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Biomarker analysis publication highlights key signals of disability worsening associatedw ith Neuromyelitis Optica Spectrum Disorder (NMOSD) Attacks, Illustrates Efficacy of UPLIZNA

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Published:2nd Jun 2023

Horizon Therapeutics plc announced the publication of new data from the Phase III N-MOmentum pivotal trial of Uplizna, which offer insights on serum biomarkers that signal acute attacks and disability worsening associated with NMOSD and illustrate the role of Ulip in reducing these biomarker levels, potentially reducing the frequency and severity of these attacks

The results of this analysis are published in The Journal of Neurology, Neurosurgery and Psychiatry.

NMOSD is most notably associated with acute attacks, which can cause irreversible damage to the optic nerve, spinal cord, brain and brain stem. Disease management goals are focused on prevention of attacks, as well as understanding and tracking biomarkers that could signal these attacks. The N-MOmentum pivotal trial of Uplizna, which demonstrated the effects of the treatment in reducing the attacks associated with NMOSD, included analyses of biomarkers linked to disease activity and neuronal injury: serum neurofilament light chain (sNfL, a structural protein increasingly recognized as a signal of neuronal injury), serum glial fibrillary acidic protein (sGFAP, a marker previously found to correlate with NMOSD attacks), ubiquitin C-terminal hydrolase L1 (sUCHL1), and tau (sTau).

The trial identified important biomarker trends associated with NMOSD attacks. Throughout the 28-week randomized controlled trial period (RCP) and the two-year open-label follow-up period, the concentration of all four biomarkers increased during NMOSD attacks, and in the days leading up to attacks. Of the four biomarkers evaluated in the trial, sNfL measured at the time of attack was the strongest predictor of worsening disability during and after attacks (as measured by Expanded Disability Status Scale, or EDSS). The strong link suggests that higher sNfL levels may be associated with more severe attacks and increased risk of residual disability. However, only levels of sGFAP were determined to be predictive of future attacks, building upon prior research studying that biomarker in particular.

“This analysis provides valuable insights into how we can improve care for people with NMOSD by integrating easily accessible serum biomarker assessments into treatment decision-making,” said Orhan Aktas, M.D., Professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany. “Conducting assessments of the sNfL biomarker during an attack can inform clinicians about the attack severity and the likelihood of residual disability in the patient, and therefore may guide therapeutic interventions to help preserve their long-term outcomes. Combination with other select serum biomarkers such as sGFAP may further increase prediction of clinical activity and, thus, prognosis in this devastating disorder.”

Importantly, biomarker changes among Uplizna-treated participants in the trial reinforced the effects of this medicine. Compared with placebo, Uplizna was shown to hinder biomarker elevation during attacks while reducing biomarker levels over time in the absence of attacks. Participants treated with Uplizna had significantly lower levels of sNfL at the end of the RCP compared to placebo (22% vs. 45% of participants with sNfL levels above 16 mg/mL, respectively), and numerically lower levels of the other three biomarkers. Among patients who did experience attacks, those treated with Uplizna had lower biomarker levels during attacks versus those who received placebo, reflecting potentially less severe events, and sGFAP levels were significantly lower with Uplizna among those who did not experience attacks versus placebo.

s Optica Spectrum Disorder" (B. Weinshenker). Poster Session: 13 Date: April 27, 8-9 a.m. EDT.

See: Aktas O, Hartung H-P, Smith MA, et al. J Neurol Neurosurg Psychiatry Epub ahead of print:.doi:10.1136/jnnp-2022- 330412.

Condition: Neuromyelitis Optica
Type: drug

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