Spinogenix宣布FDA授权SPG302的扩大使用计划，这是首个用于治疗ALS的突触再生疗法-动脉网

Spinogenix Announces FDA-Authorized Expanded Access Program for SPG302, the First Synaptic Regenerative Therapy to Treat ALS

CISION 等信源发布 2025-05-05 20:30

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FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study

FDA批准扩大获取计划（EAP），以帮助200名不符合临床研究条件的ALS患者获得SPG302

EAP Supports Real-world Data Collection Concurrent to Ongoing Clinical Studies of SPG302

EAP支持与SPG302正在进行的临床研究同步的真实世界数据收集

LOS ANGELES

洛杉矶

，

May 5, 2025

2025年5月5日

/PRNewswire/ --

Spinogenix, Inc.

旋基因公司

, a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that it has received notification from the Food and Drug Administration (FDA) to begin its Expanded Access Program (EAP) for SPG302 in .

，一家临床阶段的生物制药公司，率先开发首创的恢复突触的治疗方法以改善全球患者的生活，今天宣布已收到美国食品药品监督管理局（FDA）的通知，开始其SPG302的扩大使用计划（EAP）。

the United States

美国

(U.S.) for people living with Amyotrophic Lateral Sclerosis (ALS) that meet program eligibility criteria.

（美国）适用于符合项目资格标准的肌萎缩侧索硬化症（ALS）患者。

SPG302 is being developed as the first synaptic regenerative approach to treating ALS, with the potential to slow or reverse declines in cognitive and motor function.

SPG302 正在被开发为首个用于治疗 ALS 的突触再生方法，有可能减缓或逆转认知和运动功能的衰退。

Spinogenix CEO and Founder Dr.

Spinogenix首席执行官兼创始人博士

Stella Sarraf

斯特拉·萨拉夫

stated, 'FDA clearance to launch this new EAP supports its awareness of SPG302's safety and potential efficacy, and the need for ALS patients to have access to novel treatments. This EAP reflects our dedication to giving hope to people living with ALS and their families, providing treatment to those who do not qualify for, or may be unable to participate in, our ongoing clinical trial.

表示：“FDA批准启动这一新的早期获取计划（EAP）表明其对SPG302的安全性和潜在疗效的认可，以及ALS患者获得新型治疗的必要性。该EAP体现了我们为ALS患者及其家人带来希望的决心，为那些不符合条件或可能无法参与我们正在进行的临床试验的患者提供治疗。”

Innovation is urgently needed to tackle ALS, a disease with devastating impact, and we are committed to ensuring that no patients who may benefit are left behind.'.

创新对于应对ALS这种具有毁灭性影响的疾病至关重要，我们致力于确保不会遗漏任何可能受益的患者。'

EAPs are designed to allow individuals with serious or life-threatening conditions to access investigational treatments outside of a clinical trial that are not yet approved by the FDA.

EAP旨在让患有严重或危及生命疾病的个体在临床试验之外获得尚未得到FDA批准的试验性治疗。

Dan Doctoroff

丹·多克托罗夫

, founder and chairman of Target ALS, and the first individual to receive SPG302 in the U.S. as part of a separate EAP protocol commented, 'The notion that ALS is an untreatable disease is outdated. My own experience with SPG302, the first synaptic regenerative therapy in clinical development, has shown that partial stabilization of the disease is a possibility.

Target ALS创始人兼主席，同时也是美国首位根据单独的EAP协议接受SPG302治疗的人评论道：“ALS是一种无法治疗的疾病这一观念已经过时。我自己使用SPG302这种首个在临床开发中的突触再生疗法的经验表明，部分稳定病情是有可能的。”

I am thrilled that the FDA has recognized the value of allowing Spinogenix to expand access to SPG302, allowing others battling ALS to have a similar beneficial experience.'.

我非常激动FDA已经认识到允许Spinogenix扩大SPG302的使用范围的价值，这将使其他与ALS抗争的患者也能获得类似的有益体验。

In addition to serving the ALS community by providing access to an innovative investigational therapy, the EAP enables the collection of real-world data on SPG302 safety and efficacy that can support its clinical development. The FDA previously granted SPG302 Orphan Drug Designation for the treatment of ALS..

除了通过提供获得创新研究性疗法的机会来服务ALS社区外，EAP还能够收集有关SPG302安全性和有效性的现实世界数据，以支持其临床开发。FDA此前已授予SPG302用于治疗ALS的孤儿药资格。

Spinogenix recently completed a Phase 2 ALS trial in

Spinogenix最近完成了ALS的2期试验

Australia

澳大利亚

, and patients have been offered a continuation of treatment in an open label extension. Additional information on the trial (

，并且患者已被提供在开放标签延长期中继续治疗的机会。有关试验的更多信息 (

NCT05882695

) and open label extension (

`) 和开放标签扩展 (`

NCT06903286)

NCT06903286）

may be found on ClinicalTrials.gov.

可以在ClinicalTrials.gov上找到。

About SPG302

关于SPG302

SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function.

SPG302 是一种每日一次的口服药物，正在被开发用于治疗神经退行性和神经精神疾病，其独特之处在于能够恢复突触——神经元之间允许人们思考、计划、记忆和控制运动的关键连接。SPG302 的突触再生活性代表了一种首创新方法来治疗这些疾病，并有可能逆转认知、呼吸和运动功能的衰退。

SPG302 has been granted U.S. FDA Orphan Drug Designation for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense..

SPG302 已获得美国 FDA 授予的孤儿药资格，用于治疗 ALS，并获得了美国国立卫生研究院和国防部的临床前支持。

Spinogenix has completed a Phase 1/2 study in

Spinogenix 已完成了一项 1/2 期研究

Australia

澳大利亚

(

NCT05882695

) assessing the safety, pharmacokinetics and pharmacodynamics of once-daily dosing of SPG302 in healthy volunteers and up to 48 weeks of treatment in ALS patients. Additional information on the global clinical trials evaluating SPG302 for the treatment of schizophrenia and Alzheimer's disease can be found on ClinicalTrials.gov (.

）评估SPG302每日一次给药在健康志愿者中的安全性、药代动力学和药效学，以及ALS患者长达48周的治疗。有关全球临床试验的更多信息，评估SPG302用于治疗精神分裂症和阿尔茨海默病的信息可以在ClinicalTrials.gov上找到（。

NCT06442462

and

和

NCT06427668

）。

About Expanded Access to SPG302

关于扩大对SPG302的访问

Access to SPG302 is available in the U.S. for certain adults with ALS who meet eligibility criteria for participation in the FDA-authorized Expanded Access Program for SPG302. More information about the U.S. EAP can be requested through

在美国，符合FDA授权的SPG302扩大使用计划（EAP）参与资格的某些ALS成年患者可以获得SPG302的使用权限。有关美国EAP的更多信息，可以通过以下方式请求。

contact@spinogenix.com

。

About Spinogenix

关于Spinogenix

Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on

当前对神经退行性、神经精神和神经发育状况的治疗主要集中在

slowing

减速

disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope..

疾病进展或症状最小化，让许多人对改善失去希望。Spinogenix 旨在通过其首创的、改变范式的突触再生和突触矫正疗法，彻底改变这些病症的治疗方法，该疗法旨在恢复耗尽的突触并逆转突触退化和功能障碍，为患者及其家庭带来新的希望现实。

Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms.

Spinogenix正在开发两种新型疗法：SPG302，可触发神经元产生新的谷氨酸能突触，并在ALS、阿尔茨海默病、精神分裂症等疾病中恢复认知、运动及其他功能；以及SPG601，它在突触水平上作用，纠正脆性X综合征（FXS）中导致许多核心症状的特定功能障碍。

The company has received FDA Orphan Drug designation for both ALS and FXS, as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at .

该公司已获得FDA对ALS和FXS的孤儿药资格，以及FDA对FXS的快速通道资格。更多关于Spinogenix的信息可以在找到。

www.spinogenix.com

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