Abstract Details

Title NMO SPOTLIGHT Registry: Real-World Clinical Outcomes With Eculizumab and Ravulizumab in Anti-Aquaporin-4 Antibody-Positive (AQP4-Ab+) Neuromyelitis Optica Spectrum Disorder (NMOSD)

Topic Autoimmune Neurology

Presentation(s) P11 - Poster Session 11 (8:00 AM-9:00 AM)

Poster/Presentation
Number 8-013

Objective To describe the initial characteristics and clinical outcomes of Registry participants with AQP4-Ab+ NMOSD treated with eculizumab or ravulizumab in real-world clinical practice.

Background The Alexion complement component 5 inhibitor therapies eculizumab and ravulizumab (ALXN-C5ITs) are approved in multiple countries to treat adults with AQP4-Ab+ NMOSD. The Alexion-sponsored global NMO SPOTLIGHT Registry (NCT05966467) collects ALXN-C5IT real-world safety and clinical effectiveness data in adults aged ≥18yrs with AQP4-Ab+ NMOSD.

Design/Methods Adults with AQP4-Ab+ NMOSD receiving ALXN-C5ITs were enrolled in the Registry starting August 2023. Demographics, number and types of relapses from 1yr prior to and during ALXN-C5IT therapy, meningococcal infections, and vaccination history were collected. An NMOSD relapse was defined as new onset/worsening of neurologic symptoms for >24hrs requiring acute standard-of-care treatment preceded by ≥30d of clinical stability.

Results As of 21Jun2024, 30 adults were enrolled in the Registry and available for analysis (female, 86.7%; White, 50.0%; Black, 46.7%; median [interquartile range (IQR)] age at initial diagnosis, 47 [35-57] yrs; ALXN-C5IT monotherapy, 83.3%). Median (IQR) duration of exposure to eculizumab (n=30) and ravulizumab (n=2) was 40.2 (24.0-49.7) mo and 1.0 (0.7-1.3) mo, respectively. In the 1yr prior to ALXN-C5IT, 10 (33.3%) patients had 15 relapses (annualized relapse rate [ARR]: 0.50 [95% CI: 0.28-0.82]); 2/10 (20.0%) patients had >1 relapse. While on ALXN-C5IT, 2 (6.7%) patients had relapses (ARR: 0.02 [95% CI: 0.00-0.08]); no patients had >1 relapse. Most relapses were transverse myelitis. In the 1yr prior to ALXN-C5IT through Registry enrollment, 29 (96.7%) patients received ≥1 meningococcal vaccination. No vaccinations were reported within 4 weeks before any relapse; no meningococcal infections were reported.

Conclusions Consistent with clinical trial and real-world data previously reported on eculizumab and/or ravulizumab among patients with AQP4-Ab+ NMOSD, initial results demonstrate the strong clinical benefit of ALXN-C5ITs in relapse prevention. No new safety signals were detected; no meningococcal infections were reported.

Authors/Disclosures
Ahmed Z. Obeidat, MD, PhD (Medical College of Wisconsin) PRESENTER	Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Bristol Myers Squibb. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Genentech. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for EMD Serono. Dr. Obeidat has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi/Genzyme . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Horizon pharmaceuticals . Dr. Obeidat has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sandoz. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG Therapeutics. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Alexion. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Biogen. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Bristol Myers Squibb. Dr. Obeidat has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for EMD Serono. Dr. Obeidat has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Banner life sciences . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Sanofi/genzyme. Dr. Obeidat has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for Horizon therapeutics . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Amgen. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for TG Therapeutics . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for AstraZeneca. The institution of Dr. Obeidat has received research support from NIH. The institution of Dr. Obeidat has received research support from NMSS and PCORI. Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Expert opinion and key opinion leader with MJH life sciences . Dr. Obeidat has received personal compensation in the range of $10,000-$49,999 for serving as a Faculty Speaker with CMSC. Dr. Obeidat has a non-compensated relationship as a Board member with CMSC that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Editorial Board Member with IJMSC that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Reviewer Editor with Frontiers Neurology that is relevant to AAN interests or activities. Dr. Obeidat has a non-compensated relationship as a Board of Trustee Member with National MS Society that is relevant to AAN interests or activities.
Elias S. Sotirchos, MD (Johns Hopkins University)	Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Genentech. Dr. Sotirchos has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Sotirchos has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for TG Therapeutics. The institution of Dr. Sotirchos has received research support from National Institutes of Health. The institution of Dr. Sotirchos has received research support from National Multiple Sclerosis Society. The institution of Dr. Sotirchos has received research support from Sumaira Foundation. The institution of Dr. Sotirchos has received research support from Genentech. The institution of Dr. Sotirchos has received research support from UCB. The institution of Dr. Sotirchos has received research support from Astoria Biologica. The institution of Dr. Sotirchos has received research support from Ad Scientiam. The institution of Dr. Sotirchos has received research support from Alexion. The institution of Dr. Sotirchos has received research support from Corevitas. Dr. Sotirchos has received personal compensation in the range of $500-$4,999 for serving as a Ad Hoc Reviewer with National Institutes of Health.
Veronica A. Tkachuk	Veronica A. Tkachuk has nothing to disclose.
Ho Jin Kim, MD (National Cancer Center)	Dr. Kim has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Altos Biologics, AstraZeneca, Biogen, Daewoong Pharmaceutical, Kaigene, Kolon Life Science, MDimune, Roche, and Sanofi. Dr. Kim has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion, AstraZeneca, Eisai, GC Pharma, Merck, Mitsubishi Tanabe Pharma, and Roche. Dr. Kim has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Multiple Sclerosis Journal, Journal of Clinical Neurology.
Lindsey Przybyl	Lindsey Przybyl has received personal compensation for serving as an employee of Alexion, AstraZeneca Rare Disease. Lindsey Przybyl has stock in AstraZeneca.
Sami Fam	Sami Fam has received personal compensation for serving as an employee of Alexion Pharmaceuticals. Sami Fam has or had stock in Astra Zeneca.

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