OSAKA, Japan & SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter “Shionogi”) and Maze Therapeutics, Inc. (Head Office: South San Francisco, California, USA; Chief Executive Officer: Jason Coloma, Ph.D.; hereafter “Maze”) announced the companies have completed an exclusive worldwide license agreement for the rights to MZE001, an investigational oral glycogen synthase 1 (GYS1) inhibitor that aims to address Pompe disease by limiting disease-causing glycogen buildup..

日本大阪和加利福尼亚州南旧金山——（商业新闻短讯）——Shionogi＆Co.，Ltd.（总部：日本大阪；首席执行官：Isao Teshirogi，Ph.D.；以下简称“Shionogi”）和Maze Therapeutics，Inc.（总部：美国加利福尼亚州南旧金山；首席执行官：Jason Coloma，Ph.D.；以下简称“Maze”）宣布，两家公司已就MZE001的权利达成全球独家许可协议，MZE001是一种研究性口服糖原合酶1（GYS1）抑制剂，旨在通过限制致病糖原的积累来解决庞贝病。。

Pompe disease is a rare, inherited disorder caused by mutations in the gene coding for acid alpha-glucosidase (GAA), which can lead to the buildup of glycogen in skeletal muscle, respiratory muscle and cardiac muscle tissues resulting in progressive weakness and respiratory compromise.

庞贝氏病是一种罕见的遗传性疾病，由编码酸性α-葡萄糖苷酶（GAA）的基因突变引起，可导致骨骼肌，呼吸肌和心肌组织中糖原的积累，从而导致进行性无力和呼吸衰竭。

Under the terms of the agreement, Shionogi has acquired exclusive worldwide rights for MZE001 as well as related programs and intellectual property. Shionogi will pay an upfront fee of $150 million, and Maze will be eligible for milestone payments based on development, regulatory and commercial achievements plus tiered royalties based upon future net sales.

根据协议条款，Shionogi已获得MZE001的全球独家权利以及相关程序和知识产权。Shionogi将支付1.5亿美元的预付费，Maze将有资格根据开发、监管和商业成就获得里程碑式的付款，并根据未来的净销售额支付分层版税。

The required 30-day waiting period outlined in United States Hart-Scott-Rodino (HSR) Act has expired, and the transaction is completed..

美国哈特·斯科特·罗迪诺（HSR）法案规定的30天等待期已经到期，交易已经完成。。

“This agreement is a strong strategic fit for Shionogi. It will help meaningfully advance our commitment to develop innovative medicines that address unmet medical needs and complement Shionogi's rapidly expanding pipeline in the focus areas designated in our Medium-Term Business Plan STS2030 Revision,” said Isao Teshirogi, Ph.D., CEO of Shionogi.

Shionogi首席执行官IsaoTeshirogi博士说：“这项协议非常适合Shionogi。它将有助于有意义地推进我们开发创新药物的承诺，以满足未满足的医疗需求，并补充Shionogi在中期商业计划STS2030修订版中指定的重点领域迅速扩大的渠道。”。

“The science behind MZE001 is differentiated and promising, and we look forward to developing the compound as both mono and add-on therapy to enzyme replacement therapies.”.

“MZE001背后的科学是有区别的和有前景的，我们期待着开发该化合物作为酶替代疗法的单一疗法和附加疗法。”。

MZE001 is a small molecule and specific inhibitor of GYS1, an enzyme involved in glycogen synthesis. It reduces the glycogen concentration in muscles by inhibiting this enzyme, and the results from the Phase 1 study of MZE001 suggest that it has the potential to be the first oral therapy for the treatment of Pompe disease.

MZE001是GYS1（一种参与糖原合成的酶）的小分子特异性抑制剂。它通过抑制这种酶来降低肌肉中的糖原浓度，MZE001的第一阶段研究结果表明，它有可能成为治疗庞贝病的第一种口服疗法。

MZE001 has the potential to be used both as a monotherapy option and as an add-on therapy with enzyme replacement, the current standard of care, to enhance the treatment of patients with Pompe disease..

MZE001有可能被用作单一疗法的选择，也可以作为酶替代疗法的附加疗法，酶替代疗法是目前的护理标准，可以增强庞贝病患者的治疗效果。。

“Shionogi is committed to advancing and commercializing MZE001 because they understand the potential this therapy has for patients and the unmet medical needs it could address,” said Jason Coloma, Ph.D., CEO of Maze. “Shionogi has a track record of developing and delivering innovative medicines to patients worldwide, and we’re confident they are the right partner to continue to advance MZE001 through clinical trials so that it may reach patients with this life-threatening condition as soon as possible.”.

Maze首席执行官Jason Coloma博士说：“Shionogi致力于推进MZE001并将其商业化，因为他们了解这种疗法对患者的潜力以及它可以解决的未满足的医疗需求。”。“Shionogi在为全球患者开发和提供创新药物方面有着良好的记录，我们相信他们是正确的合作伙伴，可以通过临床试验继续推进MZE001，以便尽快将其送达这种危及生命的患者。”。

In 2022, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to MZE001. The FDA grants Orphan Drug designation to prevent, diagnose or treat a rare disease or condition. Under the FDA's Orphan Drug Act, orphan drug status provides incentives, including tax credits, grants and waiver of certain administrative fees for clinical trials, and seven years of market exclusivity following drug approval..

2022年，美国食品和药物管理局（FDA）授予MZE001孤儿药称号。FDA授予孤儿药名称，用于预防、诊断或治疗罕见疾病或病症。根据美国食品和药物管理局（FDA）的《孤儿药法案》，孤儿药地位提供了激励措施，包括税收抵免、临床试验某些行政费用的赠款和豁免，以及药物批准后七年的市场排他性。。

About Pompe Disease

关于庞贝病

Pompe Disease is a rare congenital metabolic disorder in which there is a deficiency of the enzyme necessary for the breakdown of glycogen within cells, resulting in the accumulation of glycogen in cells throughout the body, especially in muscle cells. This leads to various symptoms such as muscle weakness and delayed growth.

庞贝氏症是一种罕见的先天性代谢紊乱，其中缺乏细胞内糖原分解所必需的酶，导致糖原在整个身体的细胞中积累，尤其是在肌肉细胞中。这会导致各种症状，例如肌肉无力和生长迟缓。

In Japan, it has been designated as a 'specified rare disease' and a 'specific pediatric disease,' making it eligible for medical expense assistance programs and other forms of support..

在日本，它被指定为“特定罕见疾病”和“特定儿科疾病”，因此有资格获得医疗费用援助计划和其他形式的支持。。

About Maze

关于Maze

Maze Therapeutics is a biopharmaceutical company that is harnessing the power of human genetics to transform the lives of patients, with a focus on genetically informed therapies for common diseases such as chronic kidney disease. Maze applies variant functionalization in tandem with advanced data science methods and a robust suite of research and development capabilities to advance a pipeline of novel precision medicines.

Maze Therapeutics是一家生物制药公司，它利用人类遗传学的力量来改变患者的生活，重点是针对慢性肾病等常见疾病的遗传信息疗法。Maze将变体功能化与先进的数据科学方法以及强大的研究和开发能力相结合，以推进新型精密药物的生产线。

Maze has developed the Maze Compass Platform TM, a proprietary, purpose-built platform to understand and integrate the critical step of variant functionalization into each stage of drug development. Utilizing the Maze Compass PlatformTM, Maze is building a broad portfolio of wholly owned and partnered programs.

Maze开发了Maze Compass Platform TM，这是一个专有的专门构建的平台，用于理解变体功能化的关键步骤并将其整合到药物开发的每个阶段。利用Maze Compass PlatformTM，Maze正在建立一个由全资和合作项目组成的广泛投资组合。

Maze is based in South San Francisco. For more information, please visit mazetx.com, or follow us on LinkedIn and Twitter..

梅兹总部位于旧金山南部。有关更多信息，请访问mazetx.com，或在LinkedIn和Twitter上关注我们。。

About Shionogi

关于Shionogi

Shionogi & Co., Ltd. is a leading global research-driven pharmaceutical company dedicated to bringing benefits to patients based on its corporate philosophy of 'supplying the best possible medicine to protect the health and well-being of the patients we serve.' Shionogi has discovered and developed novel antibiotics, medicines for HIV and influenza and currently markets medicines for infectious diseases and central nervous system disorders.

Shionogi&Co.，Ltd.是一家领先的全球研究驱动型制药公司，致力于根据其“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”的企业理念为患者带来利益Shionogi发现并开发了新型抗生素、艾滋病毒和流感药物，目前正在销售治疗传染病和中枢神经系统疾病的药物。

Shionogi’s global pipeline includes research programs in infectious disease, pain/CNS, metabolic disorders, rare disease, oncology and stroke.

Shionogi的全球管道包括传染病，疼痛/中枢神经系统，代谢紊乱，罕见病，肿瘤学和中风的研究项目。