Full Press Release Details
Zentaris Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Business Outlook
Continue to advance and expand opportunities with established diversified development pipeline
- Company ended the year with
$65.3 million in cash, expected to fund operations through 2023
ONTARIO, March 29, 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 year ended December 31, 2021. The Company also provided an update on progress
in its pre-clinical and clinical development programs.
the in-licensing of six new pre-clinical development programs, 2021 was a transformational year for Aeterna Zentaris. As we work to make
a positive impact across multiple therapeutic areas with unmet needs, our team is executing on all fronts to advance these pre-clinical
assets toward in-human clinical studies," commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna. "Since
the start of 2022, we have continued to build momentum and position ourselves to unlock the full potential of our pipeline and value
for all stakeholders. Bolstered by our cash position, which provides us with the funding for operations through 2023, we are laser focused
on our development and strategic priorities."
| Expanded research program with Julius-Maximilians-University Wuerzburg aimed to accelerate development of vaccine programs on COVID-19 and Chlamydia | ||
| Announced Notice of Allowance for U.S. patent covering AEZS-150 for the potential treatment of chronic hypoparathyroidism. | ||
| Appointed Giuliano La Fratta as Chief Financial Officer. |
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)
January 2021, Aeterna entered into an exclusive patent license and research agreement with the University of Wuerzburg, Germany, for
worldwide rights to develop, manufacture, and commercialize AIM Biologicals for the potential treatment of NMOSD. Additionally, the Company
has engaged Prof. Dr. Joerg Wischhusen from the University Hospital in Wuerzburg as well as neuro-immunologist Dr. Michael Levy from
the Massachusetts General Hospital in Boston as consultants for scientific support and advice in the field of inflammatory CNS disorders,
autoimmune diseases of the nervous system, and NMOSD. In September 2021, the Company entered into an additional exclusive license with
the University of Wuerzburg for early pre-clinical development towards the potential treatment of Parkinson's disease.
Biologicals (Auto-Immunity Modifying Biologicals) is based on a natural process during pregnancy, which induces immunogenic tolerance
of the maternal immune system to the partially foreign fetal antigens. Fetal proteins are processed and presented on certain immunosuppressive
major histocompatibility complex class I molecules to induce this tolerance. In an autoimmune disease is the immune system misdirected
and targeting the body's own protein. With AIM Biologicals, the Company aims to restore the tolerance against such proteins to
treat autoimmune diseases.
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.
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).
| 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.
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 Fourth Quarter and Full Year 2021 Financial Results
amounts are in U.S. dollars
and cash equivalents