Abstract Details

Title Early Real-life Experience on Zilucoplan for Generalized Myasthenia Gravis: ZILU25 Multicenter Observational Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P9 - Poster Session 9 (5:00 PM-6:00 PM)

Poster/Presentation
Number 9-018

Objective To Assess the efficacy and safety of Zilucoplan in generalized myasthenia gravis in the real-life setting.

Background Generalized Myasthenia gravis (gMG) is a rare chronic autoimmune disease affecting
the postsynaptic membrane of the neuromuscular junction. New treatment options,
such as the C5 inhibitors, are growing with promising results. In this study we
investigated the real-world use of Zilucoplan for AChR-positive gMG in Italy.

Design/Methods Zilucoplan was self-administered by daily subcutaneous injections. Efficacy was
assessed by Myasthenia Gravis Foundation of America Post-intervention status
(MGFA-PIS), Myasthenia Gravis Activity of Daily Living (MG-ADL), Myasthenia Gravis
Composite (MGC), Myasthenia Gravis quantitative (QMG) scales and 15-items
Myasthenia Gravis Quality of Life (MG-QOL-15) questionnaire at baseline and after 1,
4, 12, 24 and 48 weeks.

Results 54 patients (37 females, aged 57.3) received zilucoplan with a mean follow-up of 26.4
weeks. We observed a clinical meaningful and significant reduction of MG-ADL, QMG,
MGC and MG-QOL-15 scores from W4 to W12 and sustained at W24 of follow-up. The
MG-ADL responder rate was 75.6% at W12 and 82.9% at W24. Minimal symptoms
expression (MSE) was obtained in in 55.6% of patients at W48 with a mean reduction
of the daily prednisone dose of 8.3 mg at W24. Previous long term IVIg was associated
to a better response to MG-ADL at W12 at multivariate analysis.

Conclusions Zilucoplan was well-tolerated and effective in most patients with AChR-positive gMG. A
clinical meaningful effect was reported since the first week and was sustained after 24
weeks. Further studies are needed to confirm these preliminary real-life data.

Authors/Disclosures
Vincenzo Di Stefano, MD, PhD PRESENTER	Dr. Di Stefano has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen.
Nicasio Rini, MD	Dr. Rini has nothing to disclose.
Paolo Emilio Alboini, MD	The institution of Dr. Alboini has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. The institution of Dr. Alboini has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Pharma. Dr. Alboini has received personal compensation in the range of $500-$4,999 for serving as an officer or member of the Board of Directors for Alexion.
Alvino Bisecco	Alvino Bisecco has nothing to disclose.
Valentina Damato (University of Florence)	Valentina Damato has nothing to disclose.
Laura Fionda, MD, PhD	Dr. Fionda has nothing to disclose.
Francesco Habetswallner	Francesco Habetswallner has nothing to disclose.
Raffaele Iorio, MD (Catholic University)	Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus Therapeutics. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Johnson & Johnson. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Argenx. Dr. Iorio has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson&Johnson.
Michelangelo Maestri Tassoni, MD, PhD	Dr. Maestri Tassoni has received personal compensation for serving as an employee of Alexion. Dr. Maestri Tassoni has received personal compensation for serving as an employee of Argenx. Dr. Maestri Tassoni has received personal compensation for serving as an employee of Ucb. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ucb. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Maestri Tassoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Ucb.
Alessia Pugliese, MD	Dr. Pugliese has nothing to disclose.
Francesco Sacca, MD, FAAN (University Federico II)	Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lexeo. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genpharm. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medpharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Madison Pharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zai Lab. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sandoz. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neopharm Israel. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson&Johnson. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Sacca has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Astrazeneca. The institution of Dr. Sacca has received research support from AIFA. The institution of Dr. Sacca has received research support from FARA.
CLAUDIA VINCIGUERRA, MD, PhD	Dr. VINCIGUERRA has received personal compensation in the range of $0-$499 for serving as a speakers, manuscript writing, and educational events with Alexion.
RITA FRANGIAMORE, MD	Dr. FRANGIAMORE has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB PHARMA. Dr. FRANGIAMORE has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ALEXION. Dr. FRANGIAMORE has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. FRANGIAMORE has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB PHARMA.

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