新同行评审研究证实Spinogenix的SPG601在脆性X综合征患者中的神经生理学疗效-动脉网

New Peer-Reviewed Research Demonstrates Neurophysiological Efficacy of Spinogenix's SPG601 in Patients with Fragile X Syndrome

PR Newswire 等信源发布 2026-04-09 20:30

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Study Results, Published in Nature Scientific Reports, Found SPG601 Reduced Signature Changes in High-Frequency Gamma Band Activity and Improved Cognition in FXS Patients

研究结果发表在《自然科学报告》上，发现SPG601减少了高频伽马波段活动的特征变化，并改善了FXS患者的认知功能。

LOS ANGELES

洛杉矶

，

April 9, 2026

2026年4月9日

/PRNewswire/ --

Spinogenix

旋基因科技

, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced the publication of a peer-reviewed article in

有限公司是一家临床阶段的生物制药公司，率先研发首创的恢复突触的治疗方法，以改善全球患者的生活，今天宣布在

Nature Scientific Reports

自然科学报告

, supporting the efficacy of SPG601 in participants with Fragile X Syndrome (FXS). The report shares the results of the Phase 2 clinical trial conducted at Cincinnati Children's Hospital demonstrating that synapse correction may offer a new approach to addressing the cognitive and neurophysiological deficits associated with FXS..

，支持SPG601在脆性X综合征（FXS）患者中的疗效。该报告分享了在辛辛那提儿童医院进行的2期临床试验结果，表明突触修复可能为解决与FXS相关的认知和神经生理缺陷提供一种新方法。

SPG601 is a first-in-class, small-molecule oral tablet designed to modulate the activity of large-conductance, calcium-activated potassium ('BK') channel to correct specific synaptic dysfunctions that underlie many core symptoms of FXS.

SPG601 是一种首创的小分子口服片剂，旨在调节大电导钙激活钾（“BK”）通道的活性，以纠正导致脆性X综合征（FXS）许多核心症状的特定突触功能障碍。

'Seeing these clinical trial results published in Nature Scientific Reports further validates the promise and potential of SPG601 as a first-in-class treatment for FXS,' said Dr. Stella Sarraf, Chief Executive Officer and Founder at Spinogenix. 'This latest milestone, together with continued support from the industry-leading FRAXA Research Foundation on this mechanism of action, provides a strong foundation as we advance this drug into a Phase 2b/3 trial.

“看到这些临床试验结果发表在《自然科学报告》上，进一步验证了SPG601作为FXS首创新疗法的前景和潜力，”Spinogenix首席执行官兼创始人斯特拉·萨拉夫博士表示，“这一最新的里程碑，加上行业领先的FRAXA研究基金会对我们这种作用机制的持续支持，为我们推进该药物进入2b/3期试验提供了坚实的基础。”

With no therapies currently FDA approved for people living with this condition, we are committed to leading the path by furthering the development of SPG601 to help people and their families who have been searching for new treatments.'.

由于目前尚无FDA批准的疗法可用于治疗这种疾病的患者，我们致力于通过进一步开发SPG601来引领道路，以帮助那些一直在寻找新疗法的人们及其家人。

In the published Phase 2a study, 10 adult male participants with genetically confirmed FXS were administered a single 800mg dose of SPG601 and placebo separated by a one-week washout period. SPG601 was well-tolerated with a favorable safety profile. For the first time in FXS treatment, SPG601 demonstrated improvement across the EEG power spectrum including reduction in excessive high frequency gamma band activity and increase in alpha band power that is reduced at baseline in fragile X.

在已发布的 2a 期研究中，10 名基因确认的脆性 X 综合征（FXS）成年男性参与者分别服用单剂 800 毫克的 SPG601 和安慰剂，两次用药间隔一周清洗期。SPG601 具有良好的耐受性和安全性。在脆性 X 综合征治疗中，SPG601 首次显示出在整个脑电图功率谱上的改善，包括降低过高的高频伽马波段活动，并增加在脆性 X 中基线水平较低的阿尔法波段功率。

These corrected EEG deficits show a normalization of brain function in the direction of normal activity levels required for learning and memory. .

这些纠正后的脑电图缺陷显示出大脑功能向学习和记忆所需的正常活动水平的正常化方向发展。

SPG601 treatment was also associated with significant improvement in the NIH TB Flanker task, which measures inhibitory control and attention. FXS is associated with lower overall Flanker scores than other intellectual disabilities, highlighting attention as a significant deficit in the disease population.

SPG601治疗还与NIH TB侧翼任务（测量抑制控制和注意力的任务）的显著改善相关。脆性X综合征（FXS）与其他智力障碍相比，整体侧翼任务得分较低，这突显了注意力是该疾病人群中的显著缺陷。

In addition, cognitive performance as measured by the Fluid Cognition Composite and Total Cognition Composite indices showed an SPG601-associated trend towards cognitive improvement. .

此外，通过流体认知复合指数和总认知复合指数衡量的认知表现显示出与SPG601相关的认知改善趋势。

'These results are the strongest sign of positive target engagement we have ever noted in the fragile X field using our single-dose trial design which has been used to test over a dozen small molecules over the last decade plus. In fact, the combination of positive EEG change and cognitive change has not been seen to this degree in the field ever before in single or chronic dosing trials,' Dr.

“这些结果是我们迄今为止在脆性X染色体领域中使用单剂量试验设计所观察到的最强烈的阳性靶点参与信号。在过去十多年里，这种设计已经被用于测试了十几种小分子化合物。事实上，在单次或长期给药试验中，该领域从未见过如此显著的脑电图和认知变化的结合。”

Craig Erickson, principal investigator of the planned Phase 2b trial, Director of the Cincinnati Fragile X Research and Treatment Center and Spinogenix Chief Medical Advisor commented. 'The rapid improvements in key neurophysiological biomarkers, as measured by EEG, together with improvements in cognitive measures, support the viability of targeting BK channels in FXS.

克雷格·埃里克森 (Craig Erickson) 是计划中的 2b 期试验的首席研究员、辛辛那提脆性 X 研究与治疗中心主任以及 Spinogenix 首席医学顾问，他表示：“通过脑电图 (EEG) 测量的关键神经生理学生物标志物的快速改善，加上认知指标的提升，支持了在脆性 X 综合征 (FXS) 中针对 BK 通道的可行性。”

I am eager to continue studying SPG601 in the next trial to assess the potential added benefits of chronic dosing and further validate these findings.'.

我渴望在接下来的试验中继续研究SPG601，以评估长期给药的潜在附加益处，并进一步验证这些发现。

SPG601 is on an expedited path to regulatory approval, having received Fast Track Designation and Orphan Drug Designation by the FDA, as well as Orphan Disease Designation by the EMA.

SPG601 正在通过快速通道获得监管批准，它已获得 FDA 的快速通道资格和孤儿药资格，以及 EMA 的孤儿病资格。

About SPG601

关于SPG601

SPG601 is an oral investigational medication being developed to treat FXS, and potentially other conditions on the autism spectrum, by mitigating key underlying abnormalities in synaptic function and neural excitability. FXS involves a reduction in the activity of large conductance, calcium-activated potassium (BK) channels, which contributes to synaptic dysfunction, cortical hyperexcitability, and multiple symptoms of FXS. SPG601, a novel small molecule, is the first positive modulator of BK channels to be clinically evaluated in FXS patients in a Phase 2a study and demonstrated the potential to improve cognitive, emotional, and sensory symptoms by boosting BK channel activity. SPG601 has received both Orphan Drug designation and Fast Track designation from the FDA for FXS, as well as orphan disease designation from the EMA.

SPG601 是一种正在开发中的口服研究性药物，用于治疗脆性X综合征（FXS）以及自闭症谱系中的其他潜在疾病，通过缓解突触功能和神经兴奋性的关键异常来发挥作用。FXS 涉及大电导钙激活钾（BK）通道活性的降低，这会导致突触功能障碍、皮层过度兴奋以及 FXS 的多种症状。SPG601 是一种新型小分子化合物，也是首个在2a期临床试验中针对FXS患者进行评估的 BK 通道正向调节剂，展示了通过增强 BK 通道活性改善认知、情绪和感官症状的潜力。SPG601 已获得 FDA 针对 FXS 的孤儿药资格和快速通道资格，同时也获得了 EMA 的孤儿药认定。

Plans for a registrational-directed Phase 2b/3 trial have been agreed to with the FDA. .

与FDA已就注册导向的2b/3期试验计划达成一致。

About Fragile X Syndrome

关于脆性X综合征

Fragile X Syndrome (FXS) is the leading inherited form of intellectual disability and a known cause of autism that results from the silencing of the Fmr1 gene. FXS is an orphan disease affecting approximately 1 in 4-5000 men and 1 in 6-8000 women globally. In addition to intellectual disability, FXS patients endure a wide range of disabling symptoms, including severe anxiety, social aversion, hyperactivity and attention deficit, sensory hypersensitivity, aggression, developmental seizures, and others.

脆性X综合征（FXS）是导致智力障碍的主要遗传形式，也是已知的由Fmr1基因沉默引发的自闭症病因。FXS是一种罕见病，全球大约每4000到5000名男性中有1人，每6000到8000名女性中有1人受到影响。除了智力障碍，FXS患者还承受着一系列致残症状，包括严重焦虑、社交回避、多动和注意力缺陷、感官过敏、攻击性行为、发育性癫痫等。

Providing care for individuals with FXS often becomes a full-time commitment for at least one parent and imposes significant financial strain, with direct family healthcare costs totaling $4.1 billion annually in the United States alone. Despite the considerable impact of FXS, there are currently no FDA-approved drugs available for those with the condition..

为患有FXS的个体提供照顾往往成为至少一位家长的全职任务，并带来巨大的经济压力，仅在美国，家庭直接医疗费用每年总计就达41亿美元。尽管FXS的影响相当大，但目前尚无FDA批准的药物可用于治疗该病症。

About Spinogenix

关于Spinogenix

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

目前，对神经退行性、神经精神和神经发育疾病的治疗主要集中在减缓疾病进展或减轻症状上，这让许多患者对改善病情失去了希望。Spinogenix 致力于通过其开创性的、同类首创且具有范式转变的突触再生和突触矫正疗法来改变这些疾病的治疗方式，旨在恢复耗竭的突触，逆转突触退化和功能障碍，为患者及其家庭带来充满希望的新前景。

Spinogenix is developing two novel therapeutics: Tazbentetol (formerly 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：Tazbentetol（之前称为SPG302），它能触发神经元生成新的谷氨酸能突触，并在ALS、阿尔茨海默病、精神分裂症及其他疾病中恢复认知、运动等功能；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 and EMA designations for ALS as well as FDA Orphan Drug and Fast Track designations for FXS. More information on Spinogenix can be found at .

该公司已获得FDA孤儿药和EMA对ALS的指定，以及FDA孤儿药和快速通道对FXS的指定。更多关于Spinogenix的信息可以在以下网站找到：。

www.spinogenix.com

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