Aspen Neuroscience宣布了帕金森病个性化细胞疗法ASPIRO 1/2a期临床试验的6个月结果-动脉网

Aspen Neuroscience Announces 6-Month ASPIRO Phase 1/2a Clinical Trial Results of Personalized Cell Therapy for Parkinson's Disease

CISION 等信源发布 2025-05-07 23:11

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ANPD001 was well tolerated with no major safety issues in low-dose cohort

ANPD001在低剂量组中耐受性良好，无重大安全问题。

Patients showed early improvements in patient-reported and clinician-reported outcomes

患者在患者报告和临床医生报告的结果中显示出早期改善。

Detailed Phase 1/2a trial data will be presented this week at IAPRD 2025

详细的1/2a期试验数据将在本周的IAPRD 2025上公布。

SAN DIEGO

圣地亚哥

and

和

NEW YORK

纽约

，

May 7, 2025

2025年5月7日

/PRNewswire/ -- Aspen Neuroscience has released 6-month data from the first three patients dosed in the ASPIRO Phase 1/2a trial of ANPD001, an investigational autologous dopaminergic neuronal precursor cell (DANPC) therapy being studied in patients with moderate to advanced Parkinson's Disease (PD)..

/PRENewswire/ -- Aspen Neuroscience发布了ASPIRE 1/2a期试验中前三位接受治疗的患者的6个月数据，该试验涉及的ANPD001是一种正在研究中的自体多巴胺能神经前体细胞（DANPC）疗法，用于治疗中度至晚期帕金森病（PD）患者。

Autologous iPSC-derived neurons (PRNewsfoto/Aspen Neuroscience, Inc.)

自体iPSC衍生神经元（PRNewsfoto/Aspen Neuroscience, Inc.）

Detailed 6-month data on the first three patients will be presented at this week's IAPRD 2025 conference in NYC

前三位患者的详细六个月数据将在本周于纽约市举行的IAPRD 2025会议上展示。

Post this

发布这个

After recent publication in

最近出版后

Parkinsonism and Related Disorders

帕金森综合征及相关疾病

，

detailed 6-month data on the first three patients will be presented this week at the 30

前三位患者详细的6个月数据将在本周的第30次

World Congress on Parkinson's Disease and Related Disorders (

帕金森病及相关疾病世界大会 (

IAPRD 2025

国际先进材料与工艺研讨会 2025

), taking place in

)，发生在

New York

纽约市

from May 7-10. Aspen Neuroscience Medical Director

从5月7日至10日。阿斯彭神经科学医学主任

Avram Fraint

亚伯拉罕·弗雷恩特

, M.D., M.S. will present '

`, 医学博士，理学硕士将会展示 '`

Safety, tolerability, and efficacy of intracranial delivery of autologous iPSC-derived dopaminergic precursors in moderate to advanced Parkinson's disease

中度至重度帕金森病患者颅内递送自体iPSC衍生的多巴胺能前体细胞的安全性、耐受性和有效性

' on

' 开启

Saturday, May 10

5月10日，星期六

during the IAPRD Guided Poster session.

在IAPRD引导的海报会议期间。

'Parkinson's disease is the second most common neurodegenerative disorder. By the time of diagnosis, most people with Parkinson's have lost the majority of their dopaminergic neurons, leading to progressive loss of motor and non-motor function,' said Dr. Fraint. 'To date, data from the first three patients in the ASPIRO study show that precision delivery of personalized DANPCs is safe and well-tolerated.'.

帕金森病是第二大最常见的神经退行性疾病。Dr. Fraint表示：“到诊断时，大多数帕金森病患者已经失去了大部分多巴胺能神经元，导致运动和非运动功能的逐渐丧失。” “迄今为止，ASPIRO研究前三位患者的数据表明，精确递送个性化的DANPCs是安全且耐受性良好的。”

'In this first-of-its-kind study, we are seeing clinician-reported and patient-reported improvements as well as a strong safety and tolerability profile at six months. Importantly, ANPD001 has the unique advantage of not requiring immunosuppression

“在这项前所未有的研究中，我们看到了临床医生报告和患者报告的改善，同时在六个月时表现出强大的安全性和耐受性。重要的是，ANPD001具有无需免疫抑制的独特优势。

，

said

说

Edward Wirth III

爱德华·沃斯三世

, MD, PhD, Chief Medical Officer of Aspen Neuroscience.

医学博士，哲学博士，阿斯彭神经科学首席医学官。

'We are very encouraged by the ASPIRO study results to date. With more than one million people currently living with Parkinson's disease in the U.S. alone, these data are promising for the Parkinson's community, and for the autologous cell therapy field,' said

“我们对ASPIRO研究迄今为止的结果感到非常鼓舞。仅在美国，目前就有超过一百万人患有帕金森病，这些数据为帕金森病患者群体以及自体细胞治疗领域带来了希望，”

Damien McDevitt

达米安·麦克德维特

, PhD, president and CEO of Aspen Neuroscience.

博士，阿斯彭神经科学公司的总裁兼首席执行官。

About the ASPIRO Study

关于ASPIRO研究

The Autologous-derived Study of a Parkinson's Investigational Regenerative therapy in an Open-label trial (ASPIRO) was launched in 2024 to assess safety and tolerability of ANPD001, an autologous, dopaminergic neuron cell replacement therapy for participants with moderate to advanced PD. It is the first multi-patient, multi-center clinical trial of an autologous cell therapy for PD..

2024年启动了自体来源的帕金森研究性再生疗法开放标签试验（ASPIRO），以评估ANPD001的安全性和耐受性，ANPD001是一种用于中度至晚期帕金森病患者的自体多巴胺能神经元细胞替代疗法。这是首个针对帕金森病的自体细胞疗法的多患者、多中心临床试验。

The ASPIRO trial enrolls levodopa-responsive patients 50–70 years of age whose Parkinson's disease duration, motor fluctuations, and 'off' time have made oral therapies alone incompletely effective. The trial excluded patients with cognitive impairment and other comorbidities that make them high-risk for an intra-cranial therapy.

ASPIRO 试验招募了 50 至 70 岁对左旋多巴有反应的患者，这些患者的帕金森病病程、运动波动和“关”期使得单独使用口服治疗效果不完全。试验排除了有认知障碍和其他使他们成为颅内治疗高风险的合并症的患者。

All enrolled patients are under the care of a movement disorder specialist..

所有登记的患者都在运动障碍专科医生的照顾下。

Primary study objectives include assessing safety and tolerability of two sequential escalating doses of ANPD001 as well as the drug delivery procedure. Secondary objectives include the evaluation of clinical efficacy, as measured by change from baseline in patient- and clinician-reported Parkinson's disease-specific outcome measures.

主要研究目标包括评估两种顺序递增剂量的ANPD001及其药物输送程序的安全性和耐受性。次要目标包括通过患者和临床医生报告的帕金森病特定结果指标从基线的变化来评估临床疗效。

This includes improvements in motor symptoms and quality of life based on standard Parkinson's disease rating scales, and improvement in 'good on' time, when patients experience periods of good symptom control without medication side effects..

这包括基于标准帕金森病评定量表的运动症状和生活质量的改善，以及“良好开启”时间的改善，这时患者会经历无药物副作用的良好症状控制期。

Primary safety and efficacy endpoints will be reported at 12 months, and continued follow up and evaluation will continue for five years. Long-term safety follow-up will continue to Year 15.

主要安全性和有效性终点将在12个月时报告，持续随访和评估将继续进行五年。长期安全性随访将持续到第15年。

Study Procedure and Results

研究程序与结果

Administration of the DANPCs into the posterior putamen was performed with participants under general anesthesia. Precision dosing of the intended target was enabled using gadoteridol-enhanced intraoperative MRI. These three patients received five million DANPCs per putamen in a single surgical procedure using a custom syringe and cannula..

在全身麻醉下，将DANPCs注入后壳核。通过使用钆特醇增强的术中MRI，实现了对目标靶点的精确给药。这三名患者在单一手术过程中，使用定制的注射器和导管，每侧壳核接受了五百万个DANPCs。

No hemorrhages nor serious intra-operative or post-operative complications were observed. No graft-induced dyskinesia have been observed. The most common adverse events in the first three patients were incision pain and tongue swelling, the latter of which is related to the prone position during surgery..

未观察到出血及严重的术中或术后并发症。未发现移植引起的运动障碍。前三位患者最常见的不良事件是切口疼痛和舌头肿胀，后者与手术期间的俯卧位有关。

At six months there was an average improvement of 45% in the physician-reported MDS-Unified Parkinsons Disease Part III 'off' score compared to baseline, and an average improvement of 71% in patient-reported MDS-UPDRS Part II scores. There was an average reduction of 2.0 hours in adjusted 'off time,' and a 1.5-hour average increase in adjusted 'good on' time..

六个月时，医生报告的MDS-统一帕金森病评定量表第三部分“关闭”期评分较基线平均改善了45%，患者报告的MDS-UPDRS第二部分评分平均改善了71%。调整后的“关闭”时间平均减少了2.0小时，而调整后的“良好开启”时间平均增加了1.5小时。

About ANPD001

关于ANPD001

ANPD001 is the most advanced autologous investigational cell therapy in the United States for treating Parkinson's disease. More information about the Phase 1/2a trial is available at clinicaltrials.gov (NCT06344026).

ANPD001 是美国最先进的自体研究性细胞疗法，用于治疗帕金森病。有关 1/2a 期试验的更多信息，请访问 clinicaltrials.gov (NCT06344026)。

Aspen's

阿斯彭的

personalized approach means that patients do not require immunosuppressive (IS) drugs to dampen the body's immune response against foreign cells. This approach will eliminate IS-associated adverse events, IS drug-monitoring requirements, and enable dosing for those with contraindications to IS therapies..

个性化方法意味着患者不需要使用免疫抑制剂（IS）药物来抑制身体对外来细胞的免疫反应。这种方法将消除与IS相关的不良事件、IS药物监测需求，并为那些对IS治疗有禁忌症的患者提供剂量方案。

Aspen's

阿斯彭的

manufacturing process starts from a small biopsy of the patient's own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) and then differentiation of the iPSCs into DANPCs. These DANPCs are transplanted into the posterior putamen, replacing cells that were lost or damaged due to disease.

制造过程从小小的患者自身皮肤细胞活检开始，随后重新编程为诱导多能干细胞 (iPSC)，然后将这些 iPSC 分化为 DANPC。这些 DANPC 被移植到后壳核中，替代因疾病而丧失或受损的细胞。

The quality of each person's cells is assessed at every manufacturing stage using Aspen's proprietary machine learning-based genomics tests..

在每个生产阶段，都会使用Aspen专有的基于机器学习的基因组学测试来评估每个人的细胞质量。

ANPD001 has received Fast Track designation by the U.S. Food & Drug Administration (FDA).

ANPD001 已获得美国食品药品监督管理局 (FDA) 的快速通道资格。

About Aspen Neuroscience

关于阿斯彭神经科学

Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company's patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for PD. .

总部位于圣地亚哥的Aspen Neuroscience公司是一家专注于自体再生医学的临床开发阶段的私人公司。该公司利用患者衍生的诱导多能干细胞（iPSC）平台来开个性化疗法，以应对具有高度未满足医疗需求的疾病，首先是用于帕金森病（PD）的自体神经元替代疗法。

Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control.

Aspen结合细胞生物学与最新的机器学习和基因组学方法，研究针对患者的恢复性细胞治疗。该公司开发了一个领先的平台，用于创建和优化多能干细胞衍生的细胞疗法，其中包括内部生物信息学、制造和质量控制。

For more information and important updates, please visit .

如需更多信息和重要更新，请访问 。

https://www.aspenneuroscience.com

。

SOURCE Aspen Neuroscience, Inc.

来源：Aspen Neuroscience, Inc.

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