Rozanolixizumab

UCB will contribute 14 presentations, including an oral presentation, selected to feature across the AANEM and MGFA meeting, emphasizing UCB's leadership in neuromuscular research Presentations showcase new data for UCB's gMG treatments, including post hoc analyses highlighting the long-term safety and efficacy of ZILBRYSQ® (zilucoplan)1 and RYSTIGGO® (rozanolixizumab-noli) 2 BRUSSELS, Oct. 11, 2024 /PRNewswire/ -- 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 October 15—18, 2024 at the Savannah Convention Center in Savannah, 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.3 Phase 3b study results on switching to subcutaneous zilucoplan from IV complement component 5 inhibitors in myasthenia gravis.4 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.5,6,7 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.8,9,10 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 a program of sponsored scientific and therapeutic updates. Visit us at our booth for more information. UCB For further information, contact UCB: U.S. Rare Disease Communications Daphne Teo T +1 (770) 880-7655 [email protected] Global Communications Nick Francis T: +44 7769 307745 [email protected] Corporate Communications, Media Relations Laurent Schots T +32.2.559.92.64 [email protected] Investor Relations Antje Witte T +32.2.559.94.14 [email protected] IMPORTANT SAFETY INFORMATION ABOUT RYSTIGGO® (rozanolixizumab-noli) in the US2 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. 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 US1 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. 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 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 () 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: @UCBUSA Forward looking statements This press release may contain forward-looking statements including, without limitation, statements containing the words "believes", "anticipates", "expects", "intends", "plans", "seeks", "estimates", "may", "will", "continue" and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guarantees of future performance and are subject to known and unknown risks, uncertainties and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to differ materially from those that may be expressed or implied by such forward-looking statements contained in this press release. Important factors that could result in such differences include: the global spread and impact of COVID-19, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring and retention of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, will progress to product approval or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products, which are the subject of partnerships, joint ventures or licensing collaborations may be subject to differences disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. 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® US PI. . (Accessed: October 2024). 2 RYSTIGGO® US PI. . (Accessed: October 2024). 3 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. 4 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. 5 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. 6 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. 7 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. 8 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. 9 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. 10 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. RYSTIGGO® and ZILBRYSQ® are registered trademarks of the UCB Group of Companies. ©2024 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-RZ-2400542 SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

On August 29, 2024, Johnson & Johnson formally filed a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) seeking the world's first approval of the FcRn monoclonal antibody Nipocalimab for the treatment of patients with generalized myasthenia gravis (gMG).   In June, the Phase III VIVACITY-MG3 study of Nipocalimab for gMG published positive results, showing that the combination of Nipocalimab with standard of care (SOC) significantly improved MG-ADL scores over 24 weeks compared with placebo-combined SOC, meeting the study's primary endpoint.   The study was a double-blind, placebo-controlled trial that enrolled a large number of patients with AChR+, MuSK+, and/or LRP4+, who accounted for approximately 95% of the total number of patients with gMG. At weeks 22, 23, and 24, patients receiving Nipocalimab+SOC improved MG-ADL scores by 4.70 points from baseline, which was significantly higher than 3.25 points in the placebo +SOC group (P=0.002). For people with gMG, a 1 to 2 point change in the MG-ADL score can mean the difference from a normal diet to frequent food choking, or from shortness of breath at rest to needing to use a ventilator.   In addition, the study achieved several key secondary endpoints: at week 22 and 24, the Nipocalimab+SOC group showed significant improvements in strength and function across different muscle groups assessed by QMG compared to the placebo +SOC group (P<0.001); At weeks 22, 23, and 24, the MG-ADL response was significantly higher in the Nipocalimab+SOC group (≥2 points improvement from baseline) than in the placebo +SOC group (P=0.021), further highlighting the potential of Nipocalimab to mitigate the impact of gMG on patients' daily lives.   Nipocalimab is a high-affinity, fully human, glycated, non-effective monoclonal antibody that selectively blocks FcRn, thereby reducing circulating immunoglobulin G (IgG) antibodies, including autoantibodies and alloantibodies that cause a variety of diseases.   Nipocalimab was originally developed by AnaptysBi and subsequently transferred to Momenta. In August 2020, Johnson & Johnson acquired Momenta for $6.5 billion, giving it the rights to the drug.   gMG is a chronic, lifelong, rare, and highly disabling autoantibody-mediated neuromuscular disease characterized by fluctuating myasthenia. Two FcRn monoclonal antibodies have previously been approved by the FDA, Argenx/ Re-ting's efgartigimod and UXbi's rozanolixizumab, both approved for gMG indications. Egamod has shown good market prospects, 2022 is its first full year of sales in overseas markets, sales exceeded 400 million US dollars, and 2023 reached 1.19 billion US dollars, among the "blockbuster" drugs.