Sanofi’s venglustat met all primary endpoints in a phase 3 study of type 3 Gaucher disease

赛诺菲的维格鲁司他（venglustat）在3型戈谢病的3期研究中达到了所有主要终点。

In the LEAP2MONO phase 3 study, venglustat, dosed orally once daily, demonstrated clinically meaningful efficacy in patients with type 3 Gaucher disease (GD3), a rare lysosomal storage disorder

在 LEAP2MONO 三期研究中，口服每日一次的 venglustat 在患有罕见溶酶体贮积症的三型戈谢病 (GD3) 患者中显示出具有临床意义的疗效。

Venglustat demonstrated superiority versus enzyme replacement therapy in addressing neurological symptoms in GD3, for which there are no approved treatments

Venglustat在解决GD3的神经系统症状方面表现出优于酶替代疗法的效果，目前尚无获批的治疗方法。

Sanofi will pursue global regulatory submissions for GD3

赛诺菲将为GD3寻求全球监管机构的批准。

In the PERIDOT phase 3 study in Fabry disease, venglustat did not show superiority on the patient-reported primary endpoint and an additional phase 3 CARAT study is ongoing

在法布里病的PERIDOT三期研究中，venglustat在患者报告的主要终点上未显示出优越性，另一项三期CARAT研究正在进行中。

Paris, February 2, 2026.

巴黎，2026年2月2日。

Positive results from the LEAP2MONO phase 3 study (clinical study identifier:

LEAP2MONO 3期研究（临床研究标识符：）的积极结果

NCT05222906

NCT05222906

) demonstrated that venglustat met the primary and three out of four key secondary endpoints in adults and pediatric patients (12 years and older) with neurological manifestations of type 3 Gaucher disease (GD3), a rare lysosomal storage disorder.

）证明了维格鲁司他达到了3型戈谢病（GD3）成人和儿科患者（12岁及以上）的主要终点和四个关键次要终点中的三个，这是一种罕见的溶酶体贮积症。

Venglustat, which works by reducing the abnormal accumulation of sugar-and-fat molecules in cells and organs, is an investigational glucosylceramide synthase inhibitor (GCSi) that crosses the blood-brain barrier with the goal of targeting some of the neurological aspects of GD3 that currently have no approved therapies.

Venglustat是一种在研的葡萄糖基神经酰胺合成酶抑制剂（GCSi），通过减少细胞和器官中糖脂分子的异常积累发挥作用，并能穿越血脑屏障，目标是针对目前尚无批准疗法的GD3的一些神经学方面。

Sanofi has supported the Gaucher disease community for decades as part of an over 40-year commitment to improving care for rare diseases..

赛诺菲作为承诺改善罕见疾病护理的一部分，几十年来一直支持戈谢病社区，这一承诺已超过40年。

The results will be shared this week at the 22nd annual WORLD

结果将在本周的第22届年度WORLD会议上分享

Symposium

研讨会

™ as late-breaking research.

™ 作为最新的研究。

'These findings underscore Sanofi's commitment to rare disease research and the promise we aim to deliver for people living with these conditions,' said

“这些发现强调了赛诺菲对罕见病研究的承诺，以及我们为患有这些疾病的患者提供希望的决心，”

Houman

侯曼

Ashrafian

阿什拉菲安

, Executive Vice President and Head of Research and Development at Sanofi. 'What excites us most is the potential to address critical unmet medical needs. A daily pill could make a serious difference for Gaucher patients facing neurological challenges. Most importantly, none of this would be possible without the courage of the patients and families who participate in our studies, and for that we owe them a debt of gratitude.”.

赛诺菲执行副总裁兼研发主管。“最让我们兴奋的是能够满足关键的未满足医疗需求。一种每日一次的药片可能会对面临神经挑战的戈谢病患者产生重大影响。最重要的是，如果没有参与我们研究的患者和家属的勇气，这一切都不可能实现，对此我们深表感激。”

In the LEAP2MONO study, GD3 patients receiving venglustat demonstrated statistically significant improvements in neurological symptoms measured by a global test score for two assessments, the Scale for Assessment and Rating of Ataxia (SARA) modified total score and the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS), at week 52 compared with those receiving enzyme replacement therapy (ERT) (p=0.007).

在 LEAP2MONO 研究中，接受 venglustat 治疗的 GD3 患者在第 52 周时，通过两项评估的全球测试评分显示出神经系统症状的统计学显著改善，这两项评估分别是改良的共济失调评估与评级量表（SARA）总分和可重复使用的神经心理状态评估电池（RBANS），相较于接受酶替代疗法（ERT）的患者（p=0.007）。

Venglustat performed as well as ERT on non-neurological outcomes, including changes in spleen volume, liver volume, and hemoglobin levels, three key secondary endpoints of the study. .

Venglustat在非神经学结果（包括脾脏体积、肝脏体积和血红蛋白水平的变化）方面表现与ERT一样好，这三项是研究的关键次要终点。

Venglustat is also being studied for the treatment of Fabry disease, another rare lysosomal storage disorder. Data from the phase 3 PERIDOT study (clinical study identifier:

Venglustat 还在研究用于治疗另一种罕见的溶酶体贮积症——法布里病。来自 3 期 PERIDOT 研究的数据（临床研究标识符：

NCT05206773

NCT05206773

) show that reduction in neuropathic and abdominal pain was observed in both study arms and the primary endpoint was not met. Additional analyses of the data are ongoing with more information to be shared at a future medical meeting. A second phase 3 study, CARAT (clinical study identifier:

）显示神经性疼痛和腹部疼痛的减轻在两个研究组中均被观察到，但主要终点未达到。数据的更多分析正在进行中，更多信息将在未来的医学会议上分享。第二项三期研究CARAT（临床研究标识符：

NCT05280548

NCT05280548

), evaluating the effect of venglustat on left cardiac ventricular mass index in men and women with Fabry disease, is ongoing.

），评估venglustat对法布里病男性和女性左心室质量指数的影响，正在进行中。

Venglustat was well tolerated overall with no new safety signals compared with previous studies. In LEAP2MONO, the most commonly reported adverse events during treatment among patients receiving venglustat (21 individuals) compared with those receiving ERT (22 individuals) were headache (14.3% in the venglustat arm versus 18.2% in the ERT arm), nausea (14.3% versus 4.5%), spleen enlargement (14.3% versus 0), and diarrhea (14.3% versus 0)..

Venglustat总体耐受性良好，与之前的研究相比没有出现新的安全性信号。在LEAP2MONO研究中，接受venglustat治疗的患者（21人）最常见的不良事件为头痛（venglustat组14.3%，ERT组18.2%）、恶心（14.3% vs 4.5%）、脾脏肿大（14.3% vs 0）和腹泻（14.3% vs 0），而接受酶替代疗法（ERT）的患者有22人。

Sanofi will pursue global regulatory filings for venglustat in GD3. Venglustat is investigational, and its safety and efficacy have not been evaluated by any regulatory authority.

赛诺菲将为venglustat在GD3中的应用继续进行全球监管申报。Venglustat为研究性药物，其安全性和有效性尚未经过任何监管机构的评估。

Sanofi currently markets Fabrazyme, an ERT for Fabry disease, and Cerezyme and Cerdelga for Gaucher disease, an ERT and oral therapy, respectively, in markets around the world. In January 2026, the US approved an expanded label for Cerezyme to include non-central nervous system (CNS) manifestations of GD3, building on its long-standing approval in the US for GD1.

赛诺菲目前在全球市场销售法布里病的酶替代疗法（ERT）药物Fabrazyme，以及戈谢病的Cerezyme和Cerdelga，分别作为酶替代疗法和口服治疗药物。2026年1月，美国批准了Cerezyme的扩展标签，将GD3的非中枢神经系统（CNS）表现纳入其中，这基于其长期以来在美国对GD1的批准。

This US supplemental label expansion was based exclusively on real-world evidence and leveraged data from the International Collaborative Gaucher Group Gaucher Registry. With the US label expansion, Cerezyme can now be prescribed globally to patients with either GD1 or GD3..

此次美国补充标签扩展完全基于真实世界证据，并利用了国际戈谢病合作组戈谢病登记处的数据。随着美国标签的扩展，Cerezyme现在可以全球范围内处方给GD1或GD3患者。

About Gaucher disease

关于戈谢病

Gaucher disease (GD) is a rare inherited lysosomal storage disease that results from a deficiency of an enzyme called glucocerebrosidase (also known as acid β-glucosidase), leading to the accumulation of molecules called glycosphingolipids (GSLs), particularly in macrophages of the spleen, liver, bone marrow, and lungs.

戈谢病 (GD) 是一种罕见的遗传性溶酶体贮积症，由于一种名为葡萄糖脑苷脂酶（又称酸性β-葡萄糖苷酶）的酶缺乏，导致称为糖鞘脂 (GSLs) 的分子在脾脏、肝脏、骨髓和肺部的巨噬细胞中特别积累。

There are three major forms within the clinical spectrum of Gaucher disease: GD1, which is characterized by the lack of (or late) central nervous system (CNS) involvement; GD2, which is the acute neuronopathic form; and GD3, which is the chronic neuronopathic form. In people with GD3, accumulation of GSLs in the CNS can result in neurological manifestations, including ataxia and cognitive deficits, in addition to the systemic manifestations seen in GD1, such as liver and spleen enlargement, anemia, thrombocytopenia, or bone disease.

戈谢病的临床谱系中有三种主要形式：GD1，其特征是缺乏（或晚期）中枢神经系统 (CNS) 受累；GD2，为急性神经病变型；GD3，为慢性神经病变型。在 GD3 患者中，CNS 中糖脂 (GSL) 的积累可能导致神经系统表现，包括共济失调和认知缺陷，此外还有 GD1 中可见的系统性表现，例如肝脾肿大、贫血、血小板减少或骨病。

Systemic manifestations of GD3 are treated with ERT, but there are no approved treatments for neurologic manifestations of GD3..

GD3的系统性表现使用ERT治疗，但目前尚无获批的GD3神经学表现的治疗方法。

About Fabry disease

关于法布里病

Fabry disease is an inherited rare lysosomal storage disease that results from a deficiency of functional alpha-galactosidase A (α-Gal A), leading to a build-up of GSLs, causing progressive cellular accumulation and organ damage in the kidney, cardiovascular and cerebrovascular systems. Neuropathic pain, abdominal pain and other gastrointestinal symptoms are among the first clinical manifestations of Fabry disease and can substantially impact activities of daily living.

法布里病是一种遗传性罕见的溶酶体贮积病，由功能性α-半乳糖苷酶A（α-Gal A）缺乏引起，导致糖鞘脂（GSLs）积累，造成肾脏、心血管和脑血管系统中的细胞渐进性堆积和器官损伤。神经性疼痛、腹痛及其他胃肠道症状是法布里病的早期临床表现，可能严重影响日常生活活动。

Clinically, there are two major subtypes: the more severe classic phenotype that presents in childhood with little or no functional α-Gal A enzymatic activity and the non-classic phenotype that presents later in life, typically in adulthood. Non-classic patients have residual α-Gal A activity but progressively accumulate GSLs..

临床上，该病分为两种主要亚型：一种是较为严重的经典表型，患者在儿童期发病，α-Gal A酶活性几乎完全缺失；另一种是非经典表型，通常在成年期发病。非经典型患者保留部分α-Gal A酶活性，但会逐渐积累糖鞘脂（GSLs）。

About venglustat

关于venglustat

Venglustat is a novel, investigational oral glucosylceramide synthase inhibitor (GCSi), designed to cross the blood brain barrier (i.e., brain-penetrant), that has the potential to slow the progression of certain diseases by inhibiting abnormal GSL accumulation and its physio-pathologic consequences.

Venglustat 是一种新型的研究性口服葡萄糖基神经酰胺合成酶抑制剂 (GCSi)，设计为可穿越血脑屏障（即能够进入大脑），具有通过抑制异常糖脂 (GSL) 积累及其生理病理后果来减缓某些疾病进展的潜力。

GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases leading to both cell dysfunction and disease progression. Venglustat was previously granted orphan designation in the EU, the USA and Japan for its potential treatment of both GD3 and Fabry disease. It also received fast-track designation in the US Food & Drug Administration (FDA) for its potential use in GD3 and Fabry disease..

GSLs是细胞构建块，其异常积累与多种罕见疾病有关，导致细胞功能障碍和疾病进展。Venglustat之前在欧盟、美国和日本获得了孤儿药资格认定，用于潜在治疗GD3和法布里病。它还获得了美国食品药品监督管理局（FDA）的快速通道资格认定，用于GD3和法布里病的潜在应用。

About the LEAP2MONO study

关于LEAP2MONO研究

The LEAP2MONO phase 3 study was a double-blind, double-dummy, active-comparator, two-arm study that evaluated the efficacy and safety of once daily oral venglustat versus intravenous ERT every two weeks in adults and pediatric patients aged 12 and older with GD3. Forty-three patients were randomized [1:1] to receive venglustat and placebo infusion or ERT and placebo tablet.

LEAP2MONO 第三阶段研究是一项双盲、双模拟、活性对照的两组研究，评估了每日一次口服维格鲁司他与每两周一次静脉注射ERT在12岁及以上的成人和儿童GD3患者中的疗效和安全性。43名患者被随机分配 [1:1] 接受维格鲁司他和安慰剂输注或ERT和安慰剂片。

Patients must have been treated with ERT for at least three years and achieved therapeutic goals for systemic disease manifestations. The primary endpoints for the study was change in SARA modified total score and change in RBANS total scale index score for patients receiving venglustat versus those receiving ERT from baseline to week 52.

患者必须接受过至少三年的ERT治疗，并达到全身性疾病表现的治疗目标。该研究的主要终点是从基线到第52周，接受venglustat治疗的患者与接受ERT治疗的患者在SARA改良总分和RBANS总分指数的变化。

Systemic key secondary endpoints include percent change in spleen volume, liver volume and platelet count and change in hemoglobin levels. Biomarker key secondary endpoints include percent change in cerebrospinal fluid and plasma GL1 and lyso-GL1. The LEAP2MONO study is ongoing and results from its open-label phase will be presented in the future when available..

系统性关键次要终点包括脾脏体积、肝脏体积和血小板计数的百分比变化以及血红蛋白水平的变化。生物标志物关键次要终点包括脑脊液和血浆中GL1和lyso-GL1的百分比变化。LEAP2MONO研究正在进行中，其开放标签阶段的结果将在未来有数据时公布。

About the PERIDOT study

关于PERIDOT研究

The PERIDOT phase 3 study was a double-blind, randomized, placebo-controlled study that evaluated the efficacy and safety of venglustat on neuropathic and abdominal pain in patients aged 16 years and older with Fabry disease. The study randomized 122 patients 1:1 to receive venglustat or placebo. Patients must have been treatment-naïve or untreated for at least six months.

PERIDOT 第三阶段研究是一项双盲、随机、安慰剂对照的研究，评估了venglustat对16岁及以上法布里病患者的神经性和腹部疼痛的疗效和安全性。该研究将122名患者以1:1的比例随机分配接受venglustat或安慰剂治疗。患者必须是未曾接受过治疗或至少六个月未接受治疗的。

The primary endpoint was the percent change from baseline on patient-defined most bothersome symptoms (neuropathic pain in upper extremities, neuropathic pain in lower extremities or abdominal pain), as assessed by the FD-PRO instrument, in those treated with venglustat versus placebo. Secondary endpoints include percent change from baseline in plasma globotriaosylsphingosine (lyso-GL-3), frequency of rescue pain medication use, change from baseline in the percentage of days with diarrhea, percent change from baseline in tiredness component of FD-PRO, and proportion of responders on the patient-defined most bothersome symptoms..

主要终点是由FD-PRO工具评估的，与安慰剂相比，接受venglustat治疗的患者自基线以来最困扰症状（上肢神经性疼痛、下肢神经性疼痛或腹痛）的百分比变化。次要终点包括血浆globotriaosylsphingosine（lyso-GL-3）自基线的百分比变化、急救止痛药使用的频率、腹泻天数比例自基线的变化、FD-PRO疲倦部分自基线的百分比变化，以及患者定义的最困扰症状反应者的比例。

About Sanofi

关于赛诺菲

Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time..

赛诺菲是一家以研发为驱动、以人工智能为助力的生物制药公司，致力于改善人们的生活并实现引人注目的增长。我们凭借对免疫系统的深刻理解，发明能够治疗和保护全球数百万人的药物和疫苗，并通过创新的研发管线使数百万人进一步受益。我们的团队秉承一个使命：追寻科学的奇迹以改善人们的生活；这激励我们推动进步，为员工及所服务的社区带来积极影响，同时应对当今最紧迫的医疗、环境和社会挑战。

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

赛诺菲在 EURONEXT（欧元区股票交易所）上市，代码为 SAN；并在 NASDAQ（纳斯达克）上市，代码为 SNY。

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Sanofi forward-looking statements

赛诺菲前瞻性声明

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions, and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance.

本新闻稿包含1995年《私人证券诉讼改革法案》（经修订）所定义的前瞻性陈述。前瞻性陈述并非历史事实。这些陈述包括预测、估计及其基本假设，关于未来财务结果、事件、运营、服务、产品开发和潜力的计划、目标、意图和期望的陈述，以及关于未来表现的陈述。

Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements.

前瞻性声明通常可以通过“预期”、“预计”、“相信”、“打算”、“估计”、“计划”等词语来识别。尽管赛诺菲的管理层认为这些前瞻性声明中反映的预期是合理的，但投资者应注意，前瞻性信息和声明受到各种风险和不确定性的约束，其中许多难以预测且通常超出赛诺菲的控制范围，可能导致实际结果和发展与这些前瞻性信息和声明中表达或暗示的内容有重大差异。

These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and com.

这些风险和不确定性包括但不限于研发、未来临床数据及分析（包括上市后）中固有的不确定性，监管机构（如FDA或EMA）的决定，涉及是否以及何时批准可能为任何此类候选产品提交的任何药物、器械或生物制品申请，以及他们关于标签和其他可能影响此类候选产品的可用性或商业潜力的事宜的决定，候选产品即便获得批准也可能无法取得商业成功的事实，未来的批准和商业化进程等。

All trademarks mentioned in this press release are the property of the Sanofi group.

本新闻稿中提到的所有商标均为赛诺菲集团的财产。

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