Rare Daily Staff
Viela Bio reported that a pivotal trial of its experimental drug inebilizumab met its primary and key secondary endpoints in patients with neuromyelitis optica spectrum disorder (NMOSD), a rare, severe, relapsing, neuroinflammatory autoimmune disease that can be fatal.
In about 80 percent of NMOSD cases, patients have autoantibodies to a water channel protein called aquaporin-4 (AQP4). These AQP4- IgG autoantibodies bind primarily to astrocytes in the central nervous system and are believed to trigger attacks, which can damage the optic nerves, spinal cord, and brain. Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain, and respiratory failure can all be manifestations of the disease. Each NMOSD attack leads to further damage and disability. There is currently no cure or approved treatment for NMOSD.
Inebilizumab is a humanized monoclonal antibody that binds with high affinity to CD19, a protein expressed on a broad range of B cells. After binding to CD19, these cells are rapidly depleted from the circulation.
The results showed a 77 percent reduction in the risk of developing an NMOSD attack in patients treated with inebilizumab monotherapy compared to placebo. The study also demonstrated a reduction in worsening of disability in patients treated with inebilizumab compared to placebo. The company said the safety and tolerability profiles for inebilizumab were acceptable and consistent with previous experience. Additional details from the trial will be presented at a medical meeting in 2019.
“These results support our hypothesis that CD19 expressing B cells including plasmablasts and plasma cells play a key role in the pathogenesis of NMOSD,” said Jorn Drappa, chief medical officer and head of research and development at Viela Bio. “This study demonstrated a highly significant and clinically meaningful reduction in attack risk and suggests a promising new treatment for patients diagnosed with NMOSD.”
The U.S. Food and Drug Administration granted Orphan Drug designation for inebilizumab for the treatment of patients with NMOSD in March 2016. The European Medicines Agency granted orphan designation to inebilizumab for the treatment of NMOSD in March 2017.
January 4, 2019
Photo: Jorn Drappa, chief medical officer and head of research & development at Viela Bio