N-MOmentum Phase II/III pivotal trial of Uplinza demonstrates reduced severity of Neuromyelitis Optica Spectrum Disorder.

Treatment of NMOSD aims to reduce acute attacks associated with the disease, which can cause irreversible damage to the optic nerve, spinal cord, brain and brain stem. During the 28-week randomized-controlled period (RCP) of the N-MOmentum Phase II/III pivotal trial, 89% of 161 patients in the Uplizna treatment group remained attack-free compared to 58% of 52 patients in the placebo group.

A new post hoc analysis of this data was conducted to understand the effect of Uplizna on the severity of attacks in the 11% of people in the clinical trial who were not attack-free after being treated with Uplizna. Attacks were graded as major or minor based on changes in neurological function according to a modified Opticospinal Impairment Scale (OSIS). The analysis also evaluated the relationship between the severity of these attacks and biomarkers of disease activity, such as serum glial fibrillary acidic protein (sGFAP) and serum neurofilament (sNfL). Key analysis findings show that of the 18 total attacks that occurred in the Uplizna treatment group during the RCP, 12 (67%) were minor and six (33%) were major, compared to 12 (55%) minor attacks and 10 (45%) major attacks among the 22 attacks occurring in the placebo group. Levels of the sGFAP biomarker were significantly higher during major attacks versus minor attacks overall (p=0.023) and trended higher for optic neuritis (ON) specific attacks (n=20, p=0.06). Concentration levels of the biomarker increased significantly from baseline at the time of attacks in those receiving placebo but not in those treated with Uplizna (p=0.31). SNfL levels were higher for major versus minor attacks overall (p=0.032), though the levels did not correlate with the severity of ON attacks. In participants that had attacks during the RCP, rates of this marker were higher among placebo versus Uplizna-treated participants at Week 26 (p=0.03).

These data are being presented at the 48th Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS 2022), February 12-17 in Austin, Texas. Uplizna is the first and only B-cell-depleting agent approved by the FDA for the treatment of NMOSD in adults who are anti-aquaporin-4 (AQP4) antibody positive.