Benzinga | Jan 26, 2022 08:20AM EST

Aeterna 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

TORONTO, ONTARIO, Jan. 26, 2022 (GLOBE NEWSWIRE) -- 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 na?ve 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 ?-synuclein-specific T cells in AAV-A53T-?-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.