Clinically Meaningful Improvement in Physical Fatigue and Muscle Weakness Fatigability with Rozanolixizumab: Post-hoc Analysis of MG Symptoms PRO Responder Rate in the MycarinG study (P4-11.001)

To illustrate the clinical meaningfulness of change in score in Myasthenia Gravis (MG) Symptoms Patient-Reported Outcome (PRO) scales in patients receiving one cycle of rozanolixizumab in the MycarinG study. In the Phase 3 MycarinG study (NCT03971422), one 6-week cycle of rozanolixizumab significantly improved Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, with 70. 6% of patients receiving rozanolixizumab and 31. 3% of patients receiving placebo achieving MG-ADL response at Day 43. In the novel MG Symptoms PRO scales, rozanolixizumab treatment significantly improved muscle weakness fatigability, physical fatigue and bulbar muscle weakness scores at Day 43, based on responder thresholds determined using anchor- and distribution-based methods as defined in a post-hoc analysis of Day 29 data. Patients received rozanolixizumab 7 mg/kg or 10 mg/kg, or placebo. Change from baseline to Day 43 in the MG Symptoms PRO scales was assessed and thresholds for clinically meaningful response applied: a decrease of ≥16. 67 points for muscle weakness fatigability and a decrease of ≥20. 00 points for physical fatigue and bulbar muscle weakness. Muscle weakness fatigability: meaningful improvement at Day 43 was achieved by 46. 9% (30/64), 56. 5% (35/62) and 28. 1% (18/64) of patients receiving rozanolixizumab 7 mg/kg, 10 mg/kg, and placebo, respectively, using the -16. 67 threshold. Physical fatigue: meaningful improvement was achieved by 31. 3% (20/64), 48. 4% (30/62) and 26. 6% (17/64) of patients receiving rozanolixizumab 7 mg/kg, 10 mg/kg, and placebo, respectively, using the -20. 00 threshold. Bulbar muscle weakness: meaningful improvement was achieved by 26. 6% (17/64), 32. 3% (20/62) and 10. 9% (7/64) of patients receiving rozanolixizumab 7 mg/kg, 10 mg/kg, and placebo, respectively, using the -20. 00 threshold. This post-hoc analysis found that a higher percentage of patients treated with rozanolixizumab than placebo had meaningful improvements in physical fatigue and muscle weakness fatigability symptoms, which are identified by patients as significant but not captured by the commonly used MG-ADL assessment. Disclosure: Dr. Habib has received personal compensation in the range of 10, 000-49, 999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of 10, 000-49, 999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of 5, 000-9, 999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of 10, 000-49, 999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of 5, 000-9, 999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Alpine Immune Sciences. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of 10, 000-49, 999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of 10, 000-49, 999 for serving on a Speakers Bureau for argenx. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from VielaBio. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron. The institution of Dr. Kaminski has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Roche. Dr. Kaminski has received personal compensation in the range of 500-4, 999 for serving as a Consultant for UCB. Dr. Kaminski has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Cabaletta Bio. Dr. Kaminski has stock in ARC Biotechnology. The institution of Dr. Kaminski has received research support from National Institutes of Health. Dr. Kaminski has received publishing royalties from a publication relating to health care. Dr. Grosskreutz has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Biogen. Dr. Grosskreutz has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Alexion. Dr. Grosskreutz has received personal compensation in the range of 500-4, 999 for serving as a Consultant for UCB. Dr. Grosskreutz has received personal compensation in the range of 5, 000-9, 999 for serving as a Consultant for Amylyx. The institution of Dr. Grosskreutz has received research support from Boris Canessa Foundation. Dr. Mantegazza 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. Mantegazza has received personal compensation in the range of 5, 000-9, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Mantegazza 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. Mantegazza has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Mantegazza has received personal compensation in the range of 500-4, 999 for serving on a Speakers Bureau for SERB. The institution of Dr. Mantegazza has received research support from Alpine Immune Sciences, Inc. Dr. Vu has received personal compensation in the range of 5, 000-9, 999 for serving as a Consultant for Dianthus. Dr. Vu has received personal compensation in the range of 500-4, 999 for serving as a Consultant for ImmunAbs. Dr. Vu has received personal compensation in the range of 10, 000-49, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for ARGENX. Dr. Vu has received personal compensation in the range of 10, 000-49, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Vu has received personal compensation in the range of 10, 000-49, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Dr. Vu has received personal compensation in the range of 10, 000-49, 999 for serving on a Speakers Bureau for Alexion. Dr. Vu has received personal compensation in the range of 10, 000-49, 999 for serving on a Speakers Bureau for CSL Behring. Dr. Vu has received personal compensation in the range of 5, 000-9, 999 for serving on a Speakers Bureau for AbbVie. Dr. Vu has received personal compensation in the range of 50, 000-99, 999 for serving on a Speakers Bureau for Argenx. Dr. Vu has received personal compensation in the range of 5, 000-9, 999 for serving on a Speakers Bureau for MedScape. The institution of Dr. Vu has received research support from CSL Behring. The institution of Dr. Vu has received research support from Alexion. The institution of Dr. Vu has received research support from RA Pharma/UCB. The institution of Dr. Vu has received research support from Mitsubishi Tanaka. The institution of Dr. Vu has received research support from Mass Gen Hospital/Healy Platform Study. The institution of Dr. Vu has received research support from Amylyx Pharma. The institution of Dr. Vu has received research support from ARGENX. The institution of Dr. Vu has received research support from Alector. The institution of Dr. Vu has received research support from Annexon. The institution of Dr. Vu has received research support from Apellis. The institution of Dr. Vu has received research support from Cytokinetics. The institution of Dr. Vu has received research support from Sanofi. The institution of Dr. Vu has received research support from Horizon. The institution of Dr. Vu has received research support from Woolsey Pharma. The institution of Dr. Vu has received research support from CSL Behring. The institution of Dr. Vu has received research support from Cartesian Therapeutics. The institution of Dr. Vu has received research support from Janssen/Momenta. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Sanofi. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Dianthus Therapeutics. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Roche. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Amicus therapeutics. Prof. Vissing has received personal compensation in the range of 5, 000-9, 999 for serving as a Consultant for Sarepta. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Horizon Therapeutics. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Novartis Pharma AG. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Alexion. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Biogen. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Genethon. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Pfizer. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB Biopharma. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Speakers Bureau for Argenx. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Speakers Bureau for Lupin. Prof. Vissing has received personal compensation in the range of 500-4, 999 for serving on a Speakers Bureau for Edgewise Therapeutics. The institution of Prof. Vissing has received research support from Rigshospitalet. The institution of Prof. Vissing has received research support from UCB Pharma. The institution of Prof. Vissing has received research support from Sanofi. The institution of Prof. Vissing has received research support from NOVO Foundation. Prof. Vissing has received research support from Reneo Pharma. Prof. Vissing has received research support from Janssen Pharmaceuticals. Marion Boehnlein has received personal compensation for serving as an employee of UCB Pharma. Marion Boehnlein has stock in UCB Pharma. Dr. Greve has received personal compensation for serving as an employee of UCB Biosciences. Dr. Greve has stock in UCB Biosciences. Dr. Grimson has received personal compensation for serving as an employee of UCB Pharma. Dr. Grimson has stock in UCB Pharma. Asha Hareendran has received personal compensation for serving as an employee of UCB. Asha Hareendran has stock in UCB. Asha Hareendran has stock in Pfizer. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for UCB. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for CSL. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Alnylam. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Janssen. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Grifols. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Takeda. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Octapharma. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Pfizer. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Powell Mansfield. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Akcea. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Ionis. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Immunovant. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Sanofi. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Roche. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Momenta (now J&J). Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for Alexion. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving as a Consultant for NovoNordisk. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Takeda. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Bril 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. Bril has received personal compensation in the range of 5, 000-9, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alnylam. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for CSL. Dr. Bril has received personal compensation in the range of 500-4, 999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. The institution of Dr. Bril has received research support from UCB. The institution of Dr. Bril has received research support from Argenx. The institution of Dr. Bril has received research support from Momenta (now J&J). The institution of Dr. Bril has received research support from Immunovant. The institution of Dr. Bril has received research support from Alexion. The institution of Dr. Bril has received research support from Takeda. The institution of Dr. Bril has received research support from Grifols. The institution of Dr. Bril has received research support from CSL. The institution of Dr. Bril has received research support from Octapharma. The institution of Dr. Bril has received research support from Akcea. The institution of Dr. Bril has received research support from Ionis. The institution of Dr. Bril has received research support from Viela Bio (now Horizon). Dr. Bril has received intellectual property interests from a discovery or technology relating to health care.