Efficacy

Among patients in the ULTOMIRIS treatment arm,

Improvements in MG-ADL total scores from baseline were observed within 1 week of treatment, and were sustained through 26 weeks of treatment.¹

ULTOMIRIS demonstrated efficacy vs placebo at Week 26 (-3.1 vs -1.4, respectively [P<0.001])¹

CHAMPION-MG STUDY LIMITATIONS: Data shown are least-squares means and 95% confidence intervals (CIs), using a mixed model for repeated measures; 95% CIs were not adjusted for multiplicity.^1,3

Time to response was part of the planned efficacy analysis, but the primary endpoint was at Week 26. Therefore, results should be interpreted with caution.

ULTOMIRIS demonstrated improvement in QMG total score through Week 26¹

In the key secondary endpoint, change in QMG total score from baseline to Week 26, ULTOMIRIS achieved a reduction of 2.8 points vs 0.8 points for placebo (P<0.001).¹

Improvement was shown in patients’ QMG total score through Week 26¹

• Improvements in QMG scores were seen across the ocular, bulbar, and limb domain scores from baseline to Week 26³
• • CHAMPION-MG STUDY LIMITATION: Change from baseline in QMG individual symptom domains was an exploratory endpoint. Efficacy or clinical significance should be interpreted with caution

The open-label extension (OLE) period began following Week 26, when all patients received ULTOMIRIS and results were observed through Week 60.²

MG-ADL total score observed through Week 60 in the open-label extension period²

CHAMPION-MG STUDY LIMITATION: Any inference of efficacy or clinical significance should be interpreted with caution since the study was designed to evaluate safety and lacked a control group.

At Week 60, 28% of patients decreased their daily dose of corticosteroids, with 6.2% discontinuing corticosteroids altogether^3,a,b

^aN=161.³

^bData cutoff date of November 9, 2021.³

The open-label extension period began following Week 26, when all patients received ULTOMIRIS and results were observed through Week 60.²

QMG total score observed through Week 60 in the OLE period²

CHAMPION-MG STUDY LIMITATION: Any inference of efficacy or clinical significance should be interpreted with caution since the study was designed to evaluate safety and lacked a control group.

The proportion of patients taking ULTOMIRIS that achieved a ≥5-point improvement in QMG total score was greater than the proportion of patients taking placebo.

30% of patients taking ULTOMIRIS had a ≥5-point improvement in QMG total score vs 11.3% taking placebo (P=0.005).

Patients treated with ULTOMIRIS were more likely to experience larger improvement in QMG total score¹

• Change from baseline to Week 26 in revised 15-Component MG-QoL15r: -3.3 for ULTOMIRIS and -1.6 for placebo
• Change from baseline to Week 26 in the Neuro-QoL Fatigue score: -7.0 for ULTOMIRIS and -4.8 for placebo
• Neither of these endpoints were statistically significant

More patients taking ULTOMIRIS achieved a ≥3-point improvement in MG-ADL total score vs placebo.

57% of patients taking ULTOMIRIS had a ≥3-point improvement in MG-ADL total score vs 34% of patients taking placebo.

1 in 4 patients treated with ULTOMIRIS achieved minimal manifestation status vs placebo³

Minimal manifestation status is achieved when a patient has no symptoms of functional limitations from MG, but has some weakness on examination of some muscles. It's another way to assess a patient's symptoms after treatment initiation.^5,c,d

Patients reaching minimal manifestation status may be better able to perform everyday activities.⁵

CHAMPION-MG STUDY LIMITATION: Minimal manifestation was an exploratory endpoint. Any inference of efficacy should be interpreted with caution.³

^cPer MGFA post-intervention status.⁵

^dThis class recognizes that some patients who otherwise meet the definition of pharmacologic remission do have weakness that is only detectable by careful examination.⁵