Exhibit
99.1
Aeterna
Zentaris Outlines Progress on Development Pipeline Programs and Provides Corporate Update
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Highlights
progress, recent developments and upcoming key milestones |
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Company
continues to build growing body of data across pipeline, advancing towards first in human studies |
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Strong
financial position with cash projected to fund operations and advancement of priority pipeline programs into 2025 |
TORONTO,
ONTARIO, July 13, 2023 – 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 an update on its therapeutic and diagnostic development pipeline programs and outlined upcoming key milestones.
“We
are encouraged by our significant progress across all of our active development programs, and based on the prioritization of our pipeline,
have strengthened our focus on our diagnostic and therapeutic programs as we execute on key decision points,” commented Dr. Klaus
Paulini, Chief Executive Officer of Aeterna. “Additionally, in our ongoing DETECT clinical trial, we are making significant progress
regarding patient recruitment and believe we are well-positioned to continue building momentum towards completion of the study by year’s
end. Further, based upon our active outreach and discussions, we expect to secure an alternate development and commercialization partner
for Macrilen® for the U.S. and Canada.”
Therapeutics
Development Pipeline Update
Autoimmunity
Modifying (“AIM”) Biologicals: Targeted, highly specific autoimmunity modifying fusion proteins for the potential treatment
of neuromyelitis optica spectrum disorder (“NMOSD”) and Parkinson’s disease (“PD”)
AIM
Biologicals represent a unique platform technology with the potential to address the cause and not only the symptoms of auto-immune and
inflammatory, neurodegenerative disorders at early stages. This approach is based on induction of selective and highly target-specific
immune de-sensibilization based on the natural concept of feto-maternal tolerance.
Dr.
Paulini added, “We have received encouraging feedback from our collaborator and scientific advisor on the NMOSD indication, Dr.
Michael Levy, Research Director of the Division of Neuroimmunology & Neuroinfectious Disease at Massachusetts General Hospital. Dr.
Levy shares our vision that presenting antigens on soluble HLA-G fusion proteins is a promising, novel approach to induce antigen specific
immune tolerance among NMOSD and PD patients. Together, we look forward to furthering the development of this program.”
Recent
Highlights
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Demonstrated
positive pre-clinical proof-of-concept in various in-vitro and in-vivo models. |
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In-depth
profiling of a limited set of candidates for potential use in clinical development. |
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Confirmed
AIM Biologicals’ mechanism of action and efficacy in ex-vivo studies in human blood samples from both NMOSD and PD patients. |
Next
Steps
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Evaluation
of manufacturing strategy and initiation of production of selected development candidates. |
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Ongoing
compilation of a comprehensive pre-clinical data package for scientific advice meetings with regulatory authorities, expected to
take place in the near future. |
Delayed
Clearance Parathyroid Hormone (“DC-PTH”, AEZS-150) Fusion Proteins: Potential treatment for chronic hypoparathyroidism
AEZS-150
is a fusion protein of PTH [1-34] coupled to a modified human growth-hormone binding protein (GHBP). Preclinical results so far indicate
the potential for maintaining regular calcium and phosphate levels in blood longer and more consistently than with current treatments.
This project aims to address this debilitating orphan disease, which is currently without satisfactory treatment options, with weekly
instead of daily injections.
“There
is a major unmet need for physiological parathyroid hormone replacement in patients with hypoparathyroidism, either congenital or acquired
from neck surgery, who have an impaired quality of life and increased morbidity and mortality on current therapy. DC-PTH will address
this unmet need,” commented Prof. Richard Ross, MD, FRCP, Department of Oncology and Metabolism at Sheffield University (UK). “Aeterna
has made great progress in the manufacturing process development of AEZS-150 and repeating validation studies in models of hypoparathyroidism
required before undertaking trials in patients.”
Recent
Highlights
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Detailed
profiling of AEZS-150 through in-vitro studies and in-vivo models of hypoparathyroidism. |
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Established
a master cell bank for a cell line expressing AEZS-150 in good yield. |
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Continued
progress in the development of a production process suitable for larger scale GMP manufacturing. |
Next
Steps
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Meet
with regulatory authorities to determine the development path forward; Company expects such a meeting in Q4/2023, and as outcome,
an abbreviated safety and toxicology program as the active principle ([1-34]-PTH fragment) is already approved and in use. |
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Initiate
IND-enabling preclinical studies in 2024. |
Macimorelin
Therapeutic (AEZS-130): Ghrelin agonist in development for the treatment of ALS (Lou Gehrig’s disease)
Amyotrophic
Lateral Sclerosis (ALS) is a debilitating and deadly orphan disease, and those who suffer from this terminal condition have few treatment
options. Macimorelin’s (AEZS-130) multiple modes of action should have the potential to improve the quality of life for these patients.
Recent
Highlights
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Successful
development of an alternative formulation suitable for use in ALS. |
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Accumulating
data for positive effects of AEZS-130 treatment on survival of motor-neurons. |
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Continued
assessment of AEZS-130 in transgenic mouse ALS models and in human patient-derived neuron cultures. |
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Completion
of initial toxicology and safety studies to support clinical development as a therapeutic, in addition to the already existing body
of safety and toxicology data from our macimorelin diagnostic development. |
Next
Steps
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Following
achievement of proof-of-concept, the Company will seek to have a scientific advice meeting with regulatory authorities to discuss
program development next steps. |
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Completion
of toxicology and safety studies to support treatment over prolonged periods. |
Diagnostics
Development and Commercialization Update
Macimorelin
Diagnostic: Approved and commercialized as test for adult growth hormone deficiency and in clinical Phase 3 development for childhood-onset
growth hormone deficiency (“CGHD”) testing.
Aeterna
is currently conducting its pivotal Phase 3 safety and efficacy study AEZS-130-P02 (the “DETECT-trial”) evaluating macimorelin
for the diagnosis of Childhood Onset Growth Hormone Deficiency (“CGHD”). Most clinical sites in the U.S. as well as European
countries are open for patient recruitment.
Recent
Highlights
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Phase
3 Study: Significant progress made with DETECT patient recruitment also by engagement of an additional Clinical Research Organization
(CRO). Currently three new countries (Armenia, Slovakia, and Turkey), have ongoing DETECT clinical trial application activities and
almost half the foreseen subjects have meanwhile concluded the trial. |
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Macimorelin
Commercialization: Having regained full rights to Macrilen® for the U.S. and Canada from Novo Nordisk in May 2023,
the Company is actively focused on identifying an alternate development and commercialization partner for Macrilen®
for U.S. and Canada. |
“We
continue to manage our cash responsibly with a balanced de-risked development approach, which we believe enables us to optimize the Company’s
capital and human resources,” added Giuliano La Fratta, Chief Financial Officer of Aeterna. “We remain focused on advancing
our programs to their next phase of development, guided by the data and financial risk/reward as we do so. Based on our current projections,
we believe the Company has the ability to meet its currently anticipated cash needs into 2025.”
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®;
Ghryvelin™), 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.
Aeterna
Zentaris is dedicated to the development of its therapeutic asset and has established a pre-clinical development 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).
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,” “aiming”, “anticipates,”
“believes,” “intends,” “potential,” “possible,” and similar expressions. Such statements,
based as they are on current expectations of management, inherently involve numerous risks, uncertainty 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 regarding: its preclinical
and clinical studies, its ability to secure regulatory approvals for Macrilen™, its efforts to obtain a development and commercialization
partner for Macrilen™ in the U.S. and Canada and the ability to meet its currently anticipated cash needs into 2025.
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 DETECT clinical trial in the European
Union and U.S. for Macrilen™ (macimorelin) in CGHD; results from our ongoing or planned pre-clinical studies and our DETECT clinical
trial under development may not be successful or may not support advancing the product further in pre-clinical studies, to human clinical
trials or regulatory approval; 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; the global instability due to the global pandemic of COVID-19 and the
war in the Ukraine, and their 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 20-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