Inebilizumab is a humanised monoclonal antibody that is in clinical development for reducing the risk of an attack in patients with Neuromyelitis Optica Spectrum Disorders (NMOSD). NMOSD, previously known as NMO and as Devic’s disease, is a rare, disabling autoimmune disease of the central nervous system. It predominantly affects the optic nerve and spinal cord, however involvement of the brain can occur. Patients with NMOSD present with acute, often severe attacks that lead to blindness, limb weakness or paralysis, difficulty controlling the bladder or bowels, fatigue, pain, and other manifestations.
The main objective of current treatments is to reduce the rate of acute NMOSD attacks. This is the most important goal in treating these patients. There are no approved attack-preventing therapies and different off label immunosuppression medications are being used with low level of evidence for efficacy. Inebilizumab is an experimental therapy that specifically targets B lymphocytes which are the source of the pathogenic auto antibody in this disease, the AQP4-IgG. Depleting B cells by Inebilizumab will potentially reduce risk of NMOSD attack. Inebilizumab binds to and depletes CD19+ B-cells including plasmablasts and plasma cells. These CD19+ plasmablasts are the type of B cells that produce the AQP4-IgG which in turn acts against a key water channel protein expressed on astrocytes in the central nervous system. If licensed, inebilizumab may offer a first line disease-modifying treatment option for patients with NMOSD.
Edaravone as an intravenous injection is in clinical development for people with amyotrophic lateral sclerosis (ALS). ALS is a neurological condition that affects nerve cells in the brain and
spinal cord. It results in gradual weakness and wasting of muscles of the body. Respiratory muscles are involved as the disease progresses, leading to shortness of breath and ultimately
death. Little is known about the cause of the disease, and there is currently no cure.