Aeterna Zentaris Expands Orphan Drug Development Pipeline with Targeted Immunosuppressive Therapeutics - Seite 2

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 the scientific basis for the concept of AIM Biologicals.

AIM 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 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 potential to 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 University and the University clinic, Aeterna plans to conduct further pre-clinical research to 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.