Aeterna Zentaris Expands Orphan Drug Development Pipeline with Targeted Immunosuppressive Therapeutics -Company licenses exclusive worldwide rights to develop, manufacture and commercialize targeted, highly specific immunosuppressive therapeutic proteins for the potential treatment of neuromyelitis optica spectrum disorder(“NMOSD”) from Julius-Maximilians-University of Wuerzburg, Germany-Initial step in growth strategy to build-out pipeline of assets -Aeterna Zentaris to develop potential therapeutic treatment option for neuromyelitis optica spectrum disorder (“NMOSD”), an orphan indication with strong unmet medical need and significant market opportunity CHARLESTON, S.C., January 28, 2021--Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZS), through its wholly-owned subsidiary Aeterna Zentaris GmbH, (“Aeterna” or the “Company”), a specialty biopharmaceutical company commercializing and developing therapeutics and diagnostic tests,today announced that it has licensed the exclusive worldwide rights to develop, manufacture and commercialize targeted, highly specific,autoimmunity modifying proteins (“AIM Biologicals”)for the potential treatment of neuromyelitis optica spectrum disorder(“NMOSD”) from the Julius-Maximilians-University Wuerzburg (the “University”). “This demonstrates Aeterna’scommitment to execute on our stated plans of expanding our pipeline to have multiple assets in research and development. The work that Dr. Valentin Bruttel and Prof. Joerg Wischhusen have conducted at the University represents what we believe to be a compelling opportunity for innovative development in the high-value indication NMOSD, an orphan indication with significant unmet need,” commented Dr. Klaus Paulini, Chief Executive Officer of Aeterna.Prof. Joerg Wischhusenof the University added, “Based on pre-clinical data obtained by the University to date, the AIM Biologicals technology has the potential to be a breakthrough in the treatment of autoimmune-related diseases. It is based on a mechanism developed by nature to protect the fetus from the mother’s immune system without compromising immune protection against foreign antigens. This has the potential to offer a new treatment for NMOSD patients. We believe that the collaboration with Aeternawill accelerate the further development towards the clinic.” Autoimmunity Modifying (“AIM”) Biologicals -Targeted Immunosuppressive Therapeutics
During pregnancy, the maternal immune system tolerates paternal antigens from the embryo but is still effective to protect mother and embryo from foreign antigens. Parts of the natural mechanisms responsible for this feto-maternal immune tolerance form thescientific basis for the concept of AIM Biologicals.AIM Biologicals utilize a novel mechanism which isbelieved 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 immunosuppressive therapeutics are being designed as optimized soluble molecules with the goal that they may be adapted to selectively induce tolerance to various autoantigens. Pre-clinical studies conducted by the 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 naïve T cells. AIM Biologicals thus have the potentialto become highly specific and effective yet not personalized treatments of NMOSD.For 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 system. In collaboration with the Universityand the University clinic, Aeterna plans to conduct further pre-clinical researchto identify and characterize an AIM Biologicals based development candidate for the treatment of NMOSD, including meeting with the regulatory authorities to confirm the further pre-clinical data required as we work towards advancing such candidate into human clinical trials.About Neuromyelitis Optica Spectrum Disorder (NMOSD)NMOSD is an antibody mediated inflammatory central nervous system (“CNS”) disorder that affects about one per million population per year. NMOSD, also known as Devic disease, is a chronic disorder of the brain and spinal cord dominated by inflammation of the optic nerve (optic neuritis) and of the spinal cord (myelitis). Typical symptoms include visual loss, muscle spasms, paraparesis, and incontinence. If left untreated, 50% of individuals with NMOSD will be wheelchair bound and blind, and 30% will have died within five years after the first attack. The water channel protein AQP4 is widely expressed in the brain, spinal cord, and optic nerves. Auto-antibodies directed against the AQP4 channel play an important role in the pathogenesis of NMOSD.Currently there are only three approved medications available for the treatment of NMOSD with very high annual treatment costs, and the risk of the patient contracting serious infections. Therefore, there is a strong medical need to offer new therapeutic options to the patients. |
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