・S-606001 has the potential to become the first oral substrate reduction therapy for Pompe disease

・S-606001有潜力成为首个用于庞贝病的口服底物减少疗法。

OSAKA, Japan, March 19, 2026

日本大阪，2026年3月19日

– Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) announced the first patients were enrolled in Esprit, a global Phase 2 clinical trial evaluating S-606001, an investigational drug for the treatment of late-onset Pompe disease (LOPD)..

盐野义制药株式会社（总部：日本大阪；首席执行官：手代木功博士；以下简称“盐野义”）宣布，已在Esprit中招募了首批患者。Esprit是一项全球性的二期临床试验，旨在评估S-606001这种用于治疗晚发型庞贝病（LOPD）的在研药物。

Esprit is a multicenter, randomized, placebo-controlled, double-blind study evaluating the safety, pharmacodynamics and preliminary efficacy of S-606001 as an oral substrate reduction therapy (SRT) in addition to standard of care enzyme replacement therapy (ERT) in adults with a confirmed diagnosis of LOPD..

Esprit是一项多中心、随机、安慰剂对照、双盲研究，评估S-606001作为口服底物减少疗法（SRT）在标准护理酶替代疗法（ERT）基础上，用于确诊为LOPD的成年人中的安全性、药效学和初步疗效。

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This 52-week study will enroll participants across the U.S., European Union and United Kingdom.

这项为期 52 周的研究将在美国、欧盟和英国招募参与者。

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Pompe disease is a rare genetic metabolic disorder that presents in children and adults.

庞贝病是一种罕见的遗传性代谢疾病，儿童和成人均可发病。

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In people with Pompe disease, a deficiency of the acid alpha-glucosidase (GAA), an enzyme necessary for the breakdown of glycogen, results in the accumulation of glycogen in tissues throughout the body, especially in muscles.

在庞贝病患者中，酸性α-葡萄糖苷酶（GAA）的缺乏是一种必需用于分解糖原的酶，导致糖原在全身各组织中积累，尤其是在肌肉中。

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In LOPD, GAA activity is partially reduced.

在LOPD中，GAA活性部分降低。

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This can cause severe weakness and respiratory issues leading to respiratory insufficiency, wheelchair dependency and a shortened lifespan.

这可能会导致严重的虚弱和呼吸问题，进而引发呼吸功能不全、依赖轮椅以及寿命缩短。

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LOPD affects about one in every 22,000 people worldwide and may be identified at any age.

LOPD 在世界范围内大约每 22,000 人中就有一人受到影响，并且可能在任何年龄被识别出来。

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Despite significant progress in diagnosis in countries with newborn screening programs, identifying LOPD in people who are not screened at birth remains challenging.

尽管在实施新生儿筛查项目的国家中，诊断方面取得了显著进展，但在未进行出生筛查的人群中识别晚发型庞贝病（LOPD）仍然具有挑战性。

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Its rarity, wide range of clinical presentations, and overlap with other neuromuscular disorders often lead to delays in diagnosis.

其罕见性、广泛的临床表现以及与其他神经肌肉疾病的重叠往往导致诊断延误。

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S-606001 is an investigational SRT that is believed to work by limiting glycogen buildup in muscle lysosome by inhibiting glycogen synthase (GYS1).

S-606001 是一种研究性 SRT，据信其通过抑制糖原合酶 (GYS1) 来限制肌肉溶酶体中的糖原积累而发挥作用。

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ERT, the current approved treatment for Pompe disease, infuses more GAA enzyme to increase glycogen breakdown.

ERT，目前获批的庞贝病治疗方法，通过注入更多的GAA酶来促进糖原分解。

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SRT blocks the GYS1 enzyme to slow down glycogen buildup.

SRT抑制GYS1酶以减缓糖原积累。

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Because SRT targets the opposite side of the glycogen buildup problem from ERT, it has the potential to work alone or in combination with ERT.

由于SRT针对的是糖原积累问题的另一面，它有可能单独起作用或与ERT联合使用。

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“The Pompe community is greatly appreciative of Shionogi’s commitment to developing new treatment options for people living with late-onset Pompe disease. Each person deserves alternatives to help them best manage their condition,” said Brad Crittenden, Chairman, International Pompe Association and Executive Director, Canadian Association of Pompe..

“庞贝氏症社区非常感谢盐野义致力于为晚发性庞贝氏症患者开发新的治疗方案。每个人都应该有选择，以帮助他们更好地管理自己的病情，”国际庞贝协会主席兼加拿大庞贝协会执行董事布拉德·克里滕登说道。

“Currently, ERTs are the standard of care for LOPD, but their efficacy can wane over time, leading to continued decline in skeletal muscle function. There is a significant unmet need for new treatment approaches that can be complementary to existing treatments to further slow disease progression,” said Juan Carlos Gomez, M.D., Chief Medical Officer, Shionogi & Co., Ltd.

“目前，ERT 是 LOPD 的标准治疗方法，但其疗效可能会随着时间的推移而减弱，导致骨骼肌功能持续下降。对于能够补充现有治疗并进一步延缓疾病进展的新疗法，仍然存在显著的未满足需求。” Shionogi & Co., Ltd. 首席医学官 Juan Carlos Gomez 医学博士表示。

“This is an important milestone for Shionogi, as we continue to expand our work in rare disease, and we hope it will prove to be an important step forward for the Pompe community.”.

“这对Shionogi来说是一个重要的里程碑，因为我们继续扩展在罕见病领域的工作，我们希望这将证明是庞贝病社区向前迈出的重要一步。”

Shionogi acquired exclusive worldwide rights for S-606001 (previously known as MZE001) from Maze Therapeutics, Inc. in 2024. In 2025, S-606001 received a rare pediatric disease designation from the U.S. Food and Drug Administration (FDA) for the treatment of Pompe disease, a designation granted for serious and life-threatening diseases that primarily affect children ages 18 years or younger with fewer than 200,000 people in the U.S.

2024年，盐野义制药从Maze Therapeutics公司获得了S-606001（之前称为MZE001）的全球独占权利。2025年，S-606001获得美国食品药品监督管理局（FDA）授予的罕见儿科疾病认定，用于治疗庞贝病。该认定针对主要影响18岁及以下儿童且在美国患者少于20万人的严重或危及生命的疾病。

The FDA also granted Orphan Drug Designation to the compound in 2022..

FDA 还在 2022 年授予该化合物孤儿药资格。

Additional details about Esprit are available at

有关Esprit的更多详细信息，请访问

ClinicalTrials.gov ID: NCT07123155

ClinicalTrials.gov ID: NCT07123155

and on

并且继续

www.espritstudy.com

www.espritstudy.com

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。

S-606001 is currently under investigation in clinical trials for the treatment of late-onset Pompe disease. The safety and effectiveness of S-606001 have not been established, nor has it been approved by FDA or any health authority.

S-606001 正在针对晚发型庞贝病的治疗进行临床试验研究。S-606001 的安全性和有效性尚未确定，也未获得 FDA 或任何卫生机构的批准。

About Shionogi in Rare Disease

关于盐野义在罕见病领域

Shionogi is committed to the research and development of innovative medicines that address unmet medical needs for people worldwide. Rare diseases affect individuals and families around the world and treatment options for rare diseases are often limited. Shionogi is advancing clinical programs for rare diseases and disorders including Fragile X syndrome, Jordan’s Syndrome and Pompe disease.

盐野义致力于研究和开发满足全球人民未满足医疗需求的创新药物。罕见疾病影响着世界各地的个人和家庭，而罕见疾病的治疗选择通常有限。盐野义正在推进包括脆性X综合征、乔丹综合征和庞贝病等罕见疾病和障碍的临床项目。

In December 2025, Shionogi announced plans to expand its rare disease portfolio through the acquisition of all global rights, including in Japan and the United States, to the treatment for amyotrophic lateral sclerosis (ALS) developed and marketed by Tanabe Pharma Co., Ltd..

2025年12月，盐野义制药宣布计划通过收购田边制药株式会社开发并销售的肌萎缩侧索硬化症（ALS）治疗药物的全球权利（包括日本和美国）来扩展其罕见病产品组合。

About Shionogi & Co., Ltd.

关于盐野义制药株式会社

Shionogi & Co., Ltd. is a 148-year-old global, research-driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain, CNS disorders and cardiovascular diseases.  Shionogi’s research and development currently targets two therapeutic areas: infectious diseases and diseases with unmet medical needs in pain/CNS, including Alzheimer’s disease, oncology, rare diseases, and sleep apnea. For more information on Shionogi & Co., Ltd., please visit .

盐野义制药株式会社是一家拥有148年历史的全球性研发驱动型制药公司，总部位于日本大阪，秉承“为患者提供尽可能最好的药物以保护我们服务的患者的健康和福祉”的企业理念，致力于为患者带来益处。公司目前在多个治疗领域销售产品，包括抗感染药、镇痛药、中枢神经系统疾病和心血管疾病。盐野义的研发目前专注于两个治疗领域：传染病以及疼痛/中枢神经系统中未满足医疗需求的疾病，包括阿尔茨海默病、肿瘤学、罕见病和睡眠呼吸暂停。欲了解有关盐野义制药株式会社的更多信息，请访问。

https://www.shionogi.com/global/en

https://www.shionogi.com/global/en

.

。

Forward-Looking Statements

前瞻性声明

This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate.

本公告包含前瞻性陈述。这些陈述是基于当前可获得的信息和预期，而这些假设受风险和不确定性的影响，可能导致实际结果与这些陈述有重大差异。风险和不确定性包括一般国内和国际经济状况，如一般行业和市场状况，以及利率和汇率的变化。

These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations.

这些风险和不确定性尤其适用于与产品相关的前瞻性声明。产品风险和不确定性包括但不限于：临床试验的完成和终止；获得监管批准；有关产品安全性和有效性的索赔和顾虑；技术进步；重要诉讼的不利结果；国内外医疗改革以及法律法规的变化。

Also for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise..

对于现有产品，也存在生产和营销风险，包括但不限于无法建立产能满足需求、原材料供应不足以及竞争产品进入市场。公司否认任何更新或修改任何前瞻性声明的意图或义务，无论是否由于新信息、未来事件或其他原因。

For Further Information, Contact:

如需更多信息，请联系：

SHIONOGI Website Inquiry Form:

SHIONOGI网站咨询表：

https://www.shionogi.com/global/en/contact.html

https://www.shionogi.com/global/en/contact.html

U.S. Media:

美国媒体：

ShionogiCommunications@shionogi.com

ShionogiCommunications@shionogi.com

EU Media:

欧盟媒体：

pressoffice@shionogi.eu

pressoffice@shionogi.eu

References

参考文献

1. Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants with Late-onset Pompe Disease (LOPD). Clinicaltrials.gov. Available at:

1. S-606001作为酶替代疗法（ERT）的补充疗法在晚发型庞贝病（LOPD）患者中的研究。Clinicaltrials.gov。可访问：

https://clinicaltrials.gov/study/NCT07123155?intr=S-606001%20&rank=1

https://clinicaltrials.gov/study/NCT07123155?intr=S-606001%20&rank=1

2. Pompe disease. Boston Children’s Hospital. Available at:

2. 庞贝病。波士顿儿童医院。可访问：

https://www.childrenshospital.org/conditions-treatments/pompe-disease

https://www.childrenshospital.org/conditions-treatments/pompe-disease

3. Musumeci O, & Toscano A. Diagnostic tools in late onset Pompe disease (LOPD).

3. Musumeci O, & Toscano A. 晚发型庞贝病（LOPD）的诊断工具。

Annals of Translational Medicine

转化医学年鉴

. 2019 Jul 15;

. 2019年7月15日；

7

7

(13):286–286.

(13):286–286。

https://doi.org/10.21037/atm.2019.06.60

https://doi.org/10.21037/atm.2019.06.60

4. Pompe disease - symptoms, causes,

4. 庞贝病 - 症状，原因，

treatment. National Organization for Rare Disorders. Available at:

治疗。国家罕见疾病组织。可用地址：

https://rarediseases.org/rare-diseases/pompe-disease/

https://rarediseases.org/rare-diseases/pompe-disease/

5. Colburn R, & Lapidus D. An analysis of Pompe newborn screening data: A new prevalence at birth, insight and discussion.

5. Colburn R, & Lapidus D. 庞贝病新生儿筛查数据的分析：新的出生时发病率、见解与讨论。

Frontiers in Pediatrics

儿科前沿

. 2024 Jan 7;(

. 2024年1月7日;(

11)

11)

.

。

https://doi.org/10.3389/fped.2023.1221140

https://doi.org/10.3389/fped.2023.1221140

6. Van der Ploeg AT, & Reuser AJJ. Pompe’s Disease.

6. 范德普洛格 AT，& 雷乌瑟 AJJ。庞贝病。

The Lancet.

柳叶刀。

2008 Oct 11;372(9646): 1342-1353.

2008年10月11日；372（9646）：1342-1353。

https://doi.org/10.1016/s0140-6736(08)61555-x

https://doi.org/10.1016/s0140-6736(08)61555-x

7. Huang D, & Mozaffar T. Misdiagnosis of late-onset Pompe Disease: A case series.

7. 黄D，莫扎法尔T。晚发型庞贝病的误诊：病例系列。

Neurology

神经学

. 2023 Apr 25;100(17_supplement_2).

. 2023年4月25日；100（17_补充_2）。

https://doi.org/10.1212/wnl.0000000000201775

https://doi.org/10.1212/wnl.0000000000201775

8. Lagler FB, Moder A, Rohrbach M, Hennermann J, Mengel E, et al. Extent, impact, and predictors of diagnostic delay in Pompe disease: A combined survey approach to unveil the diagnostic odyssey.

8. 拉格勒 FB，莫德尔 A，罗尔巴赫 M，亨纳曼 J，门格尔 E，等。庞贝病诊断延迟的程度、影响及预测因素：一种综合调查方法揭示诊断之旅。

JIMD Reports.

JIMD报告。

2019 Jul 17;49(1):89-95.

2019年7月17日；49（1）：89-95。

https://doi.org/10.1002/jmd2.12062

https://doi.org/10.1002/jmd2.12062

9. Ullman JC, Dick RA, Linzner D, Minga T, Tep S, Satterfield TF, Xi Y, Beattie DT, Marmon T, Neutel JM, Chung B, Leeds JM, Noonberg SB, Green EM, & Bernstein HS. First‐in‐human evaluation of safety, pharmacokinetics and muscle glycogen lowering of a novel glycogen synthase 1 inhibitor for the treatment of Pompe disease.

9. Ullman JC, Dick RA, Linzner D, Minga T, Tep S, Satterfield TF, Xi Y, Beattie DT, Marmon T, Neutel JM, Chung B, Leeds JM, Noonberg SB, Green EM, & Bernstein HS。首次人体评估一种新型糖原合成酶1抑制剂在治疗庞贝病中的安全性、药代动力学和肌肉糖原降低效果。

.

。

Clinical Pharmacology & Therapeutics

临床药理学与治疗学

. 2024 Oct 22;116(6): 1580–1592.

2024年10月22日；116（6）：1580-1592。

https://doi.org/10.1002/cpt.3470

https://doi.org/10.1002/cpt.3470

10. Corsini A. Improving the treatment of Pompe disease with enzyme replacement therapy: Current strategies and clinical evidence.

10. Corsini A. 改善庞贝病的酶替代疗法：当前策略与临床证据。

Expert Opinion on Pharmacotherapy

药物治疗专家意见

. 2024 Nov 7;26(7): 835–848.

2024年11月7日；第26卷第7期：835-848页。

https://doi.org/10.1080/14656566.2025.2491508

https://doi.org/10.1080/14656566.2025.2491508

11. Ullman JC, Mellem KT, Xi Y, Ramanan V, Merritt H, Choy R, Gujral T, Young LEA, Blake K, Tep S, Homburger JR, O’Regan A, Ganesh S, Wong P, Satterfield TF, Lin B, Situ E, Yu C, Espanol B, et al. Small-molecule inhibition of glycogen synthase 1 for the treatment of Pompe disease and other glycogen storage disorders.

11. Ullman JC, Mellem KT, Xi Y, Ramanan V, Merritt H, Choy R, Gujral T, Young LEA, Blake K, Tep S, Homburger JR, O’Regan A, Ganesh S, Wong P, Satterfield TF, Lin B, Situ E, Yu C, Espanol B, 等。小分子抑制糖原合成酶1用于治疗庞贝病及其他糖原贮积病。

.

。

Science Translational Medicine

科学转化医学

. 2024 Jan 17;16(730).

. 2024年1月17日；16（730）。

https://doi.org/10.1126/scitranslmed.adf1691

https://doi.org/10.1126/scitranslmed.adf1691