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P.054 Long-term safety and efficacy of zilucoplan in myasthenia gravis: additional interim analyses of RAISE-XT

Published online by Cambridge University Press:  24 May 2024

A Genge
Affiliation:
(Montreal)*
JF Howard Jr.
Affiliation:
(Chapel Hill)
M Freimer
Affiliation:
(Columbus)
C Hewamadduma
Affiliation:
(Sheffield)
Y Hussain
Affiliation:
(Austin)
A Maniaol
Affiliation:
(Oslo)
R Mantegazza
Affiliation:
(Milan)
M Smilowski
Affiliation:
(Katowice)
K Utsugisawa
Affiliation:
(Hanamaki City)
T Vu
Affiliation:
(Tampa)
MD Weiss
Affiliation:
(Seattle)
PW Duda
Affiliation:
(Cambridge)
B Boroojerdi
Affiliation:
(Monheim)
M Vanderkelen
Affiliation:
(Brussels)
G de la Borderie
Affiliation:
(Colombes)
MI Leite
Affiliation:
(Oxford)
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Abstract

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Background: Zilucoplan, a macrocyclic peptide complement component 5 inhibitor, sustained efficacy for up to 60 weeks of treatment, with a favourable safety profile in patients with acetylcholine receptor autoantibody-positive generalised myasthenia gravis in an interim analysis of RAISE-XT (NCT04225871). We evaluate the safety and efficacy of zilucoplan up to 96 weeks. Methods: RAISE-XT, a Phase 3, multicentre, open-label extension study, included patients who participated in the double-blind Phase 2 (NCT03315130) and Phase 3 (NCT04115293) zilucoplan studies. Patients self-administered daily subcutaneous zilucoplan 0.3mg/kg injections. Primary outcome was incidence of treatment-emergent adverse events (TEAEs). Secondary outcomes included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score. Results: At data cut-off (11 May 2023), median (range) exposure to zilucoplan was 1.8 (0.11–5.1) years (N=200). TEAEs occurred in 191 (95.5%) patients; the most common TEAE was COVID-19 (n=64; 32.0%). At Week 96, mean (standard error) change in MG-ADL score from double-blind study baseline was –6.33 (0.49) and –7.83 (0.60) for patients who received zilucoplan 0.3mg/kg and placebo in the double-blind studies, respectively. Conclusions: Zilucoplan demonstrated a favourable long-term safety profile. Efficacy was sustained for 96 weeks in patients who had previously received zilucoplan and who switched from placebo.

Type
Abstracts
Copyright
© The Author(s), 2024. Published by Cambridge University Press on behalf of Canadian Neurological Sciences Federation