Things are continuing to look up for Bayer’s Parkinson’s disease cell therapy, with the German pharma giant finally offering a deeper dive into phase 1 data that confirms the candidate hit its key safety endpoints.

事情正在继续寻找拜耳的帕金森病细胞疗法，德国制药巨头最终提供了对第一阶段数据的更深入的了解，证实候选人达到了关键的安全终点。

The stem-cell-derived therapy, bemdaneprocel, has been in development at Bayer’s BlueRock Therapeutics subsidiary. The phase 1 trial kicked off in 2021 with the aim of testing the safety and tolerability of surgically transplanting dopamine-producing cells in the brains of 12 people with Parkinson’s.

干细胞衍生疗法bemdaneprocel已在拜耳BlueRock Therapeutics子公司开发。第一阶段试验于2021年启动，目的是测试12名帕金森病患者大脑中手术移植多巴胺产生细胞的安全性和耐受性。

The goal was to assess the rate of serious adverse events and abnormal tissue overgrowth in the year after transplantation..

目的是评估移植后一年内严重不良事件和异常组织过度生长的发生率。。

The early signs from the study were already promising, with Bayer highlighting in June that bemdaneprocel appeared safe based on a first glance at the data. Now, the company has confirmed the trial hit its primary endpoint of demonstrating safety and tolerability in all 12 patients.

该研究的早期迹象已经很有希望，拜耳在6月份强调，bemdaneprocel基于乍一看的数据似乎是安全的。现在，该公司已确认该试验的主要终点是证明所有12名患者的安全性和耐受性。

Ahead of presenting detailed phase 1 data at the International Congress on Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark, Bayer’s head of R&D Christian Rommel told Fierce Biotech that the company is now preparing for a phase 2 study of bemdaneprocel, which will be focused on “strengthening the power of the data.”.

拜耳研发主管克里斯蒂安·罗梅尔（Christian Rommel）在丹麦哥本哈根举行的帕金森病和运动障碍国际大会上提交详细的第一阶段数据之前，告诉Violet Biotech，该公司目前正在为bemdaneprocel的第二阶段研究做准备，重点是“加强数据的力量”。

“Hopefully in two years from now, we [have] the next inflection point or milestone, then we can go all-in for registration data and a lot more emphasis on efficacy,” Rommel said.

Rommel说：“希望在两年之后，我们[有]下一个拐点或里程碑，那么我们就可以全面注册数据，更加重视疗效。”。

Today’s readout showed that patients who received the higher dose of 2.7 million cells saw a 2.16-hour improvement in time spent each day in the “on” state—a term for when Parkinson’s symptoms are well controlled—without troubling involuntary movements known as dyskinesia. Conversely, time spent in the “off” state—where symptoms worsen—reduced by 1.91 hours a day..

今天的读数显示，接受较高剂量270万个细胞的患者每天在“开”状态下花费的时间改善了2.16小时-这是帕金森氏症状得到良好控制而不会引起称为运动障碍的不自主运动的术语。相反，在症状恶化的“关闭”状态下花费的时间每天减少1.91小时。。

Those patients who received the lower dose of 0.9 million cells also saw an improvement, although to a lesser extent, with an increase of 0.72 hours in the “on” state and 0.75 hours less in the “off” state, Bayer explained.

拜耳解释说，那些接受较低剂量90万个细胞的患者也有所改善，尽管程度较小，“开”状态增加了0.72小时，“关”状态减少了0.75小时。

What’s more, PET imaging scans demonstrated evidence of the engrafted cells surviving in both the low- and high-dose cohorts, Bayer noted in its Aug. 28 release. One of the trial’s secondary endpoints was assessing a change in motor function, and, here, the high-dose cohort demonstrated the greater improvement, Bayer added..

拜耳在其8月28日的版本中指出，PET成像扫描显示在低剂量和高剂量组中存活的移植细胞的证据。拜耳补充说，该试验的次要终点之一是评估运动功能的变化，在这里，高剂量队列显示出更大的改善。。

No serious adverse events (SAEs) related to bemdaneprocel were reported in the 12-month data. Of the two SAEs that were reported, one was a seizure attributed to the surgical procedure itself and the other was a case of COVID-19.

在12个月的数据中没有报告与bemdaneprocel相关的严重不良事件（SAE）。在报告的两种SAE中，一种是由于外科手术本身引起的癫痫发作，另一种是COVID-19病例。

While bemdaneprocel needs to be administered directly into the brain via surgery, Rommel rejected the idea that the complex procedure would necessarily reduce uptake should the therapy win approval.

虽然bemdaneprocel需要通过手术直接进入大脑，但Rommel拒绝了这样一种观点，即如果治疗获得批准，复杂的手术必然会减少摄取。

“If you consider what surgeons do in neurology, they are very familiar with those settings, so for them it’s not out of the ordinary,” he said. “The question is at which stage of the disease can we [treat], because of the invasive nature.”

他说：“如果你考虑外科医生在神经病学方面的工作，他们非常熟悉这些设置，所以对他们来说这并不常见。”。“由于侵入性，问题是我们可以[治疗]疾病的哪个阶段。”

Bayer’s vision for bemdaneprocel is to find a drug that can be used in combination with the cell therapy to “further enhance the effect,” which will in turn broaden the range of patients who can access the treatment, Rommel said.

Rommel说，拜耳的bemdaneprocel愿景是找到一种可与细胞疗法结合使用的药物，以“进一步增强疗效”，从而扩大可以接受治疗的患者范围。

“Our dream is to have this single-time intervention,” he added. “If that’s going to work and it’s safe then it will be used, because at the moment there is no alternative.”

他补充说：“我们的梦想是一次性干预。”。“如果这将起作用并且安全，那么它将被使用，因为目前没有其他选择。”

Ever since partnering with Versant Ventures back in 2016 to get BlueRock off the ground, Bayer has held out hope that the biotech’s pluripotent-stem-cell-based platform could be used to address unmet medical needs. The German pharma went on to buy out the company for $240 million, and Rommel said Bayer “continues to see BlueRock as a key building block of our cell and gene therapy strategy.”.

自从2016年与Versant Ventures合作以实现BlueRock的发展以来，拜耳一直希望生物技术的多能干细胞平台可用于解决未满足的医疗需求。德国制药继续以2.4亿美元的价格收购该公司，罗美尔表示拜耳“继续将BlueRock视为我们细胞和基因治疗策略的关键组成部分”。

Bayer already has plans to turn the platform to retinopathy next year, said Rommel, who “wouldn’t rule out” seeing whether the tech can also be effective in Alzheimer’s disease and dementia.

Rommel说，拜耳已经计划在明年将平台转向视网膜病变，他“不会排除”该技术是否也能有效治疗阿尔茨海默病和痴呆症。