EFFICACY

Rapid efficacy in action for a broad population of adults with gMG1,2*

In a clinical study, RYSTIGGO demonstrated statistically significant improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs. -0.8; P<0.001), with improvements observed as early as Week 1.1

*The clinical study included adults who were anti-AChR Ab+ and anti-MuSK Ab+, 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

Rapid efficacy in action for a broad population of adults with gMG1,2*

In a clinical study, RYSTIGGO demonstrated statistically significant improvements vs placebo in MG-ADL total score at Week 6 (-3.4 vs. -0.8; P<0.001), with improvements observed as early as Week 1.1

*The clinical study included adults who were anti-AChR Ab+ and anti-MuSK Ab+, 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

Adults taking RYSTIGGO experienced rapid, clinically meaningful, and statistically significant improvements in activities of daily living in a 6-week treatment cycle1,2†

Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+1-3‡

Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+.
Change from baseline to Week 6 (Day 43) in MG-ADL total score in adults who are anti-AChR Ab+ or anti-MuSK Ab+.

†Clinically meaningful was established as a ≥2-point improvement in MG-ADL total score.2
‡During the treatment period, RYSTIGGO or placebo were administered subcutaneously once a week for 6 weeks.1
§Treatment initiation on Day 1.2

Adults taking RYSTIGGO achieved maximum efficacy at Week 6 (Day 43).1

The efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG 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). The study included a 4-week screening period and a 6-week treatment period followed by 8 weeks of observation.1

GO TO THE STUDY DESIGN

MG-ADL OUTCOME MEASURE

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. An improvement of ≥2 points was established as clinically meaningful.1,2

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 Rate

Proportion of MG-ADL responders from baseline at Week 6 (Day 43)2

MG-ADL responders: ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43)2

MG-ADL >2x more responders vs placebo.
MG-ADL >2x more responders vs placebo.

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: MG-ADL IN ANTI-MuSK Ab+ PATIENTS

The first and only treatment indicated for adults with anti-MuSK Ab+ gMG1

Subgroup analysis of the primary efficacy endpoint: Mean change from baseline to Week 6 (Day 43) in MG-ADL total score in adults with anti–MuSK Ab+ gMG2,5

Subgroup analysis of study participants with anti-MuSK Ab+ gMG showed improvements in MG-ADL scores vs placebo after the 6-week treatment cycle.
Subgroup analysis of study participants with anti-MuSK Ab+ gMG showed improvements in MG-ADL scores vs placebo after the 6-week treatment cycle.

All adults with anti-MuSK Ab+ gMG who received RYSTIGGO and had data available at Week 6 (Day 43) were MG-ADL responders2

Subgroup analysis of MG-ADL responder rate: ≥2.0-point improvement in MG-ADL total score from baseline at Week 6 (Day 43) in adults with anti-MuSK Ab+ gMG2

100% of patients with anti-MuSK Ab+ gMG were clinical responders to RYSTIGGO (n=12).
100% of patients with anti-MuSK Ab+ gMG were clinical responders to RYSTIGGO (n=12).

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 and conclusions cannot be drawn.

OTHER MG-ADL EFFICACY ENDPOINT

Minimal Symptom Expression (MSE) rates observed during the pivotal study2

Other efficacy endpoint: Proportion of adults achieving MSE during treatment and observation in the pivotal study2

Patients who reached MSE achieved an MG-ADL total score of 0 to 1 at any time up to and including Week 6 (Day 43) of the pivotal study.
Patients who reached MSE achieved an MG-ADL total score of 0 to 1 at any time up to and including Week 6 (Day 43) of the pivotal study.

MSE was an exploratory endpoint and not controlled for multiplicity. Therefore, data should be interpreted with caution and conclusions cannot be drawn.

Watch a video featuring Dr. Christyn Edmundson, an expert in gMG, discussing the MG-ADL results from the RYSTIGGO pivotal study.
RYSTIGGO Pivotal Study: MG-ADL Results

Review the RYSTIGGO MG-ADL results and MSE rates with a gMG expert

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Ab+=antibody positive; AChR=acetylcholine receptor; CI=confidence interval; gMG=generalized myasthenia gravis; LS=least squares; MG-ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGFA=Myasthenia Gravis Foundation of America; MuSK=muscle-specific tyrosine kinase; SE=standard error.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. 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
  3. Data on file. UCB Inc., Smyrna, GA.
  4. MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed May 13, 2024. https://myasthenia.org/Portals/0/ADL.pdf
  5. 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.

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SECONDARY ENDPOINTS

The efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG 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

GO TO THE STUDY DESIGN 

QUANTITATIVE MYASTHENIA GRAVIS (QMG)

Adults taking RYSTIGGO experienced statistically significant improvements in QMG score vs placebo1

Secondary endpoint: Mean change in QMG score from baseline to Week 6 (Day 43)1

Quantitative Myasthenia Gravis (QMG) Score clinically meaningful improvement vs placebo.
Quantitative Myasthenia Gravis (QMG) Score clinically meaningful improvement vs placebo.

†Improvements of ≥3 points were established as clinically meaningful.2

QMG OUTCOME MEASURE

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. An improvement of ≥3 points was established as clinically meaningful.1-3

Measures include3:

  • 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 (MGC)

Adults taking RYSTIGGO experienced statistically significant improvements in MGC score vs placebo2,4

Secondary endpoint: Mean change in MGC score from baseline to Week 6 (Day 43)2,4

Myasthenia Gravis Composite (MGC) Score clinically meaningful improvement vs placebo.
Myasthenia Gravis Composite (MGC) Score clinically meaningful improvement vs placebo.

‡Improvements of ≥3 points were established as clinically meaningful.2

MGC OUTCOME MEASURE

MGC 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. An improvement of ≥3 points was established as clinically meaningful.2,5,6 

Measures include5:

  • Ptosis
  • Eye closure
  • Chewing
  • Breathing
  • Shoulder abduction
  • Diplopia
  • Talking
  • Swallowing
  • Neck flexion or extension
  • Hip flexion

MYASTHENIA GRAVIS SYMPTOMS PATIENT-REPORTED OUTCOME (MG SYMPTOMS PRO)

MG Symptoms PRO evaluated gMG symptoms across 3 domains2:

MG SYMPTOMS PRO MEASURE

MG Symptoms PRO is a novel outcome measure developed by UCB and used in the RYSTIGGO clinical trials, that complements established clinical outcome measures and is the first outcome measure to include a standalone assessment of physical fatigue.2,6,7

Symptoms are measured over a 7-day period. A score ranging from 0-100 is calculated for each domain, with a higher score indicating greater symptom severity.6

3 domains of MG Symptoms PRO were evaluated in the RYSTIGGO pivotal study2,6:

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 the symptoms/manifestations associated with bulbar muscle weakness, including:

  • Mouth drooping
  • Speech and voice problems
  • Difficulties with chewing and swallowing
  • Facial muscles
Video features Dr. Christyn Edmundson, an expert in gMG, discussing the secondary endpoint results, including MG Symptoms PRO, from the RYSTIGGO pivotal study.
RYSTIGGO Pivotal Study: Secondary Endpoint Results Including MG Symptoms PRO

Watch a leading expert in gMG review the RYSTIGGO secondary endpoint results

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OTHER ENDPOINT

Reduction in immunoglobulin G (IgG) levels1

A reduction in total IgG serum concentrations was observed in adults in both RYSTIGGO treatment groups. Decreases in AChR autoantibody and MuSK autoantibody levels followed a similar pattern.1

Other endpoint: Mean % change from baseline to Week 6 (Day 43) in total IgG level4

Reduction in circulating IgG.
Reduction in circulating IgG.

§Treatment initiation on Day 1.4

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; LS=least squares; MG-ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGFA=Myasthenia Gravis Foundation of America; MG Symptoms PRO/MGSPRO=Myasthenia Gravis Symptoms Patient-Reported Outcome; MuSK=muscle-specific tyrosine kinase; SE=standard error.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. 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
  3. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed June 6, 2024. https://myasthenia.org/Portals/0/QMG.pdf
  4. Data on file. UCB Inc., Smyrna, GA.
  5. MG composite scale. Myasthenia Gravis Foundation of America. 2012. Accessed June 6, 2024. https://myasthenia.org/Portals/0/MG%20composite%20score.pdf
  6. Regnault A, Morel T, de la Loge C, et al. Measuring overall severity of myasthenia gravis (MG): evidence for the added value of the MG Symptoms Pro. Neurol Ther. 2023;12(5):1573-1590. doi:10.1007/s40120-023-00464-x
  7. Cleanthous S, Mork AC, Regnault A, et al. Development of the Myasthenia Gravis (MG) Symptoms PRO: a case study of a patient-centred outcome measure in rare disease. Orphanet J Rare Dis. 2021;16(1):457. doi:10.1186/s13023-021-02064-0

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EXTENSION STUDIES

The long-term safety and efficacy of RYSTIGGO for the treatment of adults with anti-AChR Ab+ and anti-MuSK Ab+ gMG was established in two Phase 3 extension studies, MG0004 and MG0007. Extension study results shown here are part of a pooled data analysis.2-4

GO TO THE STUDY DESIGN

The primary endpoints in the RYSTIGGO extension studies evaluated the proportion of patients experiencing treatment emergent adverse events (TEAEs). The most common TEAEs (≥10%) reported in adults receiving either dose of RYSTIGGO in the extension studies were headache, diarrhea, COVID-19, decreased blood immunoglobulin G, nausea, and pyrexia.3-5

MG-ADL SCORES IN EXTENSION STUDIES

Improvements were observed in adults receiving subsequent cycles of RYSTIGGO5

Improvements in MG-ADL score across each subsequent 6-week treatment cycle5

Improvements in MG-ADL across each subsequent 6-week treatment cycle.
Improvements in MG-ADL across each subsequent 6-week treatment cycle.

The most common adverse reactions being observed with repeated cyclic treatment were consistent with adverse reactions observed in the pivotal study1,5

†Treatment initiation on Day 1.5
Interim analysis as of July 8, 2022.5

MG-ADL OUTCOME MEASURE 

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. An improvement of ≥2 points was established as clinically meaningful.1,2

Measures include6:

  • Voice/speech problems
  • Chewing
  • Swallowing
  • Breathing
  • Brushing teeth and/or combing hair
  • Rising from a chair
  • Diplopia
  • Eyelid droop

MINIMAL SYMPTOM EXPRESSION (MSE) IN EXTENSION STUDIES

MSE rates through 6 subsequent cycles of RYSTIGGO

Exploratory endpoint: Proportion of adults achieving MSE with each subsequent cycle of RYSTIGGO5

Patients who reached MSE achieved an MG-ADL total score of 0 or 1 at any time up to and including Week 6 (Day 43) of the pivotal study.
Patients who reached MSE achieved an MG-ADL total score of 0 or 1 at any time up to and including Week 6 (Day 43) of the pivotal study.

‡Day 43.

MSE was an exploratory endpoint and not controlled for multiplicity. Therefore, data should be interpreted with caution and conclusions cannot be drawn.

Data should be interpreted with caution given the decreasing number of patients receiving subsequent treatment cycles.

See a video that features Dr. Christyn Edmundson, an expert in gMG, discussing the RYSTIGGO extension studies that further evaluated the safety and efficacy of RYSTIGGO over time.
RYSTIGGO Extension Studies

Hear the RYSTIGGO extension study results reviewed by an expert in gMG

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TREATMENT-FREE INTERVALS

Majority of adults had treatment-free intervals between 4 and 8 weeks5

Time to subsequent symptom-driven treatment cycle from last subcutaneous infusion5

Time to subsequent symptom-driven treatment cycle
Time to subsequent symptom-driven treatment cycle

Interim analysis as of July 8, 2022.5

  • The median time between treatment cycles was 8 weeks (56 days) for adults treated with RYSTIGGO who initiated 4 cycles1§
  • The safety of initiating subsequent cycles sooner than 9 weeks (63 days) from the start of the previous treatment cycle has not been established1

§This is equivalent to 14 weeks (98 days) from the start of the previous treatment cycle.1

Learn more about RYSTIGGO 6-week cyclic dosing

EXPLORE DOSING

Ab+=antibody positive; AChR=acetylcholine receptor; gMG=generalized myasthenia gravis; MG-ADL=Myasthenia Gravis Activities of Daily Living; MG=myasthenia gravis; MGFA=Myasthenia Gravis Foundation of America; MuSK=muscle-specific tyrosine kinase; Nsub=number of participants with an MSE assessment.

References:

  1. RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. 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
  3. A study to investigate the long-term safety, tolerability, and efficacy of rozanolixizumab in adult patients with generalized myasthenia gravis. ClinicalTrials.gov identifier: NCT04124965. Updated September 5, 2023. Accessed March 8, 2024. https://clinicaltrials.gov/ct2/shaw/NCT04124965?term=MG0004&draw=2&rank=1
  4. A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis. ClinicalTrials.gov identifier: NCT04650854. Updated February 26, 2024. Accessed March 8, 2024. https://clinicaltrials.gov/ct2/show/NCT04650854?term=MG0007&draw=2&rank=1
  5. Data on file. UCB Inc., Smyrna, GA.
  6. MG activities of daily living (MG-ADL) profile. Myasthenia Gravis Foundation of America. 1997. Accessed May 13, 2024. https://myasthenia.org/Portals/0/ADL.pdf

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SAFETY