Long-term Efficacy of Satralizumab in Adults with Aquaporin-4-IgG-seropositive (AQP4-IgG+) Neuromyelitis Optica Spectrum Disorder (NMOSD): Results from SAkuraMoon
Anthony Traboulsee1, Ingo Kleiter2, Jacqueline Palace3, Kazuo Fujihara4, Albert Saiz5, Ivana Vodopivec6, Gaelle Klingelschmitt6, Carole Marcillat6, Jeffrey Bennett7
1University of British Columbia, 2Ruhr University Bochum, 3John Radcliffe Hospital, 4Fukushima Medical University School of Medicine, 5Service of Neurology, Hospital Clinic and Institut d'Investigació Biomèdica August Pi i Sunyer (IDIBAPS), University of Barcelona, 6F. Hoffmann-La Roche Ltd, 7University of Colorado School of Medicine
Objective:
To evaluate the long-term efficacy of satralizumab in patients with AQP4-IgG+ NMOSD.
Background:

Satralizumab reduced the risk of protocol-defined relapse (PDR) and severe PDR vs placebo in the double-blind periods (DBP) of two phase 3 trials in patients with NMOSD: SAkuraSky (NCT02028884; satralizumab + baseline immunosuppressants [IST]) and SAkuraStar (NCT02073279; satralizumab monotherapy).

Design/Methods:

Patients who completed the DBPs and open-label extensions (OLEs) of SAkuraSky and SAkuraStar were rolled-over into a single-arm, open-label study (SAkuraMoon [NCT04660539]) and continued receiving satralizumab 120mg Q4W +/- IST; we evaluated all AQP4-IgG+ adults (≥18 years) who received ≥1 dose of satralizumab during these studies (data cut-off: 31 January 2022).

PDRs in the DBPs were adjudicated by a Clinical Endpoint Committee; however, PDRs in the OLEs and SAkuraMoon were determined by the investigator (iPDRs). We evaluated the annualized iPDR rate (ARR), time to first iPDR, severe iPDR (≥2 point increase in the Expanded Disability Status Scale [EDSS] score), and sustained EDSS worsening (EDSS increase of ≥2, ≥1, or ≥0.5 points for patients with baseline scores of 0, 1–5, or ≥5.5, respectively, confirmed ≥24 weeks post-initial-worsening).

Results:

Overall, 106 AQP4-IgG+ adults were included. The median (range) duration of satralizumab exposure was 5.0 (0.1–7.9) years. The overall adjusted ARR (95% CI) was 0.09 (0.06–0.12). When assessed longitudinally, the ARR did not increase with additional years of exposure (Y1: 0.16 [0.09–0.27]; Y2: 0.10 [0.05–0.20]; Y3: 0.05 [0.01–0.15]; Y4: 0.07 [0.02–0.26]). At Week 240 (4.6 years), 72% (95% CI: 62–80%) of satralizumab-treated patients were free from iPDR, 91% (84–96%) were free from severe iPDR, and 85% (75–91%) had no sustained EDSS worsening.

Conclusions:

These results demonstrate that the efficacy of satralizumab observed in the DBPs is sustained with long-term treatment. High proportions of patients remained free from relapse, severe relapse, or worsening disability, with a consistently low ARR over 4.6 years of satralizumab exposure.

10.1212/WNL.0000000000202535