UCB showcases new data for gMG management at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and MGFA Scientific Session

Brussels (Belgium), 11 Oct 2024: 07:00 (CET) - UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that it will be presenting results from across its portfolio in generalized myasthenia gravis (gMG) at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) annual meeting and the Myasthenia Gravis Foundation of America (MGFA) Scientific Session taking place between October 15 - 18, 2024 at the Savannah Convention Center in Georgia, U.S.

A total of 14 abstracts, including one oral presentation, will feature data from studies of UCB’s recently approved medicines for the treatment of gMG, RYSTIGGO^®▼ and ZILBRYSQ^®▼, along with findings from real-world studies of clinical outcomes and patient experience in gMG. 

“At UCB, we are committed to transforming the lives of those living with generalized myasthenia gravis through ongoing innovation and comprehensive clinical research”, commented Manuela Maronati, Global Asset Head, Neuroimmunology & Rare. “Our presentations at the 2024 AANEM Annual Meeting and MGFA Scientific Session underscore our dedication to advancing understanding and treatment of this challenging condition. We are particularly excited to share insights from our RAISE-XT and MycarinG studies, which reflect significant progress in the management of generalized myasthenia gravis.”

Key UCB scientific and real-world data to be presented at AANEM and the MGFA Scientific Session include: 

• An oral presentation focusing on UCB’s innovative work on enhancing global education standards for myasthenia gravis.⁵
• Phase 3b study results on switching to subcutaneous zilucoplan from IV complement component 5 inhibitors in myasthenia gravis.⁶ 
• Data from the RAISE-XT trial for zilucoplan - including an interim analysis of long-term safety and efficacy over a period of up to 120 weeks, compliance to daily self-administered subcutaneous zilucoplan, and a post-hoc analysis of corticosteroid sparing and non-steroidal immunosuppressant therapy changes up to 120 weeks - offering new insights into the management potential for generalized myasthenia gravis in a clinical setting.^7,8,9
• For rozanolixizumab, post hoc analyses from the Phase 3 MycarinG trial in patients with generalized myasthenia gravis describe the impact of treatment on specific muscle group weaknesses, evaluate rozanolixizumab in those aged 65 years and older, and highlight its use for individualized treatment regimens.^10,11,12

UCB will also host an industry-sponsored therapeutic update session on an expert-led discussion on generalized myasthenia gravis treatment choices on October 16, 2024.

“At this year's AANEM Annual Meeting and MGFA Scientific Session, we are proud to share data from UCB’s innovative work, which is advancing the standard of education for generalized myasthenia gravis globally. This program not only underscores our dedication to empowering healthcare professionals with knowledge and tools but also highlights our ongoing commitment to addressing the complex challenges faced by the MG community," commented Emmanuel Caeymaex, Executive Vice President, Head of Patient Impact, UCB. "

UCB presentations during AANEM and MGFA 2024

Additionally, for registered healthcare professionals, UCB has arranged for a program of sponsored scientific and therapeutic updates. Visit us at our booth for more information. 

UCB
For further information, contact UCB: 

Global Communications
Nick Francis
T: +44 7769 307745
nick.francis@ucb.com 

Rare Disease Communications
Daphne Teo
T +1 (770) 880-7655
daphne.teo@ucb.com 

Corporate Communications, Media Relations
Laurent Schots 
T +32.2.559.92.64 
Laurent.schots@ucb.com 

Investor Relations
Antje Witte   
T +32.2.559.94.14 
antje.witte@ucb.com

Important Safety Information about RYSTIGGO^®▼ (rozanolixizumab) in the EU³

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. 

Rystiggo is indicated as an add-on to standard therapy for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.³ 

The most commonly reported adverse reactions were headache (48.4 %), diarrhea (25.0 %) and pyrexia (12.5 %). The adverse reactions from the placebo-controlled study in gMG are as follows: Very common (≥ 1/10) headache, diarrhea and pyrexia; Common (≥ 1/100 to < 1/10) rash, angioedema, arthralgia and injection site reactions. Very common (≥1/10) headache, diarrhoea, and pyrexia; Common (≥1/100 to <1/10) rash, angioedema, arthralgia, and injection site reactions; Not known, aseptic meningitis (from spontaneous post-marketing reporting). In MG0003, headache was the most common reaction reported in 31 (48.4%) and 13 (19.4%) of the patients treated with rozanolixizumab and placebo, respectively. All headaches, except 1 (1.6%) severe headache, were either mild (28.1% [n=18]) or moderate (18.8% [n=12]) and there was no increase in incidences of headache with repeated cyclic treatment.

Rozanolixizumab is contra-indicated in patients with hypersensitivity to the active substance or to any of the excipients.

Treatment with rozanolixizumab in patients with impending or manifest myasthenic crisis has not been studied. The sequence of therapy initiation between established therapies for MG crisis and rozanolixizumab, and their potential interactions, should be considered.

Aseptic meningitis (drug induced aseptic meningitis) has been reported following rozanolixizumab treatment at a higher dose with subsequent recovery without sequelae after discontinuation. If symptoms consistent with aseptic meningitis occur, diagnostic workup and treatment should be initiated as per standard of care.

Due to its mechanism of action, the use of rozanolixizumab may increase the patient’s susceptibility to infections. Treatment with rozanolixizumab should not be initiated in patients with a clinically important active infection until the infection is resolved or is adequately treated. During treatment with rozanolixizumab, clinical signs and symptoms of infections should be monitored. If a clinically important active infection occurs, withholding rozanolixizumab until the infection has resolved should be considered. 

Hypersensitivity reactions including mild to moderate rash or angioedema were observed in patients treated with rozanolixizumab. Patients should be monitored during treatment with rozanolixizumab and for 15 minutes after the administration is complete for clinical signs and symptoms of hypersensitivity reactions. If a hypersensitivity reaction occurs during administration, rozanolixizumab infusion should be discontinued and appropriate measures should be initiated if needed. Once resolved, administration may be resumed.

Immunization with vaccines during rozanolixizumab therapy has not been studied. The safety of immunization with live or live-attenuated vaccines and the response to immunization with vaccines are unknown. All vaccines should be administered according to immunization guidelines and at least 4 weeks before initiation of treatment. For patients that are on treatment, vaccination with live or live attenuated vaccines is not recommended. For all other vaccines, they should take place at least 2 weeks after the last infusion of a treatment cycle and 4 weeks before initiating the next cycle.

Refer to the European Summary of Product Characteristics for other adverse reactions and full prescribing information. https://www.ema.europa.eu/en/documents/product-information/rystiggo-epar-product-information_en.pdf. 

Important Safety Information about ZILBRYSQ^®▼ (zilucoplan) in the EU¹

▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. 

Zilbrysq is indicated as an add-on to standard therapy for the treatment of generalized myasthenia
gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.¹ 

The most frequently reported adverse reactions were injection site reactions (injection site bruising (13.9%) and injection site pain (7.0%)) and upper respiratory tract infections (nasopharyngitis (5.2%), upper respiratory tract infection (3.5%) and sinusitis (3.5%)). The adverse reactions from the pooled placebo controlled (n=115) and open-label extension (n=213) studies in gMG are as follows: Very common adverse reactions: (≥ 1/10): Upper respiratory tract infections and Injection site reactions; Common adverse reactions (≥ 1/100 to < 1/10) Diarrhea, Lipase increased, Amylase increased and Morphea; Uncommon adverse reaction (≥ 1/1000 to < 1/100) blood eosinophils increased. 

Zilucoplan is contra-indicated in patients with hypersensitivity to the active substance or to any of the excipients, in patients who are not currently vaccinated against Neisseria meningitidis and in patients with unresolved Neisseria meningitidis infection. 

Due to its mechanism of action, the use of zilucoplan may increase the patient’s susceptibility to infections with Neisseria meningitidis. As a precautionary measure, all patients must be vaccinated against meningococcal infections, at least 2 weeks prior to the start of treatment. If treatment needs to start less than 2 weeks after vaccination against meningococcal infections, the patient must receive appropriate prophylactic antibiotic treatment until 2 weeks after the first vaccination dose. Meningococcal vaccines reduce but do not completely eliminate the risk of meningococcal infections. Vaccines against serogroups A, C, Y, W, and where available, serogroup B, are recommended for preventing the commonly pathogenic meningococcal serogroups. Vaccination and prophylactic antibiotic treatment should occur according to most current relevant guidelines. During treatment, patients should be monitored for signs and symptoms of meningococcal infection and evaluated immediately if infection is suspected. In case of a suspected meningococcal infection, appropriate measures such as treatment with antibiotics and discontinuation of treatment, should be taken until the meningococcal infection can be ruled out. Patients should be instructed to seek immediate medical advice if signs or symptoms of meningococcal infections occur. Prescribers should be familiar with the educational materials for the management of meningococcal infections and provide a patient alert card and patient/carer guide to patients treated with zilucoplan. 

In addition to Neisseria meningitidis, patients treated with zilucoplan may also be susceptible to infections with other Neisseria species, such as gonococcal infections. Patients should be informed on the importance of gonorrhea prevention and treatment. 

Prior to initiating zilucoplan therapy, it is recommended that patients initiate immunizations according to current immunization guidelines. 

Refer to the European Summary of Product Characteristics for other adverse reactions and full prescribing information. https://www.ema.europa.eu/en/documents/product-information/zilbrysq-epar-product-information_en.pdf. EC Date of approval 01 Dec 2023.

IMPORTANT SAFETY INFORMATION ABOUT RYSTIGGO^® (rozanolixizumab-noli) in the US⁴

INDICATION:
RYSTIGGO (rozanolixizumab-noli) is a neonatal Fc receptor blocker indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive.4 

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS 
Infections: RYSTIGGO may increase the risk of infection. Delay RYSTIGGO administration in patients with an active infection until the infection is resolved. During treatment with RYSTIGGO, monitor for clinical signs and symptoms of infection. If serious infection occurs, administer appropriate treatment and consider withholding RYSTIGGO until the infection has resolved.

Immunization
Immunization with vaccines during RYSTIGGO treatment has not been studied. The safety of immunization with live or live-attenuated vaccines and the response to immunization with any vaccine are unknown. Because RYSTIGGO causes a reduction in IgG levels, vaccination with live-attenuated or live vaccines is not recommended during treatment with RYSTIGGO. Evaluate the need to administer age-appropriate vaccines according to immunization guidelines before initiation of a new treatment cycle with RYSTIGGO.

Aseptic Meningitis: Serious adverse reactions of aseptic meningitis (also called drug-induced aseptic meningitis) have been reported in patients treated with RYSTIGGO. If symptoms consistent with aseptic meningitis develop, diagnostic workup and treatment should be initiated according to the standard of care.

Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and rash, were observed in patients treated with RYSTIGGO. Management of hypersensitivity reactions depends on the type and severity of the reaction. Monitor patients during treatment with RYSTIGGO and for 15 minutes after for clinical signs and symptoms of hypersensitivity reactions. If a reaction occurs, institute appropriate measures if needed.

ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of RYSTIGGO-treated patients) were headache, infections, diarrhea, pyrexia, hypersensitivity reactions, and nausea. Serious infections were reported in 4% of patients treated with RYSTIGGO. Three fatal cases of pneumonia were identified, caused by COVID-19 infection in two patients and an unknown pathogen in one patient. Six cases of infections led to discontinuation of RYSTIGGO.

Please see the full Prescribing Information for additional Important Safety Information.

IMPORTANT SAFETY INFORMATION ABOUT ZILBRYSQ^® (zilucoplan) in the US² 

INDICATION:
ZILBRYSQ is a complement inhibitor indicated for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.² 

IMPORTANT SAFETY INFORMATION INCLUDING BOXED WARNING

WARNING: SERIOUS MENINGOCOCCAL INFECTIONS
Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors; ZILBRYSQ is a complement inhibitor. Meningococcal infection may become rapidly life-threatening or fatal if not recognized and treated early.

• Complete or update meningococcal vaccination (for serogroups A, C, W, and Y, and serogroup B) at least 2 weeks prior to administering the first dose of ZILBRYSQ, unless the risk of delaying therapy outweighs the risk of developing a meningococcal infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for meningococcal vaccinations in patients receiving a complement inhibitor.
• Persons receiving ZILBRYSQ are at increased risk for invasive disease caused by N. meningitidis, even if they develop antibodies following vaccination. Monitor patients for signs of meningococcal infections and evaluate immediately if infection is suspected.

Because of the risk of serious meningococcal infections, ZILBRYSQ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called ZILBRYSQ REMS.

CONTRAINDICATIONS
ZILBRYSQ is contraindicated in patients with unresolved Neisseria meningitidis infection.

WARNINGS AND PRECAUTIONS
Serious Meningococcal Infections

Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors; ZILBRYSQ is a complement inhibitor. The use of ZILBRYSQ increases a patient’s susceptibility to serious and life-threatening meningococcal infections (septicemia and/or meningitis) caused by any serogroup, including non-groupable strains.

Complete or update meningococcal vaccination (for both serogroups A, C, W, and Y [MenACWY] and serogroup B [MenB]) at least 2 weeks prior to administering the first dose of ZILBRYSQ, according to current ACIP recommendations for meningococcal vaccinations in patients receiving a complement inhibitor.

If urgent ZILBRYSQ therapy is indicated in a patient who is not up to date with both MenACWY and MenB vaccines according to ACIP recommendations, administer meningococcal vaccine(s) as soon as possible and provide the patient with antibacterial drug prophylaxis.

Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Withhold administration of ZILBRYSQ in patients who are undergoing treatment for meningococcal infection until the infection is resolved.

ZILBRYSQ REMS
Due to the risk of meningococcal infections, ZILBRYSQ is available only through a restricted program under a REMS called ZILBRYSQ REMS.

Under the ZILBRYSQ REMS, prescribers must enroll in the program. Prescribers must counsel patients about the risk of meningococcal infection, provide the patients with the REMS educational materials, and ensure patients are vaccinated with meningococcal vaccines. Additional information on the REMS requirements is available at www.ZILBRYSQREMS.com or 1-877-414-8353.

Other Infections
ZILBRYSQ blocks terminal complement activation; therefore, patients may have increased susceptibility to infections, especially with encapsulated bacteria, such as infections caused by Neisseria meningitidis but also Streptococcus pneumoniae, Haemophilus influenzae, and to a lesser extent, Neisseria gonorrhoeae. Administer vaccinations for the prevention of Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) infections according to ACIP guidelines. Persons receiving ZILBRYSQ are at increased risk for infections due to these bacteria, even after vaccination.

Pancreatitis And Other Pancreatic Conditions
Pancreatitis and pancreatic cysts have been reported in patients treated with ZILBRYSQ. Patients should be informed of this risk before starting ZILBRYSQ. Obtain lipase and amylase levels at baseline before starting treatment with ZILBRYSQ. Discontinue ZILBRYSQ in patients with suspected pancreatitis and initiate appropriate management until pancreatitis is ruled out or has resolved.

ADVERSE REACTIONS
In a placebo-controlled study, the most common adverse reactions (reported in at least 10% of gMG patients treated with ZILBRYSQ) were injection site reactions, upper respiratory tract infections, and diarrhea.

Please see the full Prescribing Information for additional Important Safety Information.

About UCB 
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9 000 people in approximately 40 countries, the company generated revenue of € 5.3 billion in 2023. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

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Given these uncertainties, you should not place undue reliance on any of such forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labelling in any market, or at any particular time, nor can there be any guarantee that such products will be or will continue to be commercially successful in the future.

UCB is providing this information, including forward-looking statements, only as of the date of this press release and it does not reflect any potential impact from the evolving COVID-19 pandemic, unless indicated otherwise. UCB is following the worldwide developments diligently to assess the financial significance of this pandemic to UCB. UCB expressly disclaims any duty to update any information contained in this press release, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. 

Additionally, information contained in this document shall not constitute an offer to sell or the solicitation of an offer to buy any securities, nor shall there be any offer, solicitation or sale of securities in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such jurisdiction. 

References

1. ZILBRYSQ^® EU SmPC. https://www.ema.europa.eu/en/documents/product-information/zilbrysq-epar-product-information_en.pdf. (Accessed: October 2024).
2. ZILBRYSQ^® US PI. https://www.ucb.com/sites/default/files/2024-01/Zilbrysq_PI_27oct2023.pdf. (Accessed: October 2024).
3. RYSTIGGO^® EU SmPC. https://www.ema.europa.eu/en/documents/product-information/rystiggo-epar-product-information_en.pdf. (Accessed: October 2024).
4. RYSTIGGO^® US PI. https://www.ucb.com/sites/default/files/2023-08/Rystiggo_Prescribing_Information_USA.pdf. (Accessed: October 2024).
5. Howard J, et al. Developing needs-driven medical education for healthcare professionals in myasthenia gravis. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
6. Freimer M, et al. Switching to subcutaneous zilucoplan from IV complement component 5 inhibitors in myasthenia gravis: A Phase 3b study. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
7. Howard J, et al. Long-term safety and efficacy of zilucoplan in generalized myasthenia gravis: 120-week interim analysis of RAISE-XT. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
8. Ruzhansky K, et al. Compliance to daily self-administered subcutaneous zilucoplan in patients with generalized myasthenia gravis: a post hoc analysis of the RAISE-XT study. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
9. Freimer M, et al. Corticosteroid dose tapering during treatment with zilucoplan in patients with generalized myasthenia gravis: 120-week follow-up of RAISE-XT. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
10. Pascuzzi R, et al. Effect of rozanolixizumab on myasthenia gravis–specific outcome subdomain scores: post hoc analyses from the phase 3 MycarinG study. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
11. Vu T, et al. Rozanolixizumab in patients aged ≥65 years with generalized myasthenia gravis: a post hoc analysis of the Phase 3 MycarinG study. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.
12. Ali A. Habib, et al. Rozanolixizumab treatment patterns in patients with generalized myasthenia gravis: post hoc analysis. Poster presented at: The American Academy of Neurology and Electrodiagnostic Medicine Annual Meeting (AANEM); 2024, October 15-18; Georgia, USA.