Clinical Trial Data
CLINICAL TRIAL DATA
Rapid efficacy in
action for a broad
population of
adults with gMG1,2*
Week 6:
Statistically significant
improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs -0.8; P<0.001)1
Week 1:
Improvement observed
as early as Day 8
in total MG-ADL score after initial dose of RYSTIGGO (7 mg/kg: -1.2, 10 mg/kg: -1.3, placebo: -0.4)3†
†Based on post-hoc analysis.3
The clinical study included adult patients who were AChR or MuSK autoantibody positive, and who
had MGFA Class II-IVa disease, an MG-ADL score of ≥3 (with ≥3 points from non-ocular symptoms),
serum immunoglobulin G levels ≥5.5 gL, and who were on a stable dose of MG therapy prior to screening.1
Week 6:
Statistically significant
improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs -0.8; P<0.001)1
Week 1:
Improvement observed as early as Day 8
in total MG-ADL score after initial dose of RYSTIGGO (7 mg/kg: -1.2, 10 mg/kg: -1.3, placebo: -0.4)3†
†Based on post-hoc analysis.3
The clinical study included adult patients who were AChR or MuSK autoantibody positive, and who had MGFA Class II-IVa disease, an MG-ADL score of ≥3 (with ≥3 points from non-ocular symptoms), serum immunoglobulin G levels ≥5.5 gL, and who were on a stable dose of MG therapy prior to screening.1
PRIMARY ENDPOINT: MG-ADL
RYSTIGGO demonstrated rapid, clinically meaningful, and statistically significant improvements in patients' activities of daily living in a 6-week treatment cycle1,2‡
The efficacy of RYSTIGGO for the treatment of adult anti-AChR Ab+ and anti-MuSK Ab+ gMG patients was established in an up to 18-week, multicenter, randomized, double-blind, placebo-controlled study. In the study, 200 patients were randomized 1:1:1 to receive weight-tiered doses of RYSTIGGO (n=133), either 7 mg/kg of RYSTIGGO (n=66) or 10 mg/kg of RYSTIGGO (n=67), or placebo (n=67).1
myasthenia gravis activities of daily living (MG-ADL)
MG-ADL assesses the impact of gMG on daily functions of 8 symptoms on a scale of 0-3, with total scores ranging from 0-24. Higher scores are interpreted as greater impairments.1 An improvement of ≥2 points was established as clinically meaningful.2 Measures include4:
- Voice/speech problems
- Chewing
- Swallowing
- Breathing
- Brushing teeth and/or combing hair
- Rising from a chair
- Diplopia
- Eyelid droop
EXPLORATORY SECONDARY endpoint
MG-ADL Responder Rate2
MG-ADL responder (≥2.0-point improvement from baseline) at Week 6 (Day 43)2
Study limitations: MG-ADL Responder Rate was a prespecified secondary endpoint not controlled for multiplicity; therefore, data should be interpreted with caution and conclusions cannot be drawn.
SUBGROUP ANALYSIS
The first and only treatment indicated for adult patients with anti-MuSK Ab+ gMG1
Subgroup analysis: mean MG-ADL change from baseline to Week 6 (Day 43) in participants with anti-MuSK Ab+ gMG.5
In a subgroup analysis, study participants with anti-MuSK Ab+ gMG showed improvements in MG-ADL scores vs placebo after the 6-week treatment cycle2
Subgroup analysis: MG-ADL responder rate at Week 6 (Day 43)2
All patients with anti-MuSK Ab+ gMG who received RYSTIGGO and had data available at Week 6 (Day 43) were MG-ADL responders2
#A clinical responder was established as a patient with a ≥2-point improvement in the total MG-ADL score compared to baseline at Week 6 (Day 43). Clinical responder data were not collected for one patient in the RYSTIGGO 10 mg/kg group who discontinued treatment.2,3
Study limitations: MG-ADL Responder Rate was a prespecified secondary endpoint not controlled for multiplicity; therefore, data should be interpreted with caution.
SECONDARY ENDPOINTS
RYSTIGGO demonstrated statistically significant improvements across key secondary efficacy outcome measures vs placebo1-3
Change from baseline to Week 6 (Day 43) was measured in:
††Improvements of ≥3-point were established as clinically meaningful.2
‡‡Improvements of ≥3-point were established as clinically meaningful.2
MG Symptoms PRO was part of the planned efficacy analysis; however, efficacy or clinical significance should be interpreted with caution.
MG Symptoms PRO was part of the planned efficacy analysis; however, efficacy or clinical significance should be interpreted with caution.
MG Symptoms PRO was part of the planned efficacy analysis; however, efficacy or clinical significance should be interpreted with caution.
Quantitative Myasthenia Gravis (QMG)
QMG is a physician assessment scoring system that quantifies disease severity. Measures are assessed on a scale of 0-3, with total scores ranging from 0-39.1,2,6 An improvement of ≥3 points was established as clinically meaningful.2 Measures include6:
- Ptosis
- Facial muscle weakness
- Dysarthria
- Grip strength
- Neck flexion endurance
- Diplopia
- Difficulty swallowing a cup of water
- Percent predicted forced vital capacity
- Arm and leg endurance
- Ptosis
- Facial muscle weakness
- Dysarthria
- Grip strength
- Neck flexion endurance
- Diplopia
- Difficulty swallowing a cup of water
- Percent predicted forced vital capacity
- Arm and leg endurance
Myasthenia Gravis Composite (MG-C)
MG-C is a 10-item patient and physician assessment of the signs and symptoms of myasthenia gravis based on exam and patient history, with total scores ranging from 0-50. Higher scores are interpreted as greater impairments.7,8 An improvement of ≥3 points was established as clinically meaningful.2 Measures include7:
- Ptosis
- Eye closure
- Chewing
- Breathing
- Shoulder abduction
- Diplopia
- Talking
- Swallowing
- Neck flexion or extension
- Hip flexion
Myasthenia Gravis Symptoms Patient Reported Outcomes (MGSPRO)2,8
Muscle weakness fatigability: A scale that measures muscle fatigability by assessing use-induced reduction in the ability of the following muscles to function:
- Proximal
- Ocular
- Bulbar
- Respiratory
Physical fatigue: A scale that measures the symptoms/manifestations of physical fatigue, including:
- Body and limb muscle weakness
- Heaviness
- Lack of energy and strength
Bulbar muscle weakness: A scale that measures symptoms/manifestations associated with bulbar muscle weakness, including:
- Mouth drooping
- Speech and voice problems
- Difficulties with chewing and swallowing
MG Symptoms PRO was part of the planned efficacy analysis; however, efficacy or clinical significance should be interpreted with caution.
**MG Symptoms PRO was part of the planned efficacy analysis; however, efficacy or clinical significance should be interpreted with caution.
Reduction in IgG levels1,3
A reduction in total IgG serum concentrations was observed in both RYSTIGGO treatment groups. Decreases in AChR autoantibody and MuSK autoantibody levels followed a similar pattern.
Study limitations: The pharmacological effect of RYSTIGGO was assessed by measuring the decrease in serum IgG levels; the clinical significance of this data has not been established.
Ab+=antibody positive; AChR=acetylcholine receptor; CI=confidence interval; gMG=generalized myasthenia gravis; IgG=immunoglobulin G; LSM=least squares mean; MG-ADL=Myasthenia Gravis Activities of Daily Living; MGFA=Myasthenia Gravis Foundation of America; MuSK=muscle-specific tyrosine kinase; PRO=Patient-Reported Outcome; SE=standard error.
References:
- RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
- Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383-394. doi:10.1016/S1474-4422(23)00077-7
- Data on file. UCB Inc., Smyrna, GA.
- MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed January 31, 2023. https://myasthenia.org/Portals/0/ADL.pdf
- Habib A, Kaminski H, Drużdż A, et al. Efficacy of rozanolixizumab in muscle specific kinase antibody-positive generalized myasthenia gravis: outcomes from the randomized, phase 3 MycarinG study. Paper presented at: AANEM 2022; September 21-24, 2022; Nashville, TN.
- QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed January 31, 2023. https://myasthenia.org/Portals/0/QMG.pdf
- MG composite scale. Myasthenia Gravis Foundation of America. 2012. Accessed January 31, 2023. https://myasthenia.org/Portals/0/MG%20composite%20score.pdf
- Regnault A, Morel T, de la Loge C, Mazerolle F, Kaminski HJ, Habib AA. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms PRO. Neurol Ther. 2023:1-18. doi:10.1007/s40120-023-00464-x
