- UCB will present additional studies and data analyses highlighting UCB's ongoing commitment to improving outcomes for people living with gMG
- Two studies assessed the bioequivalence and effectiveness of zilucoplan auto-injector (ZLP-AI) administration compared with pre-filled syringe (ZLP-PFS)1
- UCB is presenting 21 abstracts in total from its innovative neurology portfolio including 14 epilepsy abstracts and six myasthenia gravis (MG) abstracts at AAN 2026
ATLANTA, April 13, 2026 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced data from two clinical studies assessing ZLP-AI in adult patients who are anti-AChR antibody positivegMG as well as in healthy volunteers. The studies evaluated the effectiveness of a ZLP- AI as a potential new alternative administration option, and the bioequivalence of ZLP-AI relative to ZLP-PFS. ZLP-AI is an investigational administration option for the treatment of gMG in adult patients who are anti-AChR antibody positive and the presentation is currently not approved in the U.S.
DV0013 was a Phase 3b open-label, multicenter study in adult patients with gMG self-administering zilucoplan via auto-injector once daily.1 DV0012 was a Phase 1, open-label, single-center, two-period crossover study in healthy adult volunteers to establish bioequivalence of zilucoplan delivered via an auto-injector pen compared with a pre-filled syringe.1
"While symptom control is of course essential, successfully treating gMG should go beyond symptom management and consider tailored therapies that align with a patient's needs, lifestyle and quality-of-life goals," said Omar Sinno, MD, US Medical Strategy Lead, US Rare Disease.
UCB will also present five additional studies and data analyses, further highlighting UCB's ongoing commitment to improving outcomes for people living with gMG including:
- Response to Rozanolixizumab Across Treatment Cycles in Patients with Generalized Myasthenia Gravis: A Post Hoc Analysis of Final Pooled Phase 3 Data: An analysis evaluated the response to rozanolixizumab over multiple treatment cycles in adult patients with gMG, based on their Cycle 1 response.2
- Sustained Minimal Symptom Expression in Generalized Myasthenia Gravis: A 120-Week Post Hoc Analysis of RAISE-XT: A post hoc analysis assessed the durability of minimal symptom expression (MSE) response during treatment with zilucoplan in patients with gMG.3
- Treatment Goals in Myasthenia Gravis: Expert Consensus Recommendations. Using the RAND/UCLA Appropriateness Method: Seventeen myasthenia gravis experts from North America, Europe and Asia reached consensus on 21 recommendations to define and operationalize treatment goals for patients with MG. Recommendations emphasize the importance of patient engagement and education, reassessing and changing treatment to achieve goals and care coordination with other specialists.4
- Treatment Characteristics and Healthcare Resource Utilization Among Adult Patients With Generalized Myasthenia Gravis Initiating Rozanolixizumab in the United States: This real-world, retrospective, non-interventional cohort study of adult patients (N=719) receiving rozanolixizumab in the US evaluated treatment patterns and healthcare resource utilization (HCRU).5
- Missed Opportunities for Pharmacist Intervention Among Medicare Fee-for-Service Beneficiaries with Myasthenia Gravis: A Real-World Claims Analysis.6
"At UCB, our approach to rare disease goes beyond developing effective medicines; it's about improving overall experience and outcome for patients," said Kimberly Moran, PhD, SVP & Head, US Rare Disease. "By offering innovative solutions designed around patient needs and preferences, we continue in our mission to help people living with rare diseases better manage their condition through targeted therapies."
About generalized myasthenia gravis (gMG)
gMG is a rare neuromuscular autoimmune disease that has a global prevalence of 150-350 cases per 1 million people.7 People living with gMG can experience a variety of symptoms, including severe muscle weakness that can result in double vision, drooping eyelids, difficulty with swallowing, chewing and talking, as well as life-threatening weakness of the muscles of respiration.
In gMG, pathogenic autoantibodies can impair synaptic transmission at the neuromuscular junction (NMJ) by targeting specific proteins on the post-synaptic membrane. This disrupts the ability of the nerves to stimulate the skeletal muscle and results in a weaker contraction. gMG can occur in any race, gender or age.
For further information, contact UCB:
U.S. Communications
Daphne Teo
T +1 (770) 880-7655
[email protected]
Investor Relations
Antje Witte
T +32.2.559.94.14
[email protected]
Sahar Yazdian
T +32.2.559.91.37
[email protected]
Yvonne Naughton
T +44.175.344.7521
[email protected]
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 more than 9 000 people in approximately 40 countries, the company generated revenue of € 7.7 billion in 2025. UCB is listed on Euronext Brussels (symbol: UCB).
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.
Important Safety Information about ZILBRYSQ® (zilucoplan) in the US8
INDICATION
ZILBRYSQ (zilucoplan) is 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
ZILBRYSQ, a complement inhibitor, increases the risk of serious infections caused by Neisseria meningitidis. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early.
- Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of ZILBRYSQ, unless the risks of delaying therapy outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccination against meningococcal bacteria in patients receiving a complement inhibitor.
- Patients receiving ZILBRYSQ are at increased risk for invasive disease caused by Neisseria meningitidis, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious 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 for initiation in patients with unresolved serious Neisseria meningitidis infection.
WARNINGS AND PRECAUTIONS
Serious Meningococcal Infections
ZILBRYSQ, a complement inhibitor, increases a patient's susceptibility to serious, life-threatening, or fatal infections caused by meningococcal bacteria (septicemia and/or meningitis) in any serogroup, including non-groupable strains. Life-threatening and fatal meningococcal infections have occurred in both vaccinated and unvaccinated patients treated with complement inhibitors. The initiation of ZILBRYSQ treatment is contraindicated in patients with unresolved serious Neisseria meningitidis infection.
Complete or update meningococcal vaccination (for serogroups A, C, W, Y and B) at least 2 weeks prior to administration of the first dose of ZILBRYSQ, according to current ACIP recommendations for patients receiving a complement inhibitor.
If urgent ZILBRYSQ therapy is indicated in a patient who is not up to date with meningococcal vaccines according to ACIP recommendations, provide the patient with antibacterial drug prophylaxis and administer meningococcal vaccines as soon as possible.
Closely monitor patients for early signs and symptoms of meningococcal infection and evaluate patients immediately if infection is suspected. Consider interruption of ZILBRYSQ in patients who are undergoing treatment for serious meningococcal infection, depending on the risks of interrupting treatment in the disease being treated.
ZILBRYSQ REMS
Due to the risk of serious 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 serious 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
Serious infections with Neisseria species (other than Neisseria meningitidis), including disseminated gonococcal infections, have been reported in patients treated with complement inhibitors. 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 infection according to ACIP recommendations. Patients receiving ZILBRYSQ are at increased risk for infections due to these organisms, even if they develop antibodies following 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 refer to the full Prescribing Information for additional Important Safety Information.
Important Safety Information about RYSTIGGO® (rozanolixizumab-noli) in the US9
INDICATION
RYSTIGGO (rozanolixizumab-noli) is 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 refer to the full Prescribing Information for additional Important Safety Information.
RYSTIGGO® and ZILBRYSQ® are registered trademarks of the UCB Group of Companies.
©2026 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-RZ-2600254
References:
- Petrulis et al. DV0012 and DV0013 Study Teams. Bioequivalence and effectiveness of auto-injector-delivered, self-administered zilucoplan compared with pre-filled syringe. Abstract presented at the American Academy of Neurology Annual Meeting, April 2026.
- Pascuzzi et al. Response to rozanolixizumab across treatment cycles in patients with generalized myasthenia gravis: A post hoc analysis of final pooled phase 3 data. Abstract presented at the American Academy of Neurology Annual Meeting, April 2026.
- Howard et al. RAISE-XT Study Team. Sustained minimal symptom expression in generalized myasthenia gravis: A 120-week post hoc analysis of RAISE-XT. Abstract presented at the American Academy of Neurology Annual Meeting, April 2026.
- Narayanaswami et?al. Treatment goals in myasthenia gravis: Expert consensus recommendations using the RAND/UCLA Appropriateness Method. Abstract presented at the American Academy of Neurology Annual Meeting, April 2026.
- Kumar et al., Treatment characteristics and healthcare resource utilization among patients with generalized myasthenia gravis initiating rozanolixizumab in the United States. Abstract presented at the American Academy of Neurology Annual Meeting, April 2026.
- Thompson et al. Missed opportunities for pharmacist intervention among Medicare fee-for-service beneficiaries with myasthenia gravis: a real-world claims analysis. Abstract presented at: American Academy of Neurology Annual Meeting, April 2026.
- Rodrigues, E., Umeh, E., Aishwarya, Navaratnarajah, N., Cole, A., & Moy, K. (2024). Incidence and prevalence of myasthenia gravis in the United States: A claims-based analysis. Muscle & nerve, 69(2), 166-171.
- ZILBRYSQ [Prescribing Information]. Smyrna, GA: UCB, Inc.
- RYSTIGGO [Prescribing Information]. Smyrna, GA: UCB, Inc.
SOURCE UCB
