Objective:

To determine whether ravulizumab treatment enables patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR Ab+ gMG) to achieve the Myasthenia Gravis Foundation of America Post-Intervention Status (MGFA-PIS) of “improved”, with or without achieving minimal manifestation status (MMS).

Background:

The 26-week, phase 3, randomized, double-blind, placebo-controlled CHAMPION MG study demonstrated the efficacy and safety of ravulizumab in patients with AChR Ab+ gMG. Patients who completed the randomized controlled period (RCP) could receive ravulizumab in an open-label extension (OLE); blinding to original treatment was maintained.

Design/Methods:

In a prespecified exploratory analysis, modified MGFA-PIS (improved and achieved MMS, improved without MMS, unchanged, worsened) was assessed at Week 26 (patients who completed the RCP with Week 26 MGFA-PIS data) and Week 60 (patients who received≥ 1 ravulizumab dose during the OLE with Week 60 MGFA-PIS data) relative to RCP baseline. A proportional-odds cumulative logit model (cumulated across categories starting from best outcome) was used to analyze outcomes at Week 26, with treatment as a fixed categorical effect and adjusting for geographic region (estimated odds ratio [OR]> 1 indicates beneficial treatment effect).

Results:

At Week 26, improved status with and without MMS, respectively, was achieved by 25.6%(20/78) and 21.8%(17/78) of ravulizumab-treated patients, and by 9.9%(8/81) and 22.2%(18/81) of patients who received placebo (adjusted OR: 2.236, 95% confidence interval: 1.210, 4.130; p= 0.01). At Week 60, improved status, with/without MMS, was achieved by 33.9%(19/56)/32.1%(18/56) of patients who continued ravulizumab treatment and 31.6%(18/57)/42.1%(24/57) of patients who switched from placebo to ravulizumab.

Conclusions:

Ravulizumab-treated patients were more likely than those receiving placebo to achieve MGFA-PIS of improved, with or without MMS. The increase in the proportion of patients achieving MGFA-PIS of improved, with or without MMS, with continued treatment, suggests that longer-term treatment may be needed for some patients to achieve this status.

Disclosure: Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Narayanaswami 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. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami 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. Narayanaswami has received personal …