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.初创企业

[+1]

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 相关的新闻（医药）

2024-08-01

·GlobeNewswire-Health Care

Spinogenix Announces Open Enrollment for Phase 2 Study Evaluating SPG302 for the Treatment of Alzheimer’s Disease

SPG302 is a once-a-day pill with the potential to regenerate synapses to reverse declines in cognitive function in people with Alzheimer’s diseaseLOS ANGELES, Aug. 01, 2024 (GLOBE NEWSWIRE) -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that enrollment is now open in Australia for their Phase 2 clinical trial evaluating SPG302 for the treatment of people with Alzheimer’s disease (AD), the most prevalent form of dementia worldwide. “The SPG302 trials are a completely different approach to tackling Alzheimer’s Disease,” stated Bruce Brew, MD, DSc, FRACP, FAAN, neurologist and trial Principal Investigator at St. Vincent’s Hospital in Sydney, Australia. “Unlike other approaches, SPG302 has the prospect for some degree of regeneration and consequent improvement in memory, instead of just slowing the disease. This is a refreshing, long-awaited and cutting-edge approach to the disease, if it’s successful, it could be the dawn of a new era in treating Alzheimer’s disease.” “There are currently no approved drugs for Alzheimer’s disease that focus on restoring function to reverse the course of disease by targeting the synapses. At Spinogenix, our mission is to offer new hope to people battling AD, not just by slowing the disease but by potentially reversing it, offering a significant departure from conventional approaches” said Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer and Founder. The Phase 2 trial will evaluate the safety, tolerability, clinical efficacy, pharmacokinetics, and pharmacodynamics of a once-a day pill of SPG302 in adult participants with mild-to-moderate Alzheimer’s disease. Enrollment is currently open at St. Vincents Hospital, Sydney, NSW, Australia with plans for a second site opening soon at Flinders Medical Center, Adelaide, SA, Australia. Additional information is available on ClinicalTrials.gov (NCT06427668). SPG302 is also being evaluated in Amyotrophic lateral sclerosis (ALS) patients in Australia, with plans to enroll ALS patients in the U.S. following recent FDA IND clearance. Additional information on the ALS trial may be found on ClinicalTrials.gov (NCT05882695). About SPG302SPG302 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 motor functions. 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 has been granted U.S. FDA Orphan Drug Designation for the treatment of ALS. Additional information on the clinical trials evaluating SPG302 can be found on ClinicalTrials.gov (NCT05882695 and NCT06427668). SPG302 has received preclinical support from the U.S. National Institutes of Health and the Department of Defense. About Alzheimer’s DiseaseAlzheimer’s Disease is the most common cause of dementia, accounting for ~60-70% of dementia cases worldwide. Loss of synapses occurs very early in Alzheimer’s and is a major driver of progressive impairments in cognition and memory. Currently, there is no cure for AD and medications that are approved for use in AD have only modest and temporary impact on symptoms. About SpinogenixSpinogenix is dedicated to developing transformative therapeutics for conditions involving the loss or dysfunction of synapses. Our lead clinical-stage synaptic regenerative drug, SPG302, is a first-in-class therapeutic designed to reverse synapse loss and improve cognitive and motor functions in neurodegenerative and neuropsychiatric diseases such as ALS, Alzheimer’s disease, and schizophrenia. In addition, we are developing a synaptic function therapeutic, SPG601, designed to improve cognitive and other symptoms in Fragile X Syndrome by correcting specific abnormalities in synaptic communication. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. Spinogenix Contact info@spinogenix.com Media Contact Kristin Politi, Ph.D.LifeSci Communicationskpoliti@lifescicomms.com(646) 876-4783

临床2期孤儿药

2024-07-23

·GlobeNewswire-Health Care

Spinogenix Announces Enrollment is Open for its Phase 2 Clinical Trial of SPG601 in Fragile X Syndrome

U.S. FDA has granted Orphan Drug Designation to SPG601 for the Treatment of Fragile X Syndrome SPG601 is a novel small molecule that works by correcting specific synaptic dysfunctions in the brain to address core symptoms of Fragile X Syndrome LOS ANGELES, July 23, 2024 (GLOBE NEWSWIRE) -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that enrollment is now open for their Phase 2 clinical trial evaluating SPG601 for the treatment of adult men with Fragile X Syndrome (FXS), the leading inherited form of intellectual disability and a known cause of autism. The U.S. Food and Drug Administration (FDA) recently cleared the Investigational New Drug (IND) application and granted Orphan Drug Designation for SPG601 for the treatment of people with FXS. "Fragile X patients and their families compose an incredible, yet still underserved community. That is why, during this year’s National Fragile X Awareness Month, we are especially excited to announce that we have begun recruiting for our Phase 2 trial evaluating SPG601 for the treatment of individuals with this difficult condition. This aligns with our commitment to advancing research and treatment options to help people with this condition live better-quality lives, as there are currently no FDA-approved drugs available for those with FXS," said Craig Erickson, M.D., Spinogenix Chief Medical Advisor. “Notably, SPG601 is a novel once-a-day pill that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions, a therapeutic approach that we believe can bring much needed hope to individuals and families struggling with FXS.” Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer and Founder, added, "People with FXS and their families face immense challenges every day. This milestone marks a significant step forward in our mission to bring new hope to the people and families affected by this condition. At Spinogenix, we are passionately working to develop first-in-class therapeutics that restore synapses to improve the lives of people worldwide and we are accomplishing this through our trials evaluating SPG601 in FXS and SPG302 in ALS and Alzheimer’s disease. We look forward to exploring the potential of our small molecule platform to address a range of diseases involving synapse loss and dysfunction." This study is a Phase 2 randomized, double blind, placebo-controlled, cross over, single dose of SPG601 and matching placebo in patients with Fragile X syndrome. Enrollment is currently open at the Cincinnati Children’s Hospital Medical Center in Cincinnati, Ohio, United States. Additional information on the Phase 2 trial is available on ClinicalTrials.gov (NCT06413537). FXS results from the silencing of the Fmr1 gene which leads to changes in mRNA and protein expression that results in abnormalities in synaptic function. Some of these abnormalities have been shown to be a result of a reduction in the amount and activity of large-conductance, calcium-activated potassium (“BK”) channels. Many core symptoms of FXS have been linked to deficient activity of BK channels. In addition to FXS, BK channel expression and activity have been linked to a range of other conditions including neurodegenerative, vascular, and sensory disorders. SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to correct specific synaptic dysfunctions that underlie these conditions. About Fragile X SyndromeFragile 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. 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. About Spinogenix Spinogenix is dedicated to developing transformative therapeutics for conditions involving the loss or dysfunction of synapses. Our lead clinical-stage synaptic regenerative candidate is a first-in-class therapeutic designed to reverse synapse loss and improve cognitive and motor functions in neurodegenerative and neuropsychiatric diseases such as ALS, Alzheimer’s disease, and schizophrenia. In parallel, we are also developing a synaptic function therapeutic designed to improve behavior in Fragile X Syndrome. More information on Spinogenix can be found at www.spinogenix.com or follow us on LinkedIn. Spinogenix Contact info@spinogenix.com Media Contact Kristin Politi, Ph.D.LifeSci Communicationskpoliti@lifescicomms.com(646) 876-4783

临床2期孤儿药临床申请

2024-05-30

·药渡Daily

【药渡药闻报告-创新药篇】-2024年第20期/总第141期

全球药物批准/研发动态01全球新药批准情况根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）全球（不含中国）共有5个新药获批上市。其中，BLA批准5个。与上个统计周期相比，本次增加1个批准新药。5月21日，Biocon Biologics宣布FDA已批准Yesafili™（阿柏西普-jbvf）的首次申请。Yesafili™用于治疗新生血管性（湿性AMD）老年性黄斑变性、视网膜静脉闭塞（分支性RVO或中心性RVO）继发黄斑水肿引起的视力损害、糖尿病性黄斑水肿（DME）引起的视力损害以及近视性脉络膜新生血管（近视性CNV）引起的视力损害。数据显示，Yesafili™的质量、安全性和疗效与Eylea®相当。5月23日，甘李药业近日以MAH身份取得了墨西哥COFEPRIS下发的甘精胰岛素注射液预填充笔的注册批件。此次获批标志着甘李药业正式获得向墨西哥销售甘精胰岛素注射液预填充笔的商业化许可，也是甘李药业首次以MAH的身份进入墨西哥市场。在墨西哥境内，甘李的甘精胰岛素产品的获批，将导入质优价惠的胰岛素药品资源，丰富当地糖尿病患者的治疗选择。全球（不含中国）新药批准情况（部分）02全球新药申报进展根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）全球（不含中国）共有3个新药申报上市。其中，NDA申报进展1个，BLA申报进展1个,细胞疗法1个。与上次统计周期相比，本次减少1个NDA/BLA申报。5月24日，复宏汉霖与Organon共同宣布在研地舒单抗（Prolia®/Xgeva®）生物类似药HLX14的上市许可申请获EMA受理。此次受理是基于III期临床试验，旨在比较HLX14与欧盟市售原研地舒单抗（Prolia®）在高危骨折风险的绝经后骨质疏松症女性受试者中的有效性、安全性、耐受性和免疫原性。NDA/BLA申报根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）全球（不含中国）共有8个药物获监管机构特殊资格认定。其中，化学药5个，生物药2个，细胞疗法1个。与上次统计周期相比，本次增加2个获监管机构特殊资格认定的药物。5月20日，Spinogenix宣布FDA已授予SPG601孤儿药资格（ODD），用于治疗脆性X综合征（FXS）。FXS是智力障碍的主要遗传形式，也是Fmr1基因沉默导致的自闭症的已知原因，FXS的许多核心症状与大电导BK通道的活性不足有关。SPG601是一种新型小分子BK通道激活剂，通过与BK通道结合并激活BK通道来恢复突触功能。5月21日，中美瑞康宣布其自主研发的saRNA药物RAG-01获得FDA快速通道认定，用于治疗卡介苗无应答的非肌层浸润性膀胱癌。此前，RAG-01用于治疗非肌层浸润性膀胱癌的临床试验申请已获得FDA的许可，同时，RAG-01已在澳大利亚启动I期临床试验，目前已有3例受试者入组接受给药。RAG-01是全球首个获得FDA快速通道认定的saRNA项目。特殊资格认定情况（部分）03全球新药研发进展根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）全球（不含中国）新药临床研发状态更新共计53条，涉及肿瘤、血液和淋巴疾病、遗传代谢病、精神疾病、神经疾病以及皮肤病等共计13个领域。其中，肿瘤临床进展更新居各领域之首，为26条：肿瘤领域涉及化学药8条，生物药13条，细胞疗法3条，疫苗2条。5月22日，再鼎医药宣布，首位患者已在一项全球II期临床研究（NCT06380907）中给药，该研究旨在评估IL-17抑制剂ZL-1102治疗慢性斑块状银屑病（CPP）的疗效和安全性。ZL-1102是一款靶向作用于IL-17A细胞因子的新型全人源VH抗体片段（Humabody®），具有高亲和力，被开发用于治疗轻中度慢性斑块状银屑病。5月23日，迈威公布将于2024年ASCO年会上，以口头报告形式展示的靶向Nectin-4 ADC创新药 (9MW2821) I/II期临床研究数据及最新进展，包括尿路上皮癌、宫颈癌、食管癌、三阴性乳腺癌等多种晚期实体瘤。9MW2821注射体内后，可与肿瘤细胞表面的 Nectin-4结合并进入细胞，通过酶解作用，定向释放细胞毒素，实现对肿瘤的精准杀伤。全球新药研发进展详情（部分）04全球医药交易事件本次统计周期（2024.05.18-05.24）全球（含中国）医药交易时间共计40起，涉及药物权益转让、公司并购等多起交易事件。全球医药交易时间汇总表（部分）国内药物批准/研发动态01国内新药批准情况根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内共有11个新药获NMPA批准上市。其中，NDA批准3个，BLA批准1个，新复方批准2个，新适应症批准5个，拒绝批准1个。与上次统计周期相比，本次增加10个NMPA批准新药，拒接批准1个NMPA批准新药。5月21日，NMPA批准恒瑞医药公司研发的1类新药、PARP抑制剂氟唑帕利胶囊新适应症上市，用于晚期上皮性卵巢癌、输卵管癌或原发性腹膜癌成人患者在一线含铂化疗达到完全缓解或部分缓解后的维持治疗。研究结果表明，氟唑帕利单药维持治疗能够显著延长晚期卵巢癌患者的PFS，且无论是否伴有gBRCA1/2突变，患者均能从氟唑帕利单药的维持治疗中获益。5月24日，NMPA官网公示，康方生物独立自主研发的全球首创的PD-1/VEGF双特异性抗体新药依达方®（通用名：依沃西单抗注射液）正式获批上市，适应症为联合化疗治疗经表皮生长因子受体（EGFR）酪氨酸激酶抑制剂（TKI）治疗后进展的EGFR突变的局部晚期或转移性非鳞状非小细胞肺癌（nsq-NSCLC）。依达方®是全球第一个获批上市的“肿瘤免疫+抗血管生成”机制的双特异性抗体新药。中国新药批准情况（部分）02国内新药临床默示许可进展根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内共有34个新药获临床默示许可，涉及51个受理号。其中，化学药15个，治疗用生物制品19个。与上个统计周期相比，本次减少1个临床默示许可获批受理号。本周国内新药临床默示许可进展（部分）03国内新药申报进展根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内共有6个新药申报上市，涉及8个受理号。其中，化学药4个，治疗用生物制品2个。与上个统计周期相比，本次新药申报上市受理号与上个统计周期相同。国内新药申报上市情况（部分）根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内共有34个新药申报临床，涉及41个受理号。其中，化学药15个，治疗用生物制品17个，预防用生物制品2个。与上个统计周期相比，本次减少52个临床申报受理号。国内新药临床申报情况（部分）根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内共有4个药物获NMPA特殊资格认定。其中，化学药1个，生物药3个。与上个统计周期相比，本次减少2个获监管机构特殊资格认定的药物。NMPA特殊资格认定情况（部分）04国内新药研发进展根据药渡数据统计分析，本次统计周期（2024.05.18-05.24）国内新药临床研发状态更新共计10条，涉及肿瘤、血液和淋巴疾病、神经疾病共计3个领域。其中，化学药6个，生物药4个。5月24日亚盛医药宣布，公司入选2024年ASCO年会的临床研究的摘要已于ASCO官网公布，涉及中国首个获批上市的第三代BCR-ABL抑制剂奥雷巴替尼（HQP1351）、Bcl-2选择性抑制剂APG-2575和FAK/ALK/ROS1三联抑制剂APG-2449三个重点品种。奥雷巴替尼（Olverembatinib）已在中国获批用于治疗慢性髓细胞白血病患者，其耐受性良好，在SDH缺陷GIST患者中的CBR超90%，且显著延长预计的中位PFS。APG-2575联合阿扎胞苷（AZA）在成年AML患者中的安全性和有效性数据显示了其在TLS方面的安全性，且中性粒细胞减少伴发热发生率较低，并有较低的早期死亡率。Bcl-2抑制剂APG-2575（Lisaftoclax）单药或与伊布替尼或利妥昔单抗联合治疗华氏巨球蛋白血症（WM）的耐受性良好，且在初治或BTKi治疗失败的WM患者中疗效显著。国内新药研发进展情况05国内新药研发领域政策法规动态国家药监局关于适用《Q5A（R2）：来源于人或动物细胞系生物技术产品的病毒安全性评价》国际人用药品注册技术协调会指导原则的公告（2024年第63号）为推动药品注册技术标准与国际接轨，经研究，国家药品监督管理局决定适用《Q5A（R2）：来源于人或动物细胞系生物技术产品的病毒安全性评价》国际人用药品注册技术协调会指导原则（以下简称Q5A（R2）指导原则）。现就有关事项公告如下：一、申请人需在现行药学研究技术要求基础上，按照Q5A（R2）指导原则的要求开展研究；自2025年5月21日开始的相关研究（以试验记录时间点为准），均适用Q5A（R2）指导原则。二、相关技术指导原则可在国家药品监督管理局药品审评中心网站查询。国家药品监督管理局药品审评中心负责做好本公告实施过程中的相关技术指导工作。06国内新药研发领域热点新闻人用狂犬病疫苗竞争加剧：成大生物艰难守擂，康华生物喜提劲敌近日，河南新野一三岁儿童被狗咬伤注射4剂狂犬疫苗后仍因狂犬病发展死亡的事件在网上传的沸沸扬扬，这也引发了民众对狂犬疫苗质量的质疑。关于狂犬病狂犬病是狂犬病病毒感染引起的以中枢神经系统症状为主的一种动物源性传染病，临床表现为特有的恐水、怕风、咽肌痉挛、进行性瘫痪等，一旦出现症状，病死率接近100%。据WHO统计，全球超150个国家和地区存在狂犬病流行，全球每年约有5.9万人因狂犬病死亡。更多资讯，请阅读原文亚盛医药不走寻常路，以特色Biotech赢得市场？一年一度的全球肿瘤行业盛会ASCO年会即将召开。此次亚盛医药有3个重点品种的4项临床研究入选2024 ASCO年会，其中1项获口头报告，为中国首个且唯一获批上市的第三代BCR-ABL抑制剂耐立克，治疗SDH缺陷型胃肠道间质瘤和副神经节瘤患者的最新疗效数据展示。其余2款重点产品的3项临床研究为壁报展示，包括Bcl-2抑制剂APG-2575（Lisaftoclax）、FAK/ALK/ROS1三联抑制剂APG-2449。更多资讯，请阅读原文98款在研产品，中药、生物药、化学药，天士力可以“全都要”吗？一直以来，中药企业都被认为是重营销轻研发。2023年，A股73家中药上市企业研发费用合计102.1亿元，平均研发费用率仅为4.8%；同期销售费用合计873.7亿元，是研发费用的8倍多。然而，作为中药企业龙头，天士力近年来一直走在中药创新的前列。年研发费用从2021年的5.8亿元增长至2023年的9.2亿元，研发费用率10.57%，远超中药行业平均水平。高额研发投入，让天士力拥有了98款在研产品，中药、生物药、化学药“三管齐下”，产品涵盖心脑血管、消化代谢、肿瘤三大疾病领域。广撒网之下，天士力真的可以“全都要”吗？更多资讯，请阅读原文国内第五款三代EGFR抑制剂获批上市5月20日，根据国家药监局官网公示，上海倍而达药业有限公司申报的1类创新药三代EGFR抑制剂甲磺酸瑞齐替尼胶囊（研发代码BPI-7711，商品名：瑞必达）获批上市，适应症为既往经表皮生长因子受体（EGFR）酪氨酸激酶抑制剂（TKI）治疗时或治疗后出现疾病进展，并且经检测确认存在EGFR T790M突变阳性的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者的治疗。从药渡数据库检索可知，瑞齐替尼是国内第五款获批上市的三代EGFR抑制剂，2021年3月9日，石药集团宣布，其全资附属公司石药（上海）有限公司已与上海倍而达药业订立产品授权及商业化协议，通过股权认购获得第三代EGFR-TKI（BPI-7711胶囊）独家产品授权及商业化权利。更多资讯，请阅读原文恒瑞Trop2 ADC创新药SHR-A1921拟纳入突破性疗法5月21日，CDE官网公示可知，恒瑞医药自主研发的Trop2 ADC新药SHR-A1921拟纳入突破性治疗，适应症为铂耐药复发上皮性卵巢癌、输卵管癌或原发性腹膜癌。今年3月，SHR-A1921获得美国食品和药物监督管理局（FDA）授予快速通道资格（Fast Track Designation，FTD）。更多资讯，请阅读原文咨询、合作请联系作者小D有话说为方便读者阅读及保存，我们将周报原文整理成了PDF版本，如需获取全文，可点击最上方蓝字，在药渡Daily公众号后台回复“0529创新药周报”，即可下载。

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