Uplizna Reduced Disability Progression in Patients During Trial
The results were published in Neurology Neuroimmunology & Neuroinflammation, in a study, “Disability Outcomes in the N-MOmentum Trial of Inebilizumab in Neuromyelitis Optica Spectrum Disorder,” funded by Viela Bio (now part of Horizon Therapeutics), which markets Uplizna.
Uplizna is an antibody-based therapy designed to kill B-cells, a type of immune cell that drives damaging inflammation in NMOSD and other autoimmune diseases. The U.S. Food and Drug Administration approved Uplizna to treat adults with NMOSD who test positive for aquaporin-4 water channel autoantibodies (AQP4-IgG) last June.
The approval was supported by data from the N-MOmentum clinical trial (NCT02200770), which demonstrated that the treatment reduced the risk of relapses in individuals with AQP4-IgG-positive NMOSD.
In the new study, researchers reported on how the treatment affected disability progression during the trial.
In N-MOmentum, 174 adults with NMOSD were randomized to treatment with Uplizna — given by infusion into the bloodstream on trial days one and 15 — and the remaining 56 participants were given placebo. The participants’ mean age was 42.9 years, and over 90% were female.
In the first part of the study, participants were followed for up to 28 weeks (about six months), or until they had a confirmed NMOSD attack. At that point, they were allowed to enroll in an open-label extension part of the study, wherein all patients were treated with Uplizna.
The researchers assessed disability outcomes using two standardized measurements: the Expanded Disability Status Scale (EDSS) and the modified Rankin Scale (mRS).
Results demonstrated that after the first 28-week part of the trial, a significantly lower proportion of participants given Uplizna than placebo experienced a worsening of EDSS score (15.5% vs. 33.9%).
Post hoc analyses (statistical tests conducted after the trial had finished) showed that participants treated with Uplizna were significantly less likely to experience worsening disability. For example, the risk of confirmed disability worsening after three months was about 62.5% lower with Uplizna, compared with placebo.
The analyses also demonstrated that the medication’s benefits were not significantly affected by pre-treatment EDSS scores, disease duration, and number of previous attacks.
“We found that the impact of inebilizumab [Uplizna] treatment was not influenced by baseline [at the trial’s start] participant characteristics,” the researchers wrote.
Outcomes on the mRS also favored Uplizna over placebo. After the first 28-week part of the trial, individuals treated with Uplizna were 66.3% more likely to report lessened disability, compared with those given placebo.
Before starting treatment, 46.6% of participants given Uplizna had no significant disability, based on mRS scores. After 28 weeks, this proportion stayed about the same (48.3%). By contrast, the proportion decreased markedly among participants given placebo, from 41.1% to 33.9%.
“These analyses demonstrated that compared with placebo, inebilizumab improves disability outcomes in individuals with NMOSD,” the researchers concluded.
In the open-label extension portion of the trial, where all participants got Uplizna, the rates of disability worsening among participants initially given placebo decreased markedly. However, disability scores still tended to worsen more quickly among participants initially given placebo, even after they had started on active treatment.
This finding indicates “that even a 6-month delay in initiation of treatment may persistently affect disability,” the researchers wrote.