Myasthenia Gravis

RATIONALE:

Molecular Degraders of Extracellular Proteins (“MoDE™”) are bispecific molecules that target pathologic circulating proteins and direct them to the liver (or other organ systems) for degradation by the endosomal/lysosomal pathway. Hepatic asialoglycoprotein receptor (ASGPR) ligand degraders able to recognize all potentially pathogenic isoforms of IgG represent a novel, competitive platform with a differentiated profile relative to FcRN inhibitors. Specifically, high circulating levels of antibodies (monoclonal or polyclonal gammopathy) drive conditions such as myasthenia gravis, rheumatoid arthritis, systemic lupus erythematosus, pemphigus vulgaris, and many other diseases. It is hypothesized that rapid and sustained lowering of pathogenic antibody titers in blood will significantly reduce disease symptoms. BHV-1310 demonstrates 90% IgG depletion with a single dose. Therapeutic pan-IgG depletion using Biohaven’s proprietary MoDE™ platform technology is expected to have significant potential benefit for multiple diseases.