BlueRock Therapeutics报告了bemdaneprocel治疗帕金森病的I期试验的36个月积极结果-动脉网

BlueRock Therapeutics reports positive 36-month results from Phase：trial of bemdaneprocel for treating Parkinson’s disease

拜耳等信源发布 2025-10-07 14:09

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October

十月

2025

07:59 AM

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Europe/Amsterdam

欧洲/阿姆斯特丹

BlueRock Therapeutics reports positive 36-month results from Phase I trial of bemdaneprocel for treating Parkinson’s disease

BlueRock Therapeutics报告了bemdaneprocel治疗帕金森病的I期试验积极的36个月结果。

Not intended for UK Media

不适用于英国媒体

Summary

摘要

Investigational cell therapy bemdaneprocel continues to demonstrate a favorable safety profile at 36 months in Phase I trial participants / Secondary clinical efficacy endpoints related to motor outcomes remain stable compared to 24 months and continue to show positive trends from baseline / Data were presented on October 6th at the International Congress of Parkinson’s Disease and Movements Disorders.

研究性细胞疗法bemdaneprocel在I期试验参与者中，36个月时继续显示出良好的安全性 / 与24个月相比，与运动结果相关的次要临床疗效终点保持稳定，并继续显示出从基线开始的积极趋势 / 数据于10月6日在国际帕金森病和运动障碍大会上公布。

Berlin, Germany and Cambridge, MA, USA, October 7, 2025

德国柏林，美国马萨诸塞州剑桥，2025年10月7日

– Bayer AG and its wholly owned, independently operated subsidiary BlueRock Therapeutics LP, a clinical stage cell therapy company, today announced positive 36-month data from exPDite, a Phase I clinical trial of bemdaneprocel, an investigational cell therapy for the treatment of Parkinson's disease (PD).

拜耳公司及其全资独立运营的子公司 BlueRock Therapeutics LP（一家临床阶段的细胞治疗公司）今天宣布了 exPDite 的积极 36 个月数据，exPDite 是一项针对帕金森病 (PD) 治疗的在研细胞疗法 bemdaneprocel 的 I 期临床试验。

The data were presented on October 6th at the International Congress of Parkinson’s Disease and Movements Disorders..

该数据于 10 月 6 日在国际帕金森病和运动障碍大会上公布。

“Bemdaneprocel represents a new approach to restoring the dopamine inputs that are lost in Parkinson’s, and leverages substantial advances in stem cell technology,” said Claire Henchcliffe, MD, chair of the UC Irvine School of Medicine’s Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators.

“Bemdaneprocel 代表了一种恢复帕金森病中失去的多巴胺输入的新方法，并利用了干细胞技术的重大进展，”加州大学欧文分校医学院神经病学系主任、本研究的主要研究者之一 Claire Henchcliffe 医学博士表示。

“The new 3-year data is a critical next step to evaluate longer term safety. While there is a need for caution in interpreting the positive trends in clinical outcomes, initial signals are there, particularly in the higher dose cohort and the upcoming exPDite-2 clinical trial should shed further light on potential benefits.”.

“新的三年数据是评估长期安全性的关键下一步。尽管在解读临床结果的积极趋势时需要谨慎，但初步信号已经显现，尤其是在高剂量组中，即将进行的exPDite-2临床试验应该会进一步揭示潜在益处。”

Bemdaneprocel’s safety profile at 36 months is consistent with earlier findings, demonstrating that it continues to be well tolerated by patients, with no adverse events reported related to the therapy or surgical procedure. F-Dopa imaging suggests that transplanted cells continue to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months as outlined in the study’s protocol.

Bemdaneprocel在36个月的安全性表现与早期研究结果一致，表明它仍然被患者良好耐受，且未报告与治疗或手术相关的不良事件。F-Dopa影像显示，根据研究方案，在12个月停止免疫抑制治疗后，移植的细胞仍在大脑中继续存活并成功植入。

In addition, secondary clinical endpoints related to motor symptoms continue to show positive trends from baseline through the duration of follow-up, with more encouraging trends in the high dose cohort than those in the low dose cohort. These were assessed by the MDS-Unified Parkinson’s Disease Rating Scale Part II and III (MDS-UPDRS Part II & III) and the Parkinson’s disease Diary, tools used to assess Parkinson’s disease severity in motor symptoms..

此外，与运动症状相关的次要临床终点在整个随访期间持续显示出从基线开始的积极趋势，高剂量组的趋势比低剂量组更令人鼓舞。这些指标通过MDS-帕金森病综合评定量表第二部分和第三部分（MDS-UPDRS Part II & III）以及帕金森病日记进行评估，这些工具用于评估帕金森病运动症状的严重程度。

“The 36-month data from this Phase I study underscores our hope that bemdaneprocel could be a potentially meaningful and durable therapeutic option for treating people living with Parkinson’s disease,” said Gabi Belfort, MD, PhD, Senior Vice President and Bemdaneprocel Product Lead at BlueRock Therapeutics.

“这项 I 期研究的 36 个月数据让我们更加希望，bemdaneprocel 可能成为治疗帕金森病患者的一种有意义且持久的治疗选择，” BlueRock Therapeutics 高级副总裁兼 Bemdaneprocel 产品主管 Gabi Belfort 医学博士表示。

“We are excited to share these data with the Parkinson’s disease community and are committed to advancing bemdaneprocel through the next stage of clinical testing.”.

“我们很高兴与帕金森病社区分享这些数据，并致力于将bemdaneprocel推进到下一阶段的临床测试。”

In the high dose cohort (n=7), the 36-month measurement of the effects of bemdaneprocel on motor symptoms using the MDS-UPDRS Part III measured in the “OFF”-medication state, showed a mean reduction of 17.9 points compared with baseline. The low dose cohort (N=4) showed a mean decrease of 13.5 points.

在高剂量组（n=7）中，使用MDS-UPDRS第三部分在“无药物”状态下测量的36个月结果显示，bemdaneprocel对运动症状的影响与基线相比平均减少了17.9分。低剂量组（N=4）则表现出平均减少13.5分。

Both are considered to be clinically meaningful..

两者都被认为具有临床意义。

Using the PD Diary, which categorizes patients as being in the “ON” state when their symptoms are well controlled and in the “OFF” state when they experience a worsening of their symptoms, participants in the high dose cohort showed a mean increase of 1 hour in time spent in the “Good ON” state (without troublesome dyskinesias) compared with baseline after 36 months.

使用PD日记（将症状得到良好控制的患者归类为“ON”状态，而症状恶化的患者归类为“OFF”状态），高剂量组的参与者在36个月后，“良好ON”状态（无明显运动障碍）的持续时间相比基线平均增加了1小时。

Two participants in the high dose cohort did not submit a 36-month PD Diary. Time spent in the “OFF” state showed a mean decrease of - 0.93 hours from baseline after 36 months. The participants in the low dose cohort showed a mean increase of 0.23 hours in the “Good ON” state time compared with baseline and a corresponding mean decrease of –1.15 hours in “OFF” state time..

高剂量组的两名参与者未提交 36 个月的 PD 日记。在 36 个月后，“关闭”状态的时间平均减少了 -0.93 小时。低剂量组的参与者相较基线，“良好开启”状态时间平均增加了 0.23 小时，而“关闭”状态时间相应平均减少了 -1.15 小时。

In the high dose cohort, the 36-month measurement of the effects of bemdaneprocel on activities of daily living using the MDS-UPDRS Part II measured a mean reduction of -4.3 points compared with baseline. The low dose cohort showed a mean increase of 0.2 points. Participants in both cohorts continue in the long term Continued Evaluation Study..

在高剂量组中，使用MDS-UPDRS第二部分测量bemdaneprocel对日常活动的影响，36个月时与基线相比平均减少了4.3分。低剂量组显示平均增加了0.2分。两个组的参与者都继续参与长期的持续评估研究。

“The results for bemdaneprocel after 36 months mark another motivating milestone,” said Christian Rommel, Global Head of Research and Development of the Pharmaceuticals Division at Bayer. “They reinforce our commitment to developing urgently needed new treatment options for Parkinson's patients.”

“bemdaneprocel 在 36 个月后的结果标志着另一个鼓舞人心的里程碑，”拜耳制药部门全球研发主管克里斯蒂安·罗梅尔表示。“它们进一步坚定了我们为帕金森病患者开发迫切需要的新治疗方案的承诺。”

About bemdaneprocel (BRT-DA01)

关于bemdaneprocel（BRT-DA01）

Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine producing neurons that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells that continue developing into mature dopamine neurons after implantation.

Bemdaneprocel（BRT-DA01）是一种研究性细胞疗法，旨在替代帕金森病中丧失的多巴胺生成神经元。这些多巴胺能神经元前体来源于人类胚胎多能干细胞，在植入后会继续发育为成熟的多巴胺神经元。

In a surgical procedure, these neuron precursors are implanted into the brain of a person with Parkinson’s disease. When transplanted, they have the potential to re-form neural networks that have been severely affected by Parkinson’s disease and to potentially restore motor and non-motor function to patients.

在手术过程中，这些神经前体细胞会被植入帕金森病患者的大脑中。移植后，它们有可能重新形成受到帕金森病严重影响的神经网络，并有可能恢复患者的运动和非运动功能。

In 2021 bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. A pivotal Phase III trial to assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control is currently enrolling participants.

2021年，bemdaneprocel获得快速通道资格，2024年获得FDA的再生医学高级疗法（RMAT）资格。目前，一项关键的III期试验正在进行参与者招募，以评估bemdaneprocel相较于假手术对照的有效性、安全性和整体影响。

Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority..

贝达内普罗塞尔尚未获得任何卫生机构批准用于治疗任何疾病或医疗状况。

About Parkinson’s disease

关于帕金森病

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）是一种进展性的神经退行性疾病，对患者的日常生活有显著影响。在帕金森病中，大脑内产生多巴胺的神经细胞死亡，导致运动功能的持续丧失。症状包括震颤、肌肉僵硬和运动迟缓。

Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression. Symptoms typically intensify over time and make everyday tasks demanding. The prevalence of PD has doubled over the past 25 years. Today, more than 10 million people worldwide are estimated to be living with PD.

此外，帕金森病患者还会经历非运动症状，包括疲劳、精力不足、认知问题和抑郁。症状通常会随着时间的推移而加重，使日常任务变得艰巨。在过去的25年中，帕金森病的患病率翻了一番。今天，全球估计有超过1000万人患有帕金森病。

This makes it the world’s second most prevalent neurodegenerative disease. It is also the most frequent movement disorder. At present there is no cure, and current treatment options are inadequate and lack holistic management of symptoms so there is an urgent need for new therapies..

这使得它成为世界上第二普遍的神经退行性疾病，也是最常见的运动障碍。目前尚无治愈方法，现有的治疗选择不充分，缺乏对症状的整体管理，因此迫切需要新的治疗方法。

About BlueRock Therapeutics LP

关于BlueRock Therapeutics LP

BlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological and ophthalmic diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment of Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor diseases are clinical stage programs.

BlueRock Therapeutics LP是一家处于临床阶段的细胞治疗公司，专注于创造细胞药物以治疗毁灭性疾病。我们正在利用细胞疗法的力量，为遭受神经和眼科疾病困扰的患者开发一系列新药。我们有两种新型的研究性细胞疗法，分别是用于治疗帕金森病的bemdaneprocel（BRT-DA01）和用于治疗原发性光感受器疾病的OpCT-001，目前都已进入临床阶段。

Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the US FDA (Food and Drug Administration) and is being tested in a Phase III clinical trial, exPDite-2. OpCT-001 has Fast Track designation from the FDA and is being tested in a Phase I clinical trial, Clarico.

贝达内普罗赛尔已获得美国食品药品监督管理局（FDA）的RMAT（再生医学高级疗法）和快速通道资格，并正在III期临床试验exPDite-2中进行测试。OpCT-001已获得FDA的快速通道资格，并正在I期临床试验Clarico中进行测试。

BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves.

BlueRock 成立于2016年，是Versant Ventures和拜耳旗下影响力投资部门Leaps的合资企业，该部门致力于投资具有范式转变意义的突破性创新。2019年底，BlueRock成为拜耳集团的全资独立运营子公司。我们的文化以无畏挑战、坚持到底的勇气为基石，以变革医学、传递希望的紧迫感为导向，以使命指引的诚信为核心，并秉持社区意识，深知我们每个人都是比自身更宏大事业的一部分。

For more information, visit .

欲了解更多信息，请访问。

www.bluerocktx.com

。

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.

拜耳是一家在医疗保健和营养等生命科学领域具有核心竞争力的全球性企业。秉承“人人健康，无饥饿”的使命，公司通过支持应对不断增长和老龄化的全球人口所带来的重大挑战，设计其产品和服务以帮助人类和地球繁荣发展。

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 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros.

拜耳致力于通过其业务推动可持续发展并产生积极影响。同时，集团旨在通过创新和增长提高盈利能力并创造价值。拜耳品牌在全球范围内代表信任、可靠性和质量。2024财年，该集团拥有约93,000名员工，销售额达466亿欧元。

R&D expenses amounted to 6.2 billion euros. For more information, go to .

研发费用达62亿欧元。欲了解更多信息，请访问。

www.bayer.com

。

Find more information at

更多信息请访问

https://pharma.bayer.com

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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.

本发布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结果、财务状况、发展或业绩与这里提供的估计存在重大差异。

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

这些因素包括拜耳公开报告中讨论的因素，这些报告可在拜耳网站上查阅。

www.bayer.com

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

公司不承担更新这些前瞻性声明或使其符合的任何责任 whatsoever

them to future events or developments.

将它们应用于未来的事件或发展。

Hauser RA, Gordon MF, Mizuno Y, Poewe W, Barone P, Schapira AH, Rascol O, Debieuvre C, Fräßdorf M. Minimal clinically important difference in Parkinson's disease as assessed in pivotal trials of pramipexole extended release. Parkinsons Dis. 2014;2014:467131. doi: 10.1155/2014/467131. Epub 2014 Apr 1.

豪瑟 RA，戈登 MF，水野 Y，波埃 W，巴罗内 P，沙皮拉 AH，拉斯科尔 O，德比欧尔 C，弗拉索夫 M。关键性普拉米普利缓释片试验中评估的帕金森病最小临床重要差异。《帕金森病》。2014；2014：467131。doi：10.1155/2014/467131。电子版发布于2014年4月1日。

PMID: 24800101; PMCID: PMC3995302..

PMID: 24800101; PMCID: PMC3995302。

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Silke Lengemann, phone +49 173 8990997

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