– BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned, independently operated subsidiary of Bayer AG, today announced that the first patient has received randomized treatment in the pivotal Phase III clinical trial, exPDite-2, of bemdaneprocel, an investigational cell therapy for Parkinson’s disease..

– BlueRock Therapeutics LP是一家临床阶段的细胞治疗公司，也是拜耳股份公司全资拥有并独立运营的子公司，今天宣布首位患者已在关键的III期临床试验exPDite-2中接受了随机治疗，该试验涉及的是一种用于帕金森病的研究性细胞疗法bemdaneprocel。

“People living with Parkinson’s disease urgently need new therapies that truly alter the course of the disease,” said Amit Rakhit, MD, MBA, BlueRock’s Chief Development and Medical Officer. “The initiation of the exPDite-2 trial represents a major step forward toward advancing bemdaneprocel and we are excited to build on the momentum of our earlier data to further develop it as a potentially transformative cell therapy aimed at restoring motor and non-motor function.”.

“帕金森病患者迫切需要能够真正改变疾病进程的新疗法，”BlueRock首席开发和医学官Amit Rakhit博士（医学博士、工商管理硕士）表示。“exPDite-2试验的启动标志着我们在推进bemdaneprocel方面迈出了重要一步，我们非常高兴能够在早期数据的基础上继续努力，进一步将其开发为一种可能具有变革性的细胞疗法，旨在恢复运动和非运动功能。”

exPDite-2 is the first Phase III pivotal clinical trial for an investigational allogeneic pluripotent stem cell derived therapy to treat Parkinson’s disease. In a Phase I study with 12 participants, bemdaneprocel was well tolerated, with no serious adverse events related to drug product at 24 months post-surgery.

exPDite-2 是首个针对研究性同种异体多能干细胞衍生疗法治疗帕金森病的 III 期关键临床试验。在一项包含 12 名参与者的 I 期研究中，bemdaneprocel 表现良好，术后 24 个月未出现与药物相关的严重不良事件。

In addition, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. Building on these results, exPDite-2 is a multicenter, double-blind trial that will assess the efficacy, safety and overall impact of bemdaneprocel compared to sham surgery control.

此外，在手术后 24 个月与运动障碍相关的次要终点观察到了令人鼓舞的趋势。基于这些结果，exPDite-2 是一项多中心、双盲试验，将评估 bemdaneprocel 与假手术对照相比的疗效、安全性和总体影响。

The trial is designed to enroll approximately 102 participants with Parkinson’s disease. The primary endpoint of the study is change from baseline to week 78 in PD diary measure of ON-time without troublesome dyskinesia, adjusted for a 16-hour waking day. In addition, the trial will incorporate secondary endpoints designed to assess objective measures of movement, non-motor symptoms, safety and tolerability, and instruments that capture activities of daily living and quality of life..

该试验旨在招募大约102名帕金森病患者。研究的主要终点是从基线到第78周，以16小时清醒时间为调整标准，评估无困扰性异动症的“ON-time”（症状良好控制时间）在帕金森病日记中的变化。此外，试验还将纳入多个次要终点，旨在评估运动功能的客观指标、非运动症状、安全性与耐受性，以及反映日常生活活动能力和生活质量的相关工具。

“The initiation of the expDite-2 Phase III trial marks a significant milestone in our commitment to transform the treatment landscape for Parkinson’s disease through innovative therapies,” said Christian Rommel Ph.D., Head of Research and Development at Bayer’s Pharmaceuticals Division. “Bemdaneprocel aims to sustainably restore lost physiologic function in the dopaminergic system impacted by the disease, ultimately to enhance the quality of life for patients.”.

“启动expDite-2 III期试验标志着我们通过创新疗法改变帕金森病治疗领域的承诺迈出了重要的里程碑，”拜耳制药部门研发主管克里斯蒂安·罗梅尔博士表示。“Bemdaneprocel的目标是可持续地恢复受该疾病影响的多巴胺能系统的生理功能丧失，最终提高患者的生活质量。”

Depending upon the outcome, the results from this trial are intended to be part of a data package to support regulatory submissions for marketing authorization.

根据结果，本次试验的数据将作为支持监管提交以获得上市许可的数据包的一部分。

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。这些多巴胺能神经前体细胞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，它们有可能重建受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. Data from the Phase I trial’s 12 participants presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders (MDS) demonstrated good tolerability, with no serious adverse events related to drug product at 24 months post-surgery.

2021年，bemdaneprocel获得快速通道资格，2024年获得美国食品药品监督管理局（FDA）的再生医学高级疗法（RMAT）资格。在2024年国际帕金森病与运动障碍大会（MDS）上展示的I期试验中12名参与者的数据显示，该药物在手术后24个月内表现出良好的耐受性，且无与药物相关的严重不良事件。

Further, encouraging trends were observed in secondary endpoints related to motor impairments at 24 months post-surgery. These participants continue in the long term Continued Evaluation Study. Bemdaneprocel has not been approved for treatment of any disease or medical condition by any health authority..

此外，在手术后24个月与运动障碍相关的次要终点观察到了令人鼓舞的趋势。这些参与者继续参与长期的持续评估研究。Bemdaneprocel尚未被任何卫生部门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、肌肉僵硬以及运动迟缓。

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加剧，使日常任务变得困难。在过去的25年中，帕金森病的患病率翻了一番。如今，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 the holistic management of symptoms so there is an urgent need for new therapies..

这使得它成为世界上第二常见的神经退行性疾病，也是最常见的运动障碍疾病。目前尚无治愈方法，现有的治疗手段不足且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，专注于开发治疗严重疾病的细胞药物。我们正在利用细胞治疗的力量，为患有神经和眼科疾病的人群开发一系列新药。我们的两种新型研究性细胞疗法，用于治疗帕金森病的bemdaneprocel（BRT-DA01）和用于治疗原发性感光细胞疾病的OpCT-001，目前都处于临床阶段。

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 1 clinical trial, Clarico.

贝马单抗已获得美国食品药品监督管理局（FDA）的再生医学先进疗法（RMAT）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，该部门致力于投资能够带来范式转变的突破性创新。2019年底，BlueRock成为拜耳集团旗下的全资独立运营子公司。我们的文化以无畏任何挑战的勇气、变革医学并传递希望的紧迫感、以使命为导向的诚信以及心系社区的精神为核心，深知我们每个人都是比自身更宏大的事业的一部分。

For more information, visit .

欲了解更多信息，请访问。

www.bluerocktx.com.

www.bluerocktx.com.

About Bayer

关于拜耳

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。在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 .

研发费用共计62亿欧元。欲了解更多信息，请访问 。