Bayer AG and BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned independently operated subsidiary of Bayer AG today announced positive 24-month data from exPDite, a Phase 1 clinical trial of bemdaneprocel, an investigational cell therapy for the treatment of Parkinson's disease. .

拜耳公司和临床分期细胞治疗公司BlueRock Therapeutics LP，拜耳公司全资独立运营的子公司，今天宣布了exPDite的24个月阳性数据，exPDite是bemdaneprocel的一期临床试验，bemdaneprocel是一种治疗帕金森氏病的研究性细胞疗法。。

Bemdaneprocel is the most clinically advanced investigational cell therapy in the U.S. for treating patients living with Parkinson’s disease. The exPDite trial, designed to assess the safety and tolerability of bemdaneprocel, is now complete and the 24-month data will be presented at the International Congress of Parkinson's Disease and Movement Disorders in Philadelphia on September 28, 2024..

Bemdaneprocel是美国临床上最先进的研究性细胞疗法，用于治疗帕金森病患者。exPDite试验旨在评估bemdaneprocel的安全性和耐受性，现已完成，24个月的数据将于2024年9月28日在费城举行的帕金森病和运动障碍国际大会上公布。。

The safety profile at 24 months is consistent with earlier findings, demonstrating that bemdaneprocel continues to be well-tolerated by patients, with no adverse events reported related to bemdaneprocel. Transplanted cells continue to survive and engraft in the brain after discontinuing immunosuppression therapy at 12 months as outlined in the study’s protocol.

24个月时的安全性与早期的研究结果一致，表明患者对bemdaneprocel的耐受性仍然很好，没有报道与bemdaneprocel相关的不良事件。如研究方案所述，在12个月时停止免疫抑制治疗后，移植的细胞继续存活并植入大脑。

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 Hauser PD 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从基线到随访期间继续显示出积极的趋势，高剂量组的趋势比低剂量组的趋势更令人鼓舞。这些由MDS统一帕金森病评定量表第二部分和第三部分（MDS-UPDRS第二部分和第三部分）和豪泽PD日记（用于评估帕金森病运动症状严重程度的工具）进行评估。

These consistent positive trends suggest that bemdaneprocel could potentially offer sustained benefit for movement impairments caused by the disease..

这些一致的积极趋势表明，bemdaneprocel可能为该疾病引起的运动障碍提供持续的益处。。

“There is considerable momentum in the concept of restoring dopamine inputs in the brain using transplanted cells, and the positive results from the exPDite trial leads the drive forward,” said Claire Henchcliffe, MD, chair of the UCI School of Medicine Department of Neurology at the University of California, Irvine and one of the study’s Principal Investigators.

加州大学欧文分校UCI医学院神经病学系主任、该研究的主要研究者之一、医学博士克莱尔·亨奇克利夫（ClaireHenchcliffe）表示：“使用移植细胞恢复大脑中多巴胺输入的概念有相当大的发展势头，exPDite试验的积极结果引领了这一进程。”。

“The completed study demonstrates that the transplanted cells survive and there are early signs that bemdaneprocel can potentially help patients to better control their motor symptoms. These are exciting results that warrant further exploration in a next phase placebo-controlled study.”.

“已完成的研究表明，移植的细胞存活下来，并且有早期迹象表明bemdaneprocel可能有助于患者更好地控制其运动症状。这些令人兴奋的结果值得在下一阶段安慰剂对照研究中进一步探索。”。

In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on motor symptoms using MDS-UPDRS Part III measured in the “OFF”-medication state, showed a mean reduction of 21.9 points compared with baseline. The low dose cohort showed a mean decrease of 8.3 points. Using the Hauser 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, the 7 participants in the high dose cohort showed a mean increase of 1.8 hours in time spent in the “Good ON” state without troublesome dyskinesias compared with baseline after 24 months.

在高剂量队列中，使用MDS-UPDRS第三部分在“停药”状态下测量的贝美特罗对运动症状影响的24个月测量显示，与基线相比，平均降低了21.9分。低剂量队列显示平均下降8.3分。使用Hauser PD日记，当症状得到很好的控制时，将患者分类为“开启”状态，当症状恶化时，将患者分类为“关闭”状态，高剂量队列中的7名参与者显示，与24个月后的基线相比，在“良好开启”状态下花费的时间平均增加了1.8小时，没有麻烦的运动障碍。

Time spent in the “OFF” state showed a mean decrease of 1.9 hours from baseline after 24 months. The 5 participants in the low dose cohort showed a mean decrease of 0.8 hours in the “Good ON” state time compared with baseline and a corresponding mean increase of 0.4 hours in “OFF” state time..

24个月后，处于“关闭”状态的时间平均比基线减少1.9小时。低剂量队列中的5名参与者显示，“良好开启”状态时间与基线相比平均减少0.8小时，“关闭”状态时间相应平均增加0.4小时。。

In the high dose cohort, the 24-month measurement of the effects of bemdaneprocel on activities of daily living using MDS-UPDRS Part II measured a mean reduction of 3.4 points compared with baseline. The low dose cohort showed a mean increase of 2.0 points.

在高剂量队列中，使用MDS-UPDRS第二部分对bemdaneprocel对日常生活活动影响的24个月测量结果显示，与基线相比，平均降低了3.4分。低剂量队列显示平均增加2.0分。

Condition: Parkinsons

状况：帕金森氏症

Type: drug

类型：药物