Perfuse Therapeutics在美国青光眼学会会议上宣布了 PER-001在青光眼患者中的 Ph1/2a期试验的视觉、结构和安全性数据均呈现积极结果-动脉网

Perfuse Therapeutics Announces Positive Visual, Structural and Safety Data from a Ph1/2a Trial of PER-001 in Glaucoma Patients at the American Glaucoma Society Meeting

CISION 等信源发布 2025-03-03 20:30



可切换为仅中文







Twenty-four-week data from the Ph1/2a trial in glaucoma patients reveals PER-001 to be well-tolerated. Proof of concept is established with both strong functional and structural outcomes

为期24周的Ph1/2a期青光眼患者试验数据显示，PER-001具有良好耐受性。通过强大的功能和结构结果，概念验证得以确立。

Glaucoma patients treated with PER-001 on top of IOP reducing therapies experienced a mean improvement in visual field mean deviation of 1 decibel (dB) per year and a mean increase in retinal nerve fiber layer thickness of 3 microns at 24 weeks

接受PER-001联合降低眼压疗法治疗的青光眼患者，其视野平均偏差每年平均改善1分贝（dB），24周时视网膜神经纤维层厚度平均增加3微米。

SAN FRANCISCO

旧金山

，

March 3, 2025

2025年3月3日

/PRNewswire/ -- Perfuse Therapeutics, a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases announced today the presentation of 24-week data from its first-in-human Phase 1/2a trial of PER-001 intravitreal implant in patients with glaucoma. The data was presented by .

/PRNewswire/ -- Perfuse Therapeutics，一家致力于开发治疗缺血性眼病的变革性疗法的生物制药公司，今日宣布了其针对青光眼患者的PER-001玻璃体内植入物的首次人体Phase 1/2a试验的24周数据。数据由发言人展示。

Steven Mansberger

史蒂文·曼斯伯格

, MD, MPH, Chief of Ophthalmology, Director of Glaucoma Services at Legacy Devers Eye Institute,

医学博士，公共卫生硕士，Legacy Devers眼科研究所眼科主任，青光眼服务部主任，

March 1, 2025

2025年3月1日

, at the American Glaucoma Society Meeting in

，在美国青光眼学会会议上

Washington DC

华盛顿特区

。

The trial is fully enrolled with glaucoma patients (n=33) receiving a single administration of PER-001 on top of IOP reducing therapies. Data presented included results from the Ph1 (n=6) and Ph2a Dose 1 (n=12) cohorts, both of which completed the trial. The Ph2a Dose 2 (n=15) cohort is ongoing.

该试验已全部招募了青光眼患者（n=33），他们在降低眼压的治疗基础上接受了单次PER-001给药。所展示的数据包括第1阶段（n=6）和第2a阶段剂量1（n=12）组的结果，这两组均已完成试验。第2a阶段剂量2（n=15）组正在进行中。

The 24-week data demonstrated that PER-001 was well-tolerated, with only one drug-related adverse event of vitreous floater (mild and transient), no drug-related serious adverse events and no reports of intraocular inflammation. Patients treated with PER-001 showed a mean increase in optic nerve head blood flow of at least 10% from baseline starting at Week 1, which was sustained out to Week 24 and was associated with a mean improvement in visual field mean deviation of 1 dB/yr and a mean increase in retinal nerve fiber layer thickness of 3 microns, as measured by optical coherence tomography (OCT) at Week 24.

24周数据显示，PER-001耐受性良好，仅出现一例药物相关的玻璃体漂浮物不良事件（轻微且短暂），无药物相关的严重不良事件，也无眼内炎症报告。接受PER-001治疗的患者从第1周开始视神经头血流量较基线平均增加至少10%，这种效果持续到第24周，并伴随视野平均偏差每年平均改善1分贝（dB），以及通过光学相干断层扫描（OCT）测量在第24周时视网膜神经纤维层厚度平均增加3微米。

In contrast, the sham control group revealed a 0.63 dB/yr decline in visual field mean deviation and a decrease of 1.3 microns in OCT RNFL at Week 24..

相比之下，假手术对照组在第24周时视野平均偏差下降了0.63 dB/年，OCT RNFL厚度减少了1.3微米。

'These results are distinctive and noteworthy,' stated

“这些结果独特且值得注意，”

Steven Mansberger

史蒂文·曼斯伯格

, MD, MPH. 'There remains an unmet need for neuroprotective therapies for patients with glaucoma and while this is early data, the observed improvements in the optic nerve head blood flow, as well as key functional and structural outcomes are very promising.'

医学博士、公共卫生硕士。“青光眼患者对神经保护疗法的需求仍未得到满足，虽然这是早期数据，但观察到的视神经头部血流改善以及关键功能和结构结果非常有希望。”

'We are excited to share first-in-human data from the ongoing PER-001 trials,' said

“我们很高兴分享正在进行的PER-001试验的首次人体数据，”

Sevgi Gurkan

塞维吉·古尔坎

, MD, CEO and Founder. 'Endothelin is a well-established, clinically validated pathway with many approved products in systemic diseases. The Ph1/2a trial marks the first ever clinical application of an endothelin antagonist for local treatment of back of the eye diseases, and we are encouraged by the safety profile and efficacy data to date.'.

医学博士，首席执行官兼创始人。 “内皮素是一种已确立的、经过临床验证的通路，在系统性疾病中已有许多获批产品。Ph1/2a 期试验标志着内皮素拮抗剂首次用于眼部后段疾病的局部治疗，迄今为止的安全性和有效性数据让我们备受鼓舞。”

'Glaucoma is the leading cause of irreversible blindness worldwide, and there are no currently approved therapies. This trial provides the first clinical validation of the key role of endothelin pathway in glaucoma. The results of this trial are highly encouraging and the data supports the potential of PER-001 intravitreal implant as a disease-modifying therapy that could significantly improve the vision and lives of glaucoma patients,' said Phil Lai, MD, Chief Medical Officer. .

“青光眼是全球不可逆失明的主要原因，目前尚无获批的疗法。这项试验首次临床验证了内皮素通路在青光眼中的关键作用。试验结果非常令人鼓舞，数据支持PER-001玻璃体内植入物作为一种疾病修饰疗法的潜力，可以显著改善青光眼患者的视力和生活。”首席医学官Phil Lai博士表示。

About PER-001 Intravitreal Implant, a Long Acting Endothelin Receptor Antagonist

关于PER-001玻璃体内植入物，一种长效内皮素受体拮抗剂

PER-001 is a novel (NCE), first-in-class small molecule endothelin receptor antagonist. Endothelin is the most potent vasoconstrictor in the human body and is upregulated in glaucoma. Endothelin is involved in inflammation and cell death through its receptors expressed in retinal vascular and neuroretinal cells.

PER-001 是一种新型（NCE）、首创新药类的小分子内皮素受体拮抗剂。内皮素是人体中最有效的血管收缩剂，并在青光眼中上调。内皮素通过其在视网膜血管和神经视网膜细胞中表达的受体参与炎症和细胞死亡。

The PER-001 intravitreal implant is a bio-erodible implant, administered into the vitreous cavity of the eye using a single use, 25-gauge applicator and designed to provide a sustained release of PER-001, allowing for a convenient every 6 months dosing frequency. .

PER-001玻璃体内植入物是一种生物可降解植入物，使用一次性25号规格的施药器将其注入眼球的玻璃体腔内，设计用于持续释放PER-001，从而实现每6个月一次的便捷给药频率。

About the Ph1/2a Trial

关于Ph1/2a试验

The Phase 1/2a trial is a multi-center two-part trial designed to assess the safety, tolerability and pharmacodynamics of PER-001 in glaucoma patients. The Phase 1 part of the trial was a first-in-human, open-label, single ascending dose (SAD) study that evaluated the safety and tolerability of two doses of PER-001 intravitreal implant in patients with advanced glaucoma.

1/2a期试验是一项多中心两部分试验，旨在评估PER-001在青光眼患者中的安全性、耐受性和药效学。该试验的第1阶段是首次人体试验，开放标签、单剂量递增（SAD）研究，评估了两种剂量的PER-001玻璃体内植入物在晚期青光眼患者中的安全性和耐受性。

The Phase 2a part of the trial was a patient and reading center masked, randomized, sham-controlled study that evaluated the safety, tolerability, and pharmacodynamics of two doses of PER-001 in patients with progressing, mild to moderate glaucoma..

试验的第2a阶段部分是一项患者和阅读中心掩蔽、随机、假对照研究，评估了两种剂量的PER-001在进展性、轻至中度青光眼患者中的安全性、耐受性和药效学。

Patients in both phases received a single intravitreal administration of PER-001 while maintaining their pre-study IOP-lowering therapies. Two doses of PER-001 intravitreal implant were tested sequentially in cohorts of 3 patients each during the Ph1 SAD portion and the same two doses were subsequently tested in a larger cohort of patients during the Ph2a portion of the trial.

两阶段的患者均接受了一次PER-001玻璃体内注射，同时继续使用他们之前研究中的降眼压疗法。在第一阶段单剂量递增（Ph1 SAD）部分，每组3名患者依次测试了两种剂量的PER-001玻璃体内植入物，随后在第二阶段a部分（Ph2a）试验中，同样的两种剂量在更大组的患者中进行了测试。

All patients were followed for 24 weeks from their first and single dose. The Ph1 cohorts and Ph2a Dose 1 cohort have completed the trial. Ph2a Dose 2 cohort is ongoing and the topline data will be available in second quarter 2025..

所有患者均从首次单剂量接种后随访24周。1期和2a期剂量1组已经完成试验。2a期剂量2组正在进行中，预计将在2025年第二季度获得初步数据。

About Perfuse Therapeutics, Inc.

关于Perfuse Therapeutics公司

Perfuse Therapeutics is a biopharmaceutical company pioneering transformational therapies to treat ischemia-induced ocular diseases. Enabled by its proprietary sustained release drug delivery platform, the company is developing a robust pipeline of novel assets against validated targets. Headquartered in .

Perfuse Therapeutics是一家生物制药公司，致力于开发治疗缺血性眼病的变革性疗法。依托其专有的缓释药物递送平台，该公司正在开发针对已验证靶点的多样化新型资产组合。公司总部位于。

South San Francisco, California

加利福尼亚州南旧金山

and with R&D facilities in

并在以下地方设有研发设施

Durham, North Carolina

北卡罗来纳州德罕市

, Perfuse Therapeutics has established a strong team committed to advancing disease modifying therapies to patients with ocular disease around the world. For more information, please see

，Perfuse Therapeutics组建了一支强大的团队，致力于为全球眼部疾病患者推进疾病修饰疗法。欲了解更多信息，请参见

www.perfusetherapeutics.com

and on LinkedIn.

并在领英上。

Investor Relations and Media Contact

投资者关系及媒体联系

Please contact us for more information or questions at –

如需更多信息或有任何问题，请通过以下方式联系我们 –

info@perfusetherapeutics.com

SOURCE Perfuse Therapeutics

源 Perfuse Therapeutics

WANT YOUR COMPANY'S NEWS