UCB在第15届美国重症肌无力基金会（MGFA）国际会议上展示了庞大的数据集，突显了其对改善gMG治疗结果和体验的承诺-动脉网

UCB presents expansive data set at 15th Myasthenia Gravis Foundation of America (MGFA) International Conference highlighting commitment to advancing gMG treatment outcomes and experiences

优时比等信源发布 2025-05-13 13:07



可切换为仅中文







Brussels (Belgium), 13 May 2025, 07:00 (CET)

布鲁塞尔（比利时），2025年5月13日，07:00（欧洲中部时间）

UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that it will be presenting multiple important data sets from across its targeted treatment portfolio in generalized myasthenia gravis (gMG) at the Myasthenia Gravis Foundation of America (MGFA) International Conference taking place May 13-15, 2025, at The Hague, The Netherlands..

UCB（布鲁塞尔泛欧交易所：UCB），一家全球生物制药公司，今天宣布将在2025年5月13日至15日于荷兰海牙举行的美国重症肌无力基金会（MGFA）国际会议上，展示来自其针对广义重症肌无力（gMG）的靶向治疗组合的多个重要数据集。

Across a total of 21 abstracts, data will be featured from studies of UCB’s medicines for the treatment of gMG in adult patients; RYSTIGGO

在总共21篇摘要中，将展示UCB公司针对成年gMG患者治疗药物的研究数据；RYSTIGGO

▼(rozanolixizumab) and ZILBRYSQ

▼（罗扎诺利珠单抗）和 ZILBRYSQ

▼ (zilucoplan), along with findings from real-world studies of clinical outcomes and patient experience in gMG.

▼（zilucoplan），以及来自真实世界研究的临床结果和患者体验的发现。

“Our presentations at the 15th MGFA Conference underscore our dedication to advancing understanding and treatment of generalized myasthenia gravis. We are excited to share new data sets on the long-term efficacy and safety of rozanolixizumab, and the early and sustained response over time with zilucoplan,” said Donatello Crocetta, Chief Medical Officer, UCB.

“我们在第15届MGFA会议上所做的报告，彰显了我们对推进全身型重症肌无力理解和治疗的承诺。我们很高兴分享关于rozanolixizumab长期有效性和安全性的新数据集，以及zilucoplan随时间推移的早期和持续反应，”UCB首席医学官唐纳泰罗·克罗切塔表示。

“In line with our commitment to patient-centered care, we are proud that UCB has funded and is the first subscriber to the Vitaccess Real MG Registry”. .

“秉承我们以患者为中心的承诺，我们为UCB资助并成为Vitaccess Real MG注册库的首个订阅者感到自豪。”

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

将在MGFA会议上展示的关键UCB科学和真实世界数据包括：

Two final pooled analyses of the Phase 3 MycarinG study, and the Open-Label Extension Studies, focusing on the efficacy and safety of rozanolixizumab over multiple symptom-driven cycles in patients with generalized MG.

两项最终汇总分析来自第 3 阶段 MycarinG 研究和开放标签扩展研究，重点关注 rozanolixizumab 在全身性 MG 患者中多次症状驱动周期内的疗效和安全性。

1,2

A 120-week post hoc analysis of RAISE-XT, which examines early and sustained response over time with zilucoplan in the treatment of generalized myasthenia gravis.

RAISE-XT 的一项 120 周事后分析，该研究评估了 zilucoplan 在治疗全身性重症肌无力过程中随时间推移的早期和持续反应。

Findings from the Phase 3, open-label, crossover (MG0020) study evaluating patient preferences, experiences and safety of self-administered rozanolixizumab.

评估患者对自我给药的rozanolixizumab的偏好、体验和安全性的3期、开放标签、交叉（MG0020）研究结果。

Presentation of the new Vitaccess Real MG (VRMG) Registry international patient registry design for myasthenia gravis.

介绍新的Vitaccess真实MG（VRMG）登记册国际患者登记册设计，用于重症肌无力。

An evaluation of the effect of Zilucoplan on Myasthenia Gravis Activities of Daily Living (MG-ADL) scale and Quantitative Myasthenia Gravis (QMG) ocular subdomain scores in patients with generalized myasthenia gravis in RAISE and RAISE-XT.

评估Zilucoplan对RAISE和RAISE-XT中全身性重症肌无力患者的重症肌无力日常生活活动（MG-ADL）量表和定量重症肌无力（QMG）眼部子域评分的影响。

A poster for a matching-adjusted indirect comparisons (MAIC) study of zilucoplan versus eculizumab and ravulizumab in the treatment of generalized myasthenia gravis (gMG).* The findings are expected to provide insights into the potential for zilucoplan to offer improved outcomes.

一份针对zilucoplan与eculizumab和ravulizumab在治疗全身性重症肌无力（gMG）中的匹配调整间接比较（MAIC）研究的海报。* 研究结果预计将为zilucoplan提供改善疗效的潜力提供见解。

* The findings based on indirect comparisons should be interpreted with caution

基于间接比较的结果应谨慎解读。

UCB presentations at MGFA 2025

UCB在MGFA 2025的演讲

UCB

For further information, contact UCB:

如需更多信息，请联系UCB：

Global Communications

全球通讯

Nick Francis

尼克·弗朗西斯

T: +44 7769 307745

电话：+44 7769 307745

nick.francis@ucb.com

Rare Disease Communications

罕见疾病交流

Daphne Teo

黛芬妮·张

T +1 (770) 880-7655

电话：+1 (770) 880-7655

daphne.teo@ucb.com

Corporate Communications, Media Relations

企业传播、媒体关系

Laurent Schots

劳伦特·肖茨

T +32.2.559.92.64

电话：+32.2.559.92.64

Laurent.schots@ucb.com

Investor Relations

投资者关系

Antje Witte

安特耶·维特

T +32.2.559.94.14

电话：+32.2.559.94.14

antje.witte@ucb.com

Important Safety Information about RYSTIGGO

关于RYSTIGGO的重要安全信息

▼ (rozanolixizumab) in the EU

▼ （rozanolixizumab）在欧盟

▼

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.

该药品受到额外的监控。这将有助于快速识别新的安全信息。医疗专业人员被要求报告任何可疑的不良反应。

The most commonly reported adverse reactions were headache (48.4%), diarrhoea (25.0%) and pyrexia (12.5%). The adverse reactions from clinical studies in gMG are as follows: Very common (≥1/10) headache, diarrhoea, and pyrexia; Common (≥1/100 to <1/10) upper respiratory tract infections including cases of nasopharyngitis, rash, angioedema, arthralgia, and injection site reactions; Not known, aseptic meningitis (from spontaneous post-marketing reporting).

最常见的不良反应为头痛（48.4%）、腹泻（25.0%）和发热（12.5%）。gMG临床研究中的不良反应如下：非常常见（≥1/10）头痛、腹泻和发热；常见（≥1/100 至 <1/10）上呼吸道感染，包括鼻咽炎病例、皮疹、血管性水肿、关节痛和注射部位反应；未知，无菌性脑膜炎（来自自发的上市后报告）。

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.

在MG0003中，头痛是最常见的反应，分别有31例（48.4%）和13例（19.4%）的患者在使用rozanolixizumab和安慰剂治疗时报告。除了1例（1.6%）严重头痛外，所有头痛均为轻度（28.1% [n=18]）或中度（18.8% [n=12]），且随着重复周期性治疗，头痛发生率没有增加。

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. Aseptic meningitis (drug induced aseptic meningitis) has been reported following rozanolixizumab treatment.

罗扎诺利珠单抗禁用于对活性成分或任何辅料过敏的患者。尚未研究罗扎诺利珠单抗治疗即将发生或已显现的肌无力危象患者的情况。有报告称，在使用罗扎诺利珠单抗治疗后出现无菌性脑膜炎（药物引起的无菌性脑膜炎）。

If symptoms consistent with aseptic meningitis (headache, pyrexia, neck stiffness, nausea, vomiting) occur, diagnostic workup and treatment should be initiated as per standard of care. As rozanolixizumab causes transient reduction in IgG levels the risk of infections may increase. Treatment with rozanolixizumab should not be initiated in patients with a clinically important active infection until the infection resolves or is adequately treated.

如果出现与无菌性脑膜炎（头痛、发热、颈项强直、恶心、呕吐）一致的症状，应按照护理标准启动诊断和治疗。由于罗扎诺利珠单抗会导致IgG水平短暂下降，感染风险可能会增加。在临床上重要的活动性感染患者中，不应开始使用罗扎诺利珠单抗治疗，直至感染消退或得到适当治疗。

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 .

在使用罗扎诺利珠单抗治疗期间，应监测感染的临床体征和症状。如果出现重要的活动性感染，应暂停罗扎诺利珠单抗，直至感染得到解决。

Important Safety Information about ZILBRYSQ

关于ZILBRYSQ的重要安全信息

▼ (zilucoplan) in the EU

▼ （zilucoplan）在欧盟

▼

该药品受到额外的监测。这将有助于快速识别新的安全信息。医疗专业人员被要求报告任何可疑的不良反应。

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%)). Zilucoplan is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients.

最常见的不良反应是注射部位反应（注射部位瘀伤 (13.9%) 和注射部位疼痛 (7.0%)）以及上呼吸道感染（鼻咽炎 (5.2%)、上呼吸道感染 (3.5%) 和鼻窦炎 (3.5%)）。Zilucoplan 禁用于对活性成分或任何辅料过敏的患者。

The most common reactions were injection site bruising, pain, nodule, pruritus and haematoma. All cases were mild or moderate in severity, and less than 3% of reactions led to treatment discontinuation. The most common infections were nasopharyngitis, upper respiratory tract infection and sinusitis.

最常见的反应是注射部位瘀伤、疼痛、结节、瘙痒和血肿。所有病例的严重程度均为轻度或中度，不到3%的反应导致治疗中断。最常见的感染是鼻咽炎、上呼吸道感染和鼻窦炎。

More than 95% of the cases were mild or moderate in severity and did not lead to treatment discontinuation. In pooled placebo-controlled studies, upper respiratory tract infections were reported in 13.0% of patients treated with zilucoplan and in 7.8% of patients treated with placebo. Cases of lipase increase (5.2%) and/or amylase increase (6.1%) were observed.

超过 95% 的病例严重程度为轻度或中度，且并未导致治疗中断。在汇总的安慰剂对照研究中，接受 zilucoplan 治疗的患者中有 13.0% 报告出现上呼吸道感染，而接受安慰剂治疗的患者中有 7.8% 报告出现上呼吸道感染。观察到脂肪酶升高（5.2%）和/或淀粉酶升高（6.1%）的病例。

These elevations were transient and rarely led to treatment discontinuation. The majority occurred within 2 months of starting zilucoplan and normalized within 2 months. Elevations of blood eosinophils were observed. These were transient and not leading to treatment discontinuation. The majority occurred within 2 months of starting zilucoplan and normalized within 1 month.

这些升高是暂时的，很少导致治疗中断。大多数发生在开始使用zilucoplan后的2个月内，并在2个月内恢复正常。观察到嗜酸性粒细胞升高。这些升高是暂时的，并未导致治疗中断。大多数发生在开始使用zilucoplan后的2个月内，并在1个月内恢复正常。

Cases of morphoea were observed after long-term treatment during the open-label extension study. The majority of the cases had a time to onset longer than one year after start of treatment, were mild or moderate in severity and did not lead to treatment discontinuation. Due to its mechanism of action, the use of zilucoplan may increase the patient’s susceptibility to in.

在开放标签延长期研究中观察到硬斑病病例。大多数病例的发病时间在治疗开始后超过一年，严重程度为轻度或中度，并未导致治疗中断。由于其作用机制，使用zilucoplan可能会增加患者对感染的易感性。

About UCB

关于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 € 6.1 billion in 2024.

优时比公司（UCB），位于比利时布鲁塞尔（www.ucb.com），是一家全球生物制药公司，专注于发现和开发创新药物和解决方案，以改变免疫系统或中枢神经系统严重疾病患者的生活。该公司在约40个国家拥有9000多名员工，并在2024年实现了61亿欧元的收入。

UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

优时比在布鲁塞尔泛欧交易所上市（股票代码：UCB）。请在推特上关注我们：@UCB_news。

Forward looking statements

前瞻性声明

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.

本新闻稿可能包含前瞻性陈述，包括但不限于包含“相信”、“预期”、“预计”、“打算”、“计划”、“寻求”、“估计”、“可能”、“将”、“继续”等词语及类似表达的陈述。这些前瞻性陈述基于管理层当前的计划、估计和信念。

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.

所有声明，除历史事实声明外，均可能被视为前瞻性声明，包括收入、营业利润率、资本支出、现金、其他财务信息、预期法律、仲裁、政治、监管或临床结果或实践以及其他此类估计和结果。

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.

由于此类前瞻性陈述的性质，其并非未来业绩的保证，并且受已知和未知风险、不确定性及假设的影响，这可能导致本新闻稿中包含的前瞻性陈述所表达或暗示的UCB的实际结果、财务状况、业绩或成就，或行业结果，与之存在重大差异。

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 .

可能导致此类差异的重要因素包括：新冠疫情的全球传播和影响、总体经济、商业和竞争状况的变化、无法获得必要的监管批准或以可接受的条件或在预期时间内获得这些批准、与研发相关的成本、UCB在研产品或开发中产品的前景变化、未来司法裁决或政府调查的影响、安全性、质量、数据完整性或生产问题；潜在或实际的数据安全和隐私泄露，或我们信息技术系统的中断、产品责任。

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..

鉴于这些不确定性，您不应过分依赖任何此类前瞻性声明。无法保证本新闻稿中描述的在研或已获批产品将会在任何市场提交或获批销售，或获得任何额外的适应症或标签，或在任何特定时间获批，也无法保证此类产品在未来将会或将继续取得商业成功。

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仅在本新闻稿发布之日提供此信息，包括前瞻性声明，除非另有说明，否则并未反映不断演变的COVID-19大流行可能带来的影响。UCB正在密切关注全球发展，以评估这场大流行对UCB的财务重要性。

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. .

UCB明确表示，除非适用法律法规要求，否则不承担更新本新闻稿中任何信息的责任，无论这些信息是为了确认实际结果，还是为了报告或反映与其前瞻性声明相关的任何变化，或任何此类声明所基于的事件、条件或情况的变化。

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. .

此外，本文件中包含的信息不构成出售或征求购买任何证券的要约，也不得在任何此类要约、征求或销售属违法的司法管辖区进行证券的要约、征求或销售，除非已根据该司法管辖区的证券法进行注册或取得资格。

Vu, et al. Responder and MSE Rates with Rozanolixizumab in gMG: Final Pooled Analysis of MycarinG and Open-Label Extension Studies.

Vu 等人。 Rozanolixizumab 在 gMG 中的应答率和 MSE 率：MycarinG 和开放标签扩展研究的最终汇总分析。

Habib, et al. Long-Term Efficacy and Safety of Rozanolixizumab Treatment Cycles in Patients With gMG: Final Pooled Analysis of MycarinG and Open-Label Extension Studies.

Habib 等。Rozanolixizumab 治疗周期在 gMG 患者中的长期疗效与安全性：MycarinG 及开放标签扩展研究的最终汇总分析。

Hewamadduma, et al. Early and Sustained Response Over Time with Zilucoplan in gMG: 120-Week Post Hoc Analysis of RAISE-XT.

Hewamadduma 等。Zilucoplan 在 gMG 中的早期和持续时间响应：RAISE-XT 的 120 周事后分析。

Antozzi, et al. Patient Preferences and Experience With Self-Administration of Rozanolixizumab in gMG: The MG0020 Study.

安托齐等人。gMG患者自我管理罗扎诺利珠单抗的偏好与体验：MG0020研究。

Amini, et al. A Novel International Patient Registry in Myasthenia Gravis Linking Clinical and Patient-Reported Outcomes Data: The Vitaccess Real MG (VRMG) Registry.

阿米尼等人。重症肌无力中链接临床和患者报告结果数据的新型国际患者登记：Vitaccess真实MG（VRMG）登记。

Leite, et al. Improvement of Ocular Subdomain Scores With Zilucoplan in Patients With gMG in RAISE and RAISE-XT Studies.

Leite 等。在 RAISE 和 RAISE-XT 研究中，Zilucoplan 对 gMG 患者眼部亚域评分的改善。

Betts, et al. Zilucoplan versus eculizumab and ravulizumab for treating generalised myasthenia gravis: matching-adjusted indirect comparisons.

贝茨等。Zilucoplan 与 Eculizumab 和 Ravulizumab 治疗全身性重症肌无力：匹配调整后的间接比较。

RYSTIGGO

瑞斯蒂戈

EU SmPC.

欧盟药品说明书。

https://www.ema.europa.eu/en/documents/product-information/rystiggo-epar-product-information_en.pdf

. Accessed April 2025.

. 2025年4月访问。