A Phase 2a, Randomized, Double-Blinded, Study Evaluating the Neurophysiologic vs Clinical Effects of Single-Dose SPG601 and Placebo in Adult Men With Fragile X Syndrome

This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.

开始日期2024-07-05

申办/合作机构

Spinogenix, Inc.

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100 项与 SPG-601 相关的临床结果

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100 项与 SPG-601 相关的转化医学

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100 项与 SPG-601 相关的专利（医药）

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项与 SPG-601 相关的文献（医药）

Scientific Reports

SPG601-associated modulation of resting-state EEG and improvement in executive function in a fragile X syndrome randomized controlled crossover study

Article

作者: Richter, Sarah ; Nelson, Meredith ; Westerkamp, Grace ; Vanderklish, Peter W ; Erickson, Craig A ; Gargosky, Sharron ; Sarraf, Stella T ; Pedapati, Ernest V

Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and autism spectrum disorder. Despite extensive research, no targeted treatments exist for the core symptoms of FXS. SPG601 represents the first BK channel activator to enter clinical testing for FXS, designed to restore synaptic function by correcting specific ion channel dysfunction downstream of fragile X messenger ribonucleoprotein protein (FMRP) loss. We conducted a randomized, double-blind, placebo-controlled, 2-period balanced crossover study in 10 adult men with genetically confirmed full-mutation fragile X syndrome. Participants received single doses of SPG601 800 mg and placebo separated by a 1-week washout period. Given the first in FXS nature of the study, the safety and tolerability of SPG601 were evaluated as a primary outcome. Additional endpoints included resting-state EEG power spectral density analysis across predefined frequency bands and auditory-evoked gamma oscillations combined with cognitive assessments using validated instruments and clinical global impressions scales. Among EEG measurements, excessive high frequency gamma band activity has been most extensively validated as a biomarker across species in FXS and has demonstrated correlation with severity of the human FXS phenotype and therefore served as the primary neurophysiologic endpoint. SPG601 was well tolerated with a favorable safety profile. SPG601 demonstrated significant modulation of resting-state EEG power spectral density compared to placebo. Significant treatment effects were observed for gamma power (F = 5.20, p = 0.023), alpha2 power (F = 17.43, p < 0.001), and theta power (F = 7.06, p = 0.008). SPG601 also significantly modulated aperiodic EEG slope (F = 5.28, p = 0.022), indicating alterations in the broadband 1/f component reflecting excitation-inhibition balance. Cognitive improvement was observed in the NIH Toolbox Flanker Inhibitory Control and Attention Test across multiple scoring metrics (p = 0.027). No significant effects were observed on auditory-evoked gamma measures. This study provides the first clinical evidence that SPG601 produces significant neurophysiological changes in FXS, accompanied by a cognitive enhancement in executive function.

项与 SPG-601 相关的新闻（医药）

2026-07-16

·PRNewswire

Spinogenix Announces World Health Organization Approval of "Codabakalner" as International Nonproprietary Name for SPG601

Codabakalner is a First-in-Class Investigational Therapy Entering Phase 2b/3 Development for Fragile X Syndrome LOS ANGELES, July 16, 2026 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering novel therapeutics that have the potential to restore synapses to improve the lives of patients worldwide, today announced that the International Nonproprietary Names (INN) Expert Committee of the World Health Organization has approved "codabakalner " as the non-proprietary name for the Company's investigational therapy, SPG601. Codabakalner is a first-in-class, oral investigational therapy being developed for the treatment of Fragile X syndrome (FXS), the leading inherited cause of intellectual disability and a known genetic cause of autism. The therapy is designed to positively modulate large-conductance calcium-activated potassium (BK) channels to correct specific synaptic dysfunctions that underlie many of the core cognitive, behavioral, and sensory symptoms associated with FXS. "Approval of the non-proprietary name codabakalner marks another important milestone in the advancement of our Fragile X syndrome program," said Dr. Stella Sarraf, CEO and Founder of Spinogenix. "As we prepare for the CLARITY Phase 2b/3 trial, establishing a globally recognized non-proprietary name reflects the maturity of the program and our commitment to bringing this first-in-class therapy to patients living with Fragile X syndrome. We are focused on advancing codabakalner as a potential treatment capable of addressing the underlying synaptic dysfunction that drives many of the core symptoms of this devastating condition." Spinogenix is a proud sponsor of this year's NFXF International Fragile X Conference in Louisville, KY, where Chief Medical Advisor, Craig Erickson, MD, will be presenting to share the new INN, codabakalner. Dr. Erickson will also share updates regarding CLARITY, Spinogenix's Phase 2b/3 trial evaluating the efficacy, safety, and tolerability of codabakalner in male patients with FXS. Commonly referred to as a generic name, each International Nonproprietary Name (INN) is a unique name assigned by the World Health Organization to identify pharmaceutical substances and active pharmaceutical ingredients. A globally recognized non-proprietary name facilitates the clear identification, safe prescribing, and consistent communication of medicines throughout their development and eventual clinical use. About Codabakalner Codabakalner (SPG601) is an oral investigational medication being developed to treat FXS 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. Codabakalner, 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. Codabakalner has received both Orphan Drug designation and Fast Track designation from the FDA for FXS, as well as orphan medicinal product designation from the EMA. Plans for the CLARITY registrational-directed Phase 2b/3 trial have been agreed to with the FDA. About 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. Spinogenix is developing two novel therapeutics: Tazbentetol (SPG302), which is designed to trigger neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and codabakalner (SPG601), which is designed to work at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA and EMA Orphan Drug Designations for ALS as well as FDA Orphan Drug and Fast Track designations for FXS. More information on Spinogenix can be found at or follow us on LinkedIn. Media Contact Daniel Davis FINN Partners for Spinogenix [email protected] Investor Relations Sasha Damouni Ellis Spinogenix, Inc. [email protected] SOURCE Spinogenix 21% more press release views with Request a Demo

孤儿药临床2期快速通道临床结果

2026-07-02

·PRNewswire

Spinogenix Appoints Professor Joseph T. Coyle, MD, Current Chair of Psychiatry and Neuroscience at Harvard Medical School, to Scientific Advisory Board

Prof. Coyle to Provide Guidance on Company's Neuroscience Pipeline, with Emphasis on Schizophrenia LOS ANGELES, July 2, 2026 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering novel therapeutics that have the potential to restore synapses to improve the lives of patients worldwide, today announced the appointment of Prof. Joseph T. Coyle, MD, a globally recognized expert in psychiatry and neuroscience, to its Scientific Advisory Board (SAB). The appointment comes as Spinogenix advances the clinical development of tazbentetol (formerly SPG302) across multiple neurodegenerative and neuropsychiatric indications. Prof. Coyle, the Eben S. Draper Chair of Psychiatry and Neuroscience at Harvard Medical School, will provide strategic scientific guidance across the company's neuroscience pipeline, with a particular focus on schizophrenia. "We are thrilled to welcome Prof. Coyle to Spinogenix's Scientific Advisory Board at this important stage in the development of tazbentetol," said Dr. Stella Sarraf, CEO and Founder of Spinogenix. "Prof. Coyle's pioneering work in neuropsychiatric research has shaped the current understanding of schizophrenia and other brain disorders. His expertise, particularly in advancing EEG biomarkers and translating neuroscience discoveries into clinical development, will be instrumental as we advance our schizophrenia program. Prof. Coyle has spent more than four decades advancing the understanding of brain function and neuropsychiatric disease. An established expert in developmental neurobiology, glutamatergic signaling, neuronal vulnerability, and psychopharmacology, he has authored more than 500 scientific publications and is among the most highly cited researchers in neuroscience. "Schizophrenia remains a highly complex neuropsychiatric disorder, highlighting the need for therapies that address its underlying biology," said Prof. Coyle. "I have been particularly impressed by Spinogenix's novel synaptic regenerative approach to treating neurological conditions, as well as the company's commitment to integrating EEG into the clinical development of tazbentetol. I look forward to supporting the team as it continues developing this essential program." Throughout his distinguished career, Dr. Coyle has held numerous leadership positions, including Chairman of the Department of Psychiatry at Harvard Medical School, President of the Society for Neuroscience, and President of the American College of Neuropsychopharmacology. He is a member of the National Academy of Medicine, a Fellow of the American Academy of Arts and Sciences, and has served on numerous national advisory committees and editorial boards. His research has been continuously funded by the National Institutes of Health (NIH) for over 30 years and has been cited more than 87,000 times. About 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. Spinogenix is developing two novel therapeutics: Tazbentetol (SPG302), which is designed to trigger 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 is designed to work at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received Orphan Drug designations from FDA and EMA for tazbentetol in ALS as well as FDA Orphan Drug and Fast Track designations for SPG601 in FXS. More information on Spinogenix can be found at or follow us on LinkedIn. Media Contact: Daniel Davis FINN Partners [email protected] Investor Relations Contact: Sasha Damouni Ellis Spinogenix, Inc. [email protected] SOURCE Spinogenix 21% more press release views with Request a Demo

孤儿药高管变更

2026-06-26

·今日头条

智力障碍治疗有新突破了？李医生跟你说说这几个好消息🧠

大家好，我是四会慈航精康医院的李定国。门诊上经常有家长带着孩子来，一坐下就问：“李医生，我家孩子发育比别人慢、学东西吃力，是不是智力障碍？这病到底有没有办法？”以前我回答得比较谨慎，但这两年，我真的能说一句：有盼头了，而且路越走越宽了。今天不聊虚的，跟大家说几个实实在在的好消息。第一个好消息：基因治疗，从根上找办法智力障碍很大一部分跟基因突变有关。这两年基因治疗进展特别快—— 艾伦研究所的科学家设计了一种新基因疗法，在动物实验中成功逆转了SYNGAP1相关障碍的症状，包括智力障碍、癫痫和多动行为。虽然还处于临床前阶段，但这条路走通了。国内也有突破。中国科学院昆明动物研究所 Mao 团队发现，一种叫 ZC4H2 的蛋白质是调控认知功能的关键。更让人振奋的是，一种已经上市的抗癫痫药，在小鼠实验中成功恢复了认知功能，包括识别记忆、社交记忆和空间学习能力。老药新用，省去了漫长的研发周期。第二个好消息：脆性X综合征，多个新药在路上脆性X综合征是导致智力障碍和自闭症最常见的单基因遗传病，大约每2000个男孩里就有一个。加州大学洛杉矶分校的研究团队发现了一个叫 EPAC2 的新靶点，在小鼠实验中阻断它之后，改善了社交障碍、感觉过敏和癫痫易感性。与此同时， Spinogenix公司的SPG601 已经完成二期临床试验，显著降低了患者高频伽马脑电活动 ——这是学习与记忆受损的关键生物标志物。 Servier公司也以4.5亿美元收购了一款口服小分子药物 KER-0193 ，今年初已完成一期临床试验，安全性和耐受性良好。第三个好消息：基因编辑，CRISPR技术大显身手 CRISPR激活技术被用于治疗SYNGAP1相关的智力障碍，在动物模型中成功恢复了工作记忆缺陷。辉大基因的CRISPR基因编辑疗法，通过直接脑内注射在罕见病患儿中开展了临床治疗，获得了积极结果。更前沿的是，加州大学戴维斯分校的团队正在开发子宫内基因编辑疗法，针对Dup15q综合征——一种与严重智力障碍、自闭症和癫痫相关的疾病。如果成功，孩子还没出生就可能被治愈。第四个好消息：非侵入性治疗，不用开刀不用吃药新华医院李斐教授团队原创的加速连续θ波脉冲刺激疗法，全程无创无痛，单次治疗仅需2到3分钟，全疗程仅5天。研究纳入了近半数智商低于70的低功能患儿，结果证实无论是否合并智力障碍，社交沟通能力和语言表达能力均得到显著改善。此外，基于脑电图的生物电刺激也被证实能协同改善智力障碍儿童的认知功能、行为症状和生活质量。最后李医生说句掏心窝的话—— 智力障碍的病因非常复杂，目前还不能说“根治” ，但 “没办法”这三个字已经站不住脚了。从基因治疗到新药研发，从老药新用到非侵入性脑刺激，多条战线同时推进，选择越来越多。如果你发现孩子发育明显落后于同龄人，别拖，早点去医院做评估。早发现、早干预，效果最好。每个孩子都值得被看见、被理解、被科学地帮助。这条路，咱们一起慢慢走。 ——李定国，在四会慈航精康医院，陪你一起走。

100 项与 SPG-601 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
脆性X综合征	临床2期	美国	Spinogenix, Inc.	2024-07-05

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06413537 (CTgov)	临床2期	脆性X综合征	10	SPG601 (Experimental: Active SPG601 to be Administered to Participants With Fragile X Syndrome)	窪簾糧壓簾繭鑰艱膚願(夢顧襯構鏇糧壓獵觸蓋) = 觸鬱鹹鬱齋遞襯膚艱憲遞襯壓膚範繭觸膚範醖 (獵鹹淵壓齋廠齋襯衊衊, 鬱壓願廠膚鏇蓋夢製構 ~ 簾淵壓觸繭築繭鹽鹽製) 更多	-	2026-05-26
				Placebo (Placebo Comparator: Placebo Comparator to be Administered to Participants With Fragile X Syndrome)	窪簾糧壓簾繭鑰艱膚願(夢顧襯構鏇糧壓獵觸蓋) = 構襯壓廠鬱積範網餘築遞襯壓膚範繭觸膚範醖 (獵鹹淵壓齋廠齋襯衊衊, 獵夢壓範窪膚糧獵鏇廠 ~ 網襯艱窪構壓顧積膚鹽) 更多