基因治疗公司AskBio帕金森病基因疗法二期临床试验的首批随机参与者-动脉网

First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease

拜耳等信源发布 2025-01-14 15:39

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January

一月

2025

08:29 AM

上午08:29

Europe/Amsterdam

欧洲/阿姆斯特丹

First participants randomized in AskBio Phase II gene therapy trial for Parkinson’s disease

AskBio II期帕金森病基因治疗试验的首批参与者随机分组

Not intended for UK Media

不适用于英国媒体

Summary

摘要

Enrollment ongoing in the United States for REGENERATE-PD clinical trial of AB-1005 (also known as AAV2-GDNF) glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of moderate-stage Parkinson’s disease / Phase Ib 36-month data demonstrated a favorable safety profile and continued positive trends in assessed clinical measures of AB-1005 with no associated serious adverse events.

正在美国进行的AB-1005（也称为AAV2-GDNF）神经胶质细胞系衍生的神经营养因子（GDNF）研究性基因疗法治疗中度帕金森病/Ib期的REGENATE-PD临床试验36个月的数据显示，在评估的AB-1005临床措施中，有良好的安全性和持续的积极趋势，没有相关的严重不良事件。

Berlin, Germany, and Research Triangle Park, N.C., USA, January 14, 2025

2025年1月14日，德国柏林和美国北卡罗来纳州三角研究公园

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AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD).

作为拜耳公司的全资子公司独立运营的基因治疗公司AskBio Inc.（AskBio）今天宣布，首批参与者已被随机分配到帕金森病（PD）患者的II期临床试验中。

“The randomization of the first participants in REGENERATE-PD is positive news for people living with Parkinson’s disease and the physicians treating them,” said Dr Rajesh Pahwa, MD, Director, Parkinson's Disease and Movement Disorder Center, University of Kansas Medical Center, USA, and REGENERATE-PD Principal Investigator.

美国堪萨斯大学医学中心帕金森病和运动障碍中心主任、医学博士Rajesh Pahwa博士和REGENATE-PD首席研究员说：“REGENATE-PD首批参与者的随机化对帕金森病患者和治疗他们的医生来说是一个积极的消息。”。

“There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial gives hope to patients and the medical community.”.

“帕金森氏症非常需要神经修复疗法，并且在II期临床试验中看到重要的研究性基因疗法的进步为患者和医学界带来了希望。”。

In 2024, AskBio initiated recruitment in the United States arm of REGENERATE-PD. The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD. REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom..

2024年，AskBio开始在美国招募REGENATE-PD。这项随机，双盲，II期临床试验的目的是评估AB-1005在45-75岁中度PD成年参与者中递送至壳核的安全性和有效性。REGENATE-PD估计在美国，德国，波兰和英国开设的临床中心招募了大约87名参与者。。

AskBio also presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders, which was held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024. The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events.

AskBio还于2024年9月27日至10月1日在宾夕法尼亚州费城举行的国际帕金森病和运动障碍大会上提交了为期36个月的Ib期数据。数据表明，AB-1005的给药耐受性良好，无严重不良事件。

The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and motor diaries, and trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).

中度PD队列在36个月时显示出几种运动量表的改善或稳定性趋势，包括运动障碍协会统一帕金森病评定量表（MDS-UPDRS）和运动日记，以及帕金森病药物减少的趋势（左旋多巴等效日剂量[LEDD]）。

Most participants in the Mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD..

轻度PD队列的大多数参与者表现出总体稳定的临床状态，MDS-UPDRS，自我报告的PD运动日记或LEDD几乎没有变化。。

“AskBio continues to mark significant milestones in the clinical development of the investigational gene therapy AB-1005 as we strive to bring a safe and effective gene therapy to patients with moderate stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio.

“随着我们努力为中度帕金森病患者带来安全有效的基因治疗，AskBio继续标志着研究性基因治疗AB-1005临床开发的重要里程碑，”AskBio首席开发官兼首席医疗官Canwen Jiang博士说。

“With REGENERATE-PD participants now being randomized, we are excited by our progress with AB-1005 and look forward to sharing further updates at an appropriate scientific forum as the program advances through next year and beyond.”.

“随着REGENATE-PD参与者现在被随机分配，我们对AB-1005的进展感到兴奋，并期待着随着该计划明年及以后的进展，在适当的科学论坛上分享进一步的更新。”。

“The advancement of AskBio’s Parkinson’s program is a significant milestone that highlights our commitment to exploring the potential of gene therapy in an area of significant unmet medical need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development.

拜耳制药部执行委员会成员、全球研发主管克里斯蒂安·隆美尔（Christian Rommel）表示：“AskBio帕金森氏症项目的推进是一个重要的里程碑，突显了我们致力于探索基因治疗在重大未满足医疗需求领域的潜力。”。

“We believe that therapeutic modalities like gene therapy can change the treatment landscape for people living with the debilitating effects of Parkinson’s disease and look forward to seeing what we will learn in this next phase of research and development for AB-1005.”.

“我们相信，基因治疗等治疗方式可以改变帕金森病患者的治疗前景，并期待在AB-1005的下一阶段研究和开发中看到我们将学到什么。”。

AskBio is also exploring AB-1005 beyond Parkinson’s disease and is currently enrolling participants in the United States with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing condition..

AskBio还在探索超越帕金森病的AB-1005，目前正在美国招募帕金森病多系统萎缩亚型（MSA-P）的参与者进行I期临床试验，以评估这种快速发展的疾病的初步安全性，耐受性和有效性。。

AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

。

About Parkinson’s disease

关于帕金森氏病

Parkinson’s disease (PD) is a progressive neurodegenerative disease. It has a significant impact on a person’s daily life. In PD the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function. Symptoms include tremors, muscle rigidity, and slowness of movement.

帕金森病（PD）是一种进行性神经退行性疾病。它对一个人的日常生活有重大影响。在PD中，大脑中产生多巴胺的神经细胞的死亡导致运动功能的持续丧失。症状包括震颤，肌肉僵硬和运动缓慢。

Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, congestive issues, and depression. Symptoms typically intensify over time and make everyday tasks increasingly demanding. The prevalence of PD has doubled over the past 25 years..

此外，帕金森病患者会出现非运动症状，包括疲劳和缺乏能量，充血问题和抑郁症。症状通常会随着时间的推移而加剧，并使日常任务变得越来越苛刻。在过去的25年中，帕金森病的患病率翻了一番。。

Today, more than 10 million people worldwide are estimated to be living with PD. This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder.

今天，全世界估计有1000多万人患有帕金森病。这使其成为世界上第二大最普遍的神经退行性疾病。它也是最常见的运动障碍。

At present there is no cure, and current treatment options lack the holistic management of symptoms, so new therapies are needed.

目前尚无治愈方法，目前的治疗方案缺乏对症状的整体管理，因此需要新的治疗方法。

About REGENERATE-PD

关于REGENATE-PD

REGENERATE-PD is a Phase II, randomized, double-blind, sham-controlled trial of the efficacy and safety of intraputaminal AB-1005 in the treatment of adults (45-75 years) with moderate-stage Parkinson's disease. The trial will include an estimated 87 subjects with trial sites located in the United States, Germany, Poland, and the United Kingdom.

REGENATE-PD是一项II期，随机，双盲，假对照试验，研究腹腔注射AB-1005治疗中度帕金森病成人（45-75岁）的疗效和安全性。该试验将包括大约87名受试者，试验地点位于美国，德国，波兰和英国。

For more information about the REGENERATE-PD clinical trial, visit .

有关REGENATE-PD临床试验的更多信息，请访问。

clinicaltrials.gov

clinicaltrials.gov 网站

(NCT06285643), or visit

（NCT06285643），或访问

askbio.com

网站：askbio.com

About AB-1005

关于AB-1005

AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery..

AB-1005是一种基于腺相关病毒载体血清型2（AAV2）的研究性基因疗法，该载体含有人神经胶质细胞系衍生的神经营养因子（GDNF）转基因，可在直接神经外科注射MRI监测的对流增强递送后，在脑局部区域稳定持续表达GDNF。。

In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons. Recombinant GDNF has long been evaluated as a potential treatment for diseases, such as Parkinson’s disease (PD), marked by progressive degeneration of midbrain dopaminergic neurons..

在非临床研究中，GDNF已被证明可促进多巴胺能神经元的存活和形态分化。长期以来，重组GDNF一直被评估为帕金森病（PD）等疾病的潜在治疗方法，其特征是中脑多巴胺能神经元进行性变性。。

Through a combination of an investigational gene therapy and innovative neurosurgical delivery approach, we can now test the GDNF hypothesis in PD by getting this neurotrophic factor to these degenerating nigrostriatal neurons in a potentially more clinically relevant fashion.

通过研究性基因疗法和创新的神经外科递送方法的结合，我们现在可以通过以潜在的更具临床相关性的方式将这种神经营养因子引入这些退化的黑质纹状体神经元来测试PD中的GDNF假说。

About AskBio

关于AskBio

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.

AskBio Inc.是拜耳公司的全资和独立运营子公司，是一家全面整合的基因治疗公司，致力于开发挽救生命的药物和改变生命。该公司拥有一系列神经肌肉、中枢神经系统、心血管和代谢疾病适应症的临床项目组合，临床阶段包括充血性心力衰竭、肢带型肌营养不良、多系统萎缩、帕金森氏病和庞贝氏病的研究治疗。

AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing.

AskBio的基因治疗平台包括Pro10™，这是一种业界领先的专有细胞系制造工艺，以及大量衣壳和启动子。该公司的全球总部位于北卡罗莱纳州三角研究公园，欧洲总部位于苏格兰爱丁堡，已经产生了数百种专有衣壳和启动子，其中一些已经进入临床前和临床测试。

An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at .

该公司是基因治疗领域的早期创新者，在五个国家拥有900多名员工，在AAV生产和嵌合衣壳等领域拥有600多项专利和专利申请。更多信息，请访问。

www.askbio.com

网站：www.askbio.com

or follow us on LinkedIn.

或者在LinkedIn上关注我们。

About Bayer

拜耳简介

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.

拜耳是一家全球性企业，在医疗保健和营养等生命科学领域拥有核心竞争力。根据其“人人健康，无人饥饿”的使命，该公司的产品和服务旨在通过支持应对全球人口不断增长和老龄化带来的重大挑战，帮助人们和地球繁荣发展。

Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros.

拜耳致力于推动可持续发展，并对其业务产生积极影响。。拜耳品牌代表着全世界的信任、可靠性和质量。2023财年，该集团雇佣了约10万人，销售额476亿欧元。

R&D expenses before special items amounted to 5.8 billion euros. For more information, go to .

不含特殊项目的研发费用为58亿欧元。有关更多信息，请转至。

www.bayer.com

Forward-Looking Statements

前瞻性声明

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here.

本版本可能包含基于拜耳管理层当前假设和预测的前瞻性声明。各种已知和未知的风险、不确定性和其他因素可能导致公司的实际未来业绩、财务状况、发展或业绩与此处给出的估计之间存在重大差异。

These factors include those discussed in Bayer’s public reports which are available on the Bayer website at .

。

www.bayer.com

. The company assumes no liability whatsoever to update these forward-looking statements or to conform

。公司不承担任何责任更新这些前瞻性声明或遵守

them to future events or developments.

他们对未来的事件或发展。

AskBio Forward-Looking Statements

AskBio前瞻性声明

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性声明”。本新闻稿中任何不属于历史事实声明的声明都可能被视为前瞻性声明。诸如“相信”、“预期”、“计划”、“期望”、“将”、“打算”、“潜在”、“可能”等词语以及类似的表达都旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

这些前瞻性声明包括但不限于有关AskBio临床试验的声明。这些前瞻性陈述涉及风险和不确定性，其中许多超出了AskBio的控制范围。已知的风险包括：AskBio可能无法执行其业务计划和目标，包括满足其预期或计划的临床和监管里程碑和时间表，依赖第三方，临床开发计划，制造流程和计划，以及将其候选产品推向市场，原因多种多样，包括公司财务和其他资源的可能限制，可能无法及时预期或解决的制造限制，与我们的第三方合作伙伴和合作伙伴的潜在分歧或其他问题，以及监管，法院或机构的反馈或决定，如美国食品和药物管理局或美国专利和商标局的反馈和决定。

Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof..

上述任何风险都可能对AskBio的业务和经营成果产生重大不利影响。您不应过度依赖本新闻稿中的前瞻性声明。AskBio没有义务根据本协议日期后的事件或情况公开更新其前瞻性声明。。

Bayer Global Media Contact:

拜耳全球媒体联系人：

Dr. Imke Meyer, phone +49 (214) 60001275

Imke Meyer博士，电话 +49 (214) 60001275

Email:

电子邮件：

imke.meyer@bayer.com

AskBio Media Contact:

AskBio媒体联系人：

Phil McNamara, phone +1 (984) 5207211

菲尔·麦克纳马拉，电话+1（984）5207211