data demonstrating that
corresponding AIM Biologicals prevented mobility impairments and postmortem histopathological assessment confirmed the induction of favorable
in-situ immune cell composition and the rescue of substantia nigra neurons. The pre-clinical data confirmed that the translation potential
of the approach deserves further exploration.
University of Wuerzburg continues to bolster its intellectual property (IP) protection and has filed new IP on AIM-Biologicals related
to both NMOSD and PD.
| Conduct in-vitro and in-vivo assessments to select an AIM Biologicals-based development candidate. | ||
| Manufacturing process development for a 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 for primary hypoparathyroidism in adults.
Company has selected a contract manufacturing organization for the development of its manufacturing for AEZS-150.
| Work with The University of Sheffield to conduct in depth characterization of development candidate ( in-vitro and in-vivo ). | ||
| Ongoing development of 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).
| Continue investigating macimorelin efficacy in an ALS specific SOD1 mouse model. | ||
| Assess alternative formulations. | ||
| Formalize pre-clinical development plan. |
Diagnostics 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.
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.).
April 22, 2021, the U.S. FDA Investigational New Drug Application associated with this clinical trial became active.
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. Site activation and patient enrollment continues to be impacted by the ongoing COVID-19 pandemic.
The Company is actively monitoring delays to mitigate potential impact of COVID-19 on estimated trial completion dates. Additionally,
clinical trial sites originally planned in the Ukraine and Russia are being halted due to the conflict in Ukraine intensifying following
the Russian invasion. As a result, further delays with enrollment are expected as the DETECT-trial planned to recruit at least 25% (25
subjects) within those countries. Due to these circumstances and the resulting feasibility data from the Company's CRO on potential
options, Aeterna believes recruitment for the DETECT-trial may now continue until later into 2023.
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: Orally active, live-attenuated bacterial vaccine platform with potential application against viruses and
bacteria, such as 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. 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
the Company expanded its research agreement with the University of Wuerzburg 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. Under the
expanded research program, the University of Wuerzburg will validate and utilize innovative human 3D intestinal tissue models to study
the infection biology of Salmonella strains towards clinical development.
Steps - Coronavirus Vaccine
| Evaluate administration route, dose and immunization scheme. | ||
| Initiate 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. |
Steps - Chlamydia Vaccine
| Design and prepare candidate vaccine strains. | ||
| Evaluate administration route, dose and immunization scheme. | ||
| Conduct In-vivo immunology experiments with candidate strains in relevant mouse models. |
of First Quarter 2022 Financial Results