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Explore efficacy1-3

Results from the RAISE and RAISE‑XT clinical trials

Actor portrayal.

RAISE: Primary Endpoint MG-ADL

Rapid and statistically significant improvements in MG-ADL total score at Week 12 were clinically meaningful1,3

In the pivotal Phase 3 RAISE trial, ZILBRYSQ delivered a >4-point improvement in the ability to manage activities of daily living at Week 12 for adults with anti-acetylcholine receptor (AChR) antibody positive (Ab+) generalized myasthenia gravis (gMG).

Primary endpoint: Change from baseline (CFB) in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at Week 12

Primary endpoint: Change from baseline (CFB) at Week 12 in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 12.
Primary endpoint: Change from baseline (CFB) at Week 12 in Myasthenia Gravis Activities of Daily Living (MGADL) total score at week 12.

The most common adverse reactions (reported in at least 10% of patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea.1

CI=confidence interval; SD=standard deviation.

The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included adult patients with diagnosis of mild-to-severe gMG (MGFA class II-IV).1

This kind of potential for meaningful improvement in only 3 months is very appealing to me when I am considering ZILBRYSQ for my patients.”

Watch the video of Dr. Michael Weiss presenting the RAISE and RAISE-XT clinical trials.

 
MGFA=Myasthenia Gravis Foundation of America.

RAISE-XT: Secondary Endpoint MG-ADL

Sustained improvement in MG-ADL total score for over 2 years (Week E108)5,6

The primary endpoint of RAISE-XT evaluated the long-term safety and tolerability of ZILBRYSQ. Please see the results here.

Secondary endpoint: CFB in MG-ADL score in pooled data at Week E108 (mITT population)

Secondary endpoint: CFB in MG-ADL score in pooled data at Week E108 (mITT population).
Secondary endpoint: CFB in MG-ADL score in pooled data at Week E108 (mITT population).

Study Limitations: The open-label extension study was designed to evaluate safety and was not placebo controlled; therefore, efficacy or clinical significance should be interpreted with caution.

The long-term safety, tolerability, and efficacy were evaluated in an open-label extension study comprised of gMG patients who completed either Phase 2 or Phase 3 (RAISE) of the clinical trials.5

RAISE-XT evaluated 200 adult patients from either Phase 2 or 3, including 17 patients from Phase 2 who started with or switched to 0.1 mg/kg. The graphic above shows only the 183 patients continuing or switching to ZILBRYSQ at the approved dosage (0.3 mg/kg) and are solely represented in the subsequent analyses on efficacy. Data cutoff: November 11, 2023.2,5

*Includes pooled data of patients from Week E12-Week E108.6

CI=confidence interval; LS=least squares; mITT=modified intention-to-treat; SE=standard error.

MG-ADL responder rates1,2

A high proportion of patients in either RAISE or RAISE-XT taking ZILBRYSQ were MG-ADL clinical responders (≥3-point improvement from baseline) at Week 12 and Week E108.1,2

Responder rate was specified as an other secondary efficacy endpoint in the RAISE study and an exploratory efficacy endpoint in RAISE-XT. Results should be interpreted with caution.3,5

RAISE: Week 12 73% of patients were MG-ADL responders. RAISE-XT: Week E12 83% of patients were MGADL responders. RAISE-XT: Week E108 88% of patients were MG-ADL responders.
RAISE: Week 12 73% of patients were MG-ADL responders. RAISE-XT: Week E12 83% of patients were MGADL responders. RAISE-XT: Week E108 88% of patients were MG-ADL responders.

Study Limitations: The open-label extension study was designed to evaluate safety and was not placebo controlled; therefore, efficacy or clinical significance should be interpreted with caution.

Includes pooled data of patients from Phases 2 and 3 of the clinical study who either continued on ZILBRYSQ or switched to ZILBRYSQ from placebo. Data cutoff: November 11, 2023.2

Continue minimal symptom expression (MSE) for over 2 years (Week E108)2

Patients who achieved MSE (mITT population) in either RAISE or RAISE-XT achieved an MG-ADL score of 0 or 1 without rescue therapy.5

MSE was defined as an MG-ADL total score of 0-1. MSE was specified as an other secondary efficacy endpoint in the RAISE study and an exploratory endpoint in RAISE-XT. Results should be interpreted with caution.3,5

RAISE: Week 12 14% of patients were MSE responders. RAISE-XT: Week E12 32% of patients were MSE responders. RAISE-XT: Week E108 41% of patients were MSE responders.
RAISE: Week 12 14% of patients were MSE responders. RAISE-XT: Week E12 32% of patients were MSE responders. RAISE-XT: Week E108 41% of patients were MSE responders.

Study Limitations: The open-label extension study was designed to evaluate safety and was not placebo controlled; therefore, efficacy or clinical significance should be interpreted with caution.

Includes pooled data of patients from Week E12-Week E108.2

RAISE-XT evaluated 200 patients from either Phase 2 or 3, including 17 patients from Phase 2 who started with or switched to 0.1 mg/kg. The graphic above shows only the 183 patients continuing or switching to ZILBRYSQ at the approved dosage (0.3 mg/kg) and are solely represented in the subsequent analyses on efficacy. Data cutoff: November 11, 2023.2,5

mITT=modified intention-to-treat.

MG-ADL Guide

Encourage your patients to keep track of gMG symptoms between appointments.

The efficacy and safety of ZILBRYSQ for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive (Ab+) were established in RAISE, a 12-week, multicenter, randomized, double-blind, placebo-controlled study. A total of 174 patients were randomized to receive either ZILBRYSQ (n=86) or placebo (n=88).1,3

SEE STUDY DESIGN

RAISE secondary endpoints

RAISE: improvements across key clinician- and patient-reported secondary and other outcome measures at Week 121,3

Statistically significant and clinically meaningful improvement in muscle strength at Week 121,3

Secondary endpoint: CFB in QMG score at Week 12

RAISE Qualitative MG (QMG) score. Secondary endpoint: CFB in QMG score at Week 12.
RAISE Qualitative MG (QMG) score. Secondary endpoint: CFB in QMG score at Week 12.

The safety and efficacy of ZILBRYSQ were evaluated in a 12-week, multicenter, randomized, double-blind, placebo-controlled study. Patient population included adult patients with diagnosis of mild-to-severe gMG (MGFA class II-IV).1

CFB=change from baseline; CI=confidence interval; MGFA=Myasthenia Gravis Foundation of America.

RAISE secondary endpoint measures overview

RAISE-XT is an extension study of adult patients who opted to continue on ZILBRYSQ or switch to ZILBRYSQ from placebo. The primary objective was to evaluate the long-term safety and tolerability of ZILBRYSQ in study participants with generalized myasthenia gravis (gMG). Long-term efficacy was also studied through multiple measures as secondary endpoints.3

SEE STUDY DESIGN

Additional RAISE-XT secondary endpoints

RAISE-XT: consistent improvements across key clinician- and patient-reported secondary and other outcome measures2‑6

Sustained efficacy in QMG total score for over 2 years (Week E108)3,4

Secondary endpoint: CFB in the QMG total score at Week E108 (mITT population)3*

RAISE-XT Secondary endpoint: CFB in the QMG total score in pooled data at Week E108 (mITT population).
RAISE-XT Secondary endpoint: CFB in the QMG total score in pooled data at Week E108 (mITT population).

Study Limitations: The open-label extension study was designed to evaluate safety and was not placebo controlled; therefore, efficacy or clinical significance should be interpreted with caution.

The long-term safety, tolerability, and efficacy were evaluated in an open-label extension study comprised of gMG patients who participated in either Phase 2 or Phase 3 (RAISE) of the clinical trials.3

RAISE-XT evaluated 200 adult patients from either Phase 2 or 3, including 17 patients from Phase 2 who started with or switched to 0.1 mg/kg. The graphic above shows only the 183 patients continuing or switching to ZILBRYSQ at the approved dosage (0.3 mg/kg) and are solely represented in the subsequent analyses on efficacy. Data cutoff: November 11, 2023.2,3

*Includes pooled data of patients from Week E12-Week E108.4

CFB=change from baseline; CI=confidence interval; LS=least squares; mITT=modified intention-to-treat; SD=standard deviation; SE=standard error.

RAISE-XT other endpoint:

RAISE-XT post hoc data:

RAISE-XT secondary endpoint measures overview

Teacher.
Teacher.

References:

  1. ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Howard JF Jr, Freimer M, Genge A, et al. Response rates with zilucoplan in generalised myasthenia gravis: 120-week interim analysis of RAISE-XT. Presented at: International Congress on Neuromuscular Diseases; October 25-29, 2024; Perth, Australia. Session OS.03.06.
  3. Howard JF Jr, Bresch S, Genge A, et al; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, Phase 3 study. Lancet Neurol. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7
  4. Conquer MG. MG Activities of Daily Living (MG-ADL) scale. September 29, 2022. Accessed November 9, 2023. https://www.myastheniagravis.org/mg-activities-of-daily-living-mg-adl-scale/
  5. Howard JF Jr, Bresch S, Farmakidis C, et al. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. Ther Adv Neurol Disord. 2024;17(3):1-16. doi:10.1177/17562864241243186
  6. Howard JF Jr, Freimer M, Genge A, et al; on behalf of the RAISE-XT Study Team. Long-term safety and efficacy of zilucoplan in generalized myasthenia gravis:120-week interim analysis of RAISE-XT. Poster presented at: American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting & Myasthenia Gravis Foundation of America Scientific Session; October 15-18, 2024; Savannah, GA. Poster 192.
  7. Vu T, Genge A, Hussain Y, et al; on behalf of the RAISE Investigators. Efficacy and safety of zilucoplan in myasthenia gravis: responder analysis from the randomized Phase 3 RAISE trial. Poster presented at: American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting; September 21-24, 2022; Nashville, TN. Poster 200.
  1. ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Howard JF Jr, Freimer M, Genge A, et al. Response rates with zilucoplan in generalised myasthenia gravis: 120-week interim analysis of RAISE-XT. Presented at: International Congress on Neuromuscular Diseases; October 25-29, 2024; Perth, Australia. Session OS.03.06.
  3. Howard JF Jr, Bresch S, Genge A, et al; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, Phase 3 study. Lancet Neurol. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7
  4. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed November 9, 2023. https://myasthenia.org/Portals/0/QMG.pdf
  5. Sadjadi R, Conaway M, Cutter G, et al; MG Composite MG-QOL15 Study Group. Psychometric evaluation of the myasthenia gravis composite using Rasch analysis. Muscle Nerve. 2012;45(6):820-825. doi:10.1002/mus.23260
  6. Myasthenia Gravis Quality of Life-15 Revised (MG-QOL15R). Myasthenia Gravis Rare Disease Network. September 7, 2022. Accessed October 25, 2023. https://mgnet.rarediseasesnetwork.org/sites/default/files/2023-04/mg-quality-life-15-revised.pdf
  1. ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
  2. Howard JF Jr, Freimer M, Genge A, et al. Response rates with zilucoplan in generalised myasthenia gravis: 120-week interim analysis of RAISE-XT. Presented at: International Congress on Neuromuscular Diseases; October 25-29, 2024; Perth, Australia. Session OS.03.06.
  3. Howard JF Jr, Bresch S, Farmakidis C, et al. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. Ther Adv Neurol Disord. 2024;17(3):1-16. doi:10.1177/17562864241243186
  4. Howard JF Jr, Freimer M, Genge A, et al; on behalf of the RAISE-XT Study Team. Long-term safety and efficacy of zilucoplan in generalized myasthenia gravis: 120-week interim analysis of RAISE-XT. Poster presented at: American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting & Myasthenia Gravis Foundation of America Scientific Session; October 15-18, 2024; Savannah, GA. Poster 192.
  5. Howard JF Jr, Freimer M, Genge A, et al; on behalf of the RAISE-XT Study Team. Long-term safety and efficacy of zilucoplan in myasthenia gravis: additional interim analyses of RAISE-XT. Presented at: American Academy of Neurology 2024 Annual Meeting; April 13-18, 2024; Denver, CO. Presentation S15.002.
  6. Howard JF Jr, Bresch S, Genge A, et al; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, Phase 3 study. Lancet Neurol. 2023;22(5):395-406. doi:10.1016/S1474-4422(23)00080-7
  7. Data on file. UCB, Inc.
  8. Data on file. UCB, Inc.
  9. Leite MI, Bresch S, Hewamadduma C, et al; on behalf of the RAISE-XT Study Team. Effect of zilucoplan on rescue therapy use in patients with generalised myasthenia gravis: RAISE-XT post hoc analysis. Poster presented at: 11th Congress of the European Academy of Neurology; June 21-25, 2025; Helsinki, Finland. Poster EPO-475.
  10. Hewamadduma C, Bresch S, Freimer M, et al; on behalf of the RAISE-XT Study Team. Sustained minimal symptom expression in generalised myasthenia gravis: a 120-week post hoc analysis of RAISE-XT. Poster presented at: European Academy of Neurology Conference; June 21-24, 2025; Helsinki, Finland. Poster EPO-470.
  11. Hewamadduma C, Freimer M, Genge A, et al; on behalf of the RAISE-XT Study Team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025;272(7):1-9. doi.org/10.1007/s00415-025-13113-0
  12. QMG form. Myasthenia Gravis Foundation of America. 1997. Accessed November 9, 2023. https://myasthenia.org/Portals/0/QMG.pdf
  13. Sadjadi R, Conaway M, Cutter G, et al; MG Composite MG-QOL15 Study Group. Psychometric evaluation of the myasthenia gravis composite using Rasch analysis. Muscle Nerve. 2012;45(6):820-825. doi:10.1002/mus.23260
  14. Myasthenia Gravis Quality of Life-15 Revised (MG-QOL15R). Myasthenia Gravis Rare Disease Network. September 7, 2022. Accessed October 25, 2023. https://mgnet. rarediseasesnetwork.org/sites/default/files/2023-04/mg-quality-life-15-revised.pdf