VICO Therapeutics BV

First clinical data of VO659 demonstrated 28% mean reduction in CSF mutant HTT at day 85 in HD patients in 40 mg dose cohort with an effect after the first dose No sustained increase in CSF neurofilament light chain (Nf-L) protein observed through day 85 VO659 is generally safe and well tolerated with an anticipated dosing regimen of 1-2 times per year in future studies Data highlighted in oral presentation at EHDN & Enroll-HD 2024 LEIDEN, Netherlands, Sept. 13, 2024 /PRNewswire/ -- Vico Therapeutics B.V., a clinical-stage genetic medicines company developing therapies for severe neurological diseases, today announced new positive interim data in Huntington's disease (HD) from the ongoing Phase 1/2a clinical trial of VO659, an investigational allele-preferential antisense oligonucleotide (ASO) therapy. Results will be highlighted in an oral presentation at the European Huntington's Disease Network's EHDN & Enroll-HD 2024 meeting on Friday, September 13, 2024 at 9:45 a.m. CET. This is the first clinical data reported of VO659, which is designed to target the CAG repeat expansion that causes all nine known polyglutamine diseases, including HD as well as spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). VO659 has a unique allele-preferential mechanism of action that targets expanded CAG repeats in the mutant mRNA transcript and inhibits mRNA translation leading to a reduction of mutant protein, with the potential to halt or slow disease progression. VO659 is the only clinical-stage program that directly targets the underlying CAG repeat expansion that causes HD. The Phase 1/2a clinical trial is a multi-center, open-label, multiple ascending dose study designed to assess the safety and tolerability of four doses of VO659 administered intrathecally every four weeks in participants with early manifest HD, or mild to moderate SCA1 or SCA3. Exploratory endpoints include the assessment of pharmacodynamic (PD) biomarkers (mHTT, mATXN3, and Nf-L protein) in cerebrospinal fluid (CSF), pharmacokinetics in CSF and plasma, and clinical outcome measures. The interim analysis provides a look at available safety, tolerability and PD data for HD participants in the 40 mg cohort through at least day 85 of the study. The Phase 1/2a trial interim data presented at the EHDN meeting demonstrated: A mean reduction of 28% in CSF mutant huntingtin protein (mHTT) in treated HD participants at day 85 compared to baseline with an immediate effect observed after the first dose at day 29 No sustained increase or decrease in Nf-L protein in CSF across treated participants at day 85 compared to baseline VO659 appears generally safe and well tolerated at the 40 mg dose level in treated HD patients through the available clinical follow-up period VO659 has a long half-life, indicating the potential for infrequent dosing estimated at 1-2 times per year "We are very pleased to announce these first data from our Phase 1/2a clinical trial of VO659 in participants living with HD," said Micah Mackison, chief executive officer at Vico. "It is highly encouraging that we saw immediate reductions in CSF mHTT and no changes in Nf-L protein, two key biomarkers in HD, in treated patients over the available follow-up period. Given VO659's long anticipated half-life, there is clear potential for this therapy to have an infrequent dosing schedule, and we look forward to exploring this further in clinical trials. These data are a significant milestone for our team and for the HD community that so desperately needs treatment options, and we look forward to continuing assessments from the trial and advancing VO659 as quickly as possible for patients and their families." These interim data build on preclinical data of VO659 demonstrating potent target engagement across multiple disease models. Preclinical research showed significant and dose-dependent reductions of mHTT and improvement in motor function in vivo in disease mouse models of HD and allele-preferential reductions of mHTT in vitro in HD patient cell models. "VO659's allele-preferential action and direct targeting of the CAG repeat expansion that causes HD is a unique approach to treating this disease with a promising future for the HD community," said Scott Schobel, M.D., chief medical officer at Vico. "The Phase 1/2a interim clinical data reinforce the potent, targeted nature of VO659 and add to a growing body of evidence supporting VO659's advancement in the clinic for HD as well as its broad potential applications for all CAG repeat expansion diseases." The Phase 1/2a trial remains ongoing and Vico plans to meet with regulators later this year to discuss the optimal path forward for the VO659 program. The company plans to provide an update from the SCA1 and SCA3 patient cohorts at a future scientific meeting. About Huntington's Disease and VO659 Huntington's disease (HD) is a rare genetic, progressive neurodegenerative disorder characterized by movement disorders, cognitive impairment, and psychiatric manifestations including depression and psychosis. The disease varies in terms of age of onset, typically starting in mid-adulthood, and it is ultimately fatal. The disease is caused by a CAG trinucleotide repeat expansion in the coding region of exon 1 of the huntingtin (HTT) gene, which results in production of mutant HTT protein (mHTT) with an elongated polyglutamine (polyQ) stretch at its N-terminus. This expanded polyQ stretch confers a toxic gain-of-function to mutant protein forms, ultimately resulting in widespread neuronal death. Currently available therapies for HD focus on alleviating some of the movement and psychiatric symptoms, with a significant unmet need for disease-modifying therapies. VO659 is designed to suppress mHTT in an allele-preferential manner and slow or halt disease progression. VO659 was granted both FDA and EMA Orphan Drug designation for the treatment of HD. About Vico Therapeutics B.V. Vico Therapeutics is a clinical-stage genetic medicines company developing antisense oligonucleotide (ASO) RNA modulating therapies for patients with severe neurological diseases. Our lead product candidate, VO659, is currently in Phase 1/2a clinical development for the treatment of spinocerebellar ataxia types 1 and 3 and Huntington's disease and is the only ASO in the clinic that targets the underlying CAG repeat expansions that cause all nine known polyglutamine diseases. Vico's research platform designs fit-for-purpose ASOs by applying precision chemistry with mechanisms of action ideally suited to target genetic neurological diseases. For more information, visit . Media Contact Jenna Urban Berry & Company Public Relations [email protected] 212-253-8881 SOURCE Vico Therapeutics B.V. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Plus, news about ONL Therapeutics, Cidara Therapeutics, Emergent BioSolutions, Vico Therapeutics and Centessa: Valneva gets €61.2M private placement: Valneva said the funds will go toward supporting ongoing Phase 3 and Phase 4 studies of its chikungunya vaccine and commercialization of that shot. It will also use the money to help fund planned Phase 2 trials for its Shigella and Zika vaccine candidates. Immuneering’s small Phase 2a dataset: The company said two of five first-line pancreatic cancer patients responded to its experimental drug, called IMM-1-104, which is designed to inhibit the MAPK pathway and “achieve universal-RAS activity.” One patient had a complete response and another had an unconfirmed partial response. Its stock $IMRX was up about 55% on Friday morning. ONL Therapeutics closes $65M Series D: Johnson & Johnson Innovation led the round . The funds will be used to advance the company’s ONL1204 program, a small molecule Fas inhibitor that aims to protect retinal cells across different eye diseases like macula-off retinal detachment and geographic atrophy. Cidara Therapeutics restructures, lays off 30% of workforce: The biotech took the measures after J&J returned a flu drug earlier this year . Cidara raised $240 million in a PIPE financing in conjunction with the move in April. The reprioritization, announced Thursday, will see the company solely focus on the flu drug, called CD388, and seek partners for its oncology pipeline. Cidara had 69 employees as of April. Emergent BioSolutions gets BARDA award for Ebola, settles case: The company will get $41.9 million to scale up its commercial efforts for Ebanga, its Ebola drug. Separately, Emergent said Friday it had agreed to pay $40 million — mostly from its insurance — to settle a class action lawsuit brought in 2021. Emergent’s shares $EBS were up about 6% on Friday morning. Vico Therapeutics presents interim Phase 1/2a Huntington’s data: The private Dutch biotech said six patients saw a 28% decrease, on average, in CSF mutant huntingtin protein after 12 weeks. There was also no effect on neurofilament light chain. Vico raised a $60 million Series B in January. Centessa Pharmaceuticals upsized its offering to $225 million from $150 million.

▎药明康德内容团队编辑寡核苷酸药物因其候选靶点丰富、研发周期短、药效持久、特异性强、临床开发成功率高等优势，成为医药企业重点开发领域，获得众多投资者青睐。据不完全统计，2024年至少已有10家寡核苷酸药物开发公司完成了新一轮融资。值得关注的亮点如下：超半数公司融资金额逾亿元: 在10起融资事件中，有6起的融资规模超过亿元。疾病开发领域多元化：主要开发疾病领域为心血管、传染病；中枢神经系统疾病及代谢疾病（如肥胖）等为创新开发疾病领域方向。肝脏外靶点是创新重点：靶向肝脏外其他组织，如胎盘、肌肉、皮肤等靶点的寡核苷酸疗法公司受到业界关注，如Comanche Biopharma、Dyne Therapeutics、安龙生物等。欧洲融资回升：获得资金支持的寡核苷酸药物开发公司中，有3家位于欧洲，专注于反义寡核苷酸（ASO）等疗法的开发。Dyne完成总额达3.451亿美元公开募股1月11日，Dyne Therapeutics宣布其在研抗体偶联反义寡核苷酸（ASO）药物DYNE-101与抗体偶联寡聚物DYNE-251分别在1/2期临床试验中获得积极数据。Dyne并宣布完成总额达3.451亿美元的公开募股。Dyne Therapeutics是一家临床阶段的肌肉疾病公司。凭借其专有的FORCE平台，Dyne正在开发创新寡核苷酸治疗药物，旨在克服递送至肌肉组织的局限性。Dyne具有广泛、用于治疗严重肌肉疾病的管线，包括强直性肌营养不良1型（DM1）和杜氏肌营养不良（DMD）的临床项目和面肩肱型肌营养不良症（FSHD）的临床前项目。DYNE-101由与片段抗体（Fab）偶联的反义寡核苷酸组成，Fab与肌肉上高表达的转铁蛋白受体1（TfR1）结合。Dyne近期公布的数据显示，在1/2期ACHIEVE试验中，DYNE-101在DM1患者中表现出剂量依赖性的剪接矫正、肌肉递送和DMPK敲除。Comanche Biopharma完成7500万美元B轮融资Comanche Biopharma宣布完成7500万美元的B轮融资，获得的资金将用于开发治疗先兆子痫（preeclampsia）的RNAi疗法。Comanche公司的主要RNAi候选药物CBP-4888由两种siRNA构成，靶向胎盘产生的sFLT1 mRNA亚型。它通过减少胎盘产生的sFLT1蛋白，降低母体血液中的sFLT1蛋白水平。该公司计划于今年晚些时候启动CBP-4888在妊娠先兆子痫患者中的临床研究。该公司最近已完成一项在育龄女性健康志愿者中进行的1期临床试验。新闻稿指出，该疗法有望成为首款治疗这一疾病根本原因的疗法。Vico Therapeutics完成6000万美元B轮融资1月5日，Vico Therapeutics宣布完成6000万美元B轮融资。Vico Therapeutics是一家临床阶段的基因药物公司，为患有严重神经系统疾病的患者开发反义寡核苷酸（ASO）RNA调节疗法。该公司的主要候选产品VO659目前正处于1/2a期临床开发阶段，用于治疗脊髓小脑共济失调3型（SCA3）和1型（SCA1）以及亨廷顿病（HD）。据新闻稿报道，这是临床上用于靶向治疗由CAG重复扩增引起的九种已知多聚谷氨酰胺疾病的潜在首个ASO药物。此次融资将用于支持公司的整体运营，包括推进正在进行的VO659临床1/2a期多中心、开放标签篮子试验，该试验旨在评估轻度至中度SCA3、SCA1和早期HD患者鞘内注射多次递增剂量VO659的安全性和耐受性，首例患者于2023年4月接受给药。这笔融资还将有助于支持该公司进一步拓展其在其他遗传性神经疾病领域的产品管线。Basking Biosciences完成5500万美元融资2月1日，Basking Biosciences公司宣布完成5500万美元融资。所得资金将用于加速其潜在“first-in-class”的可逆RNA适配体溶栓疗法BB-031的临床开发，并推进能快速中和BB-031药理活性的逆转寡核苷酸BB-025进入1期临床研究。Basking Biosciences是一家处于临床阶段的生物医药公司，其成立的目的是为了解决急性血栓形成领域的最大需求——提供一种快速起效、短效的溶栓药物，这种药物能够重新打开堵塞的动脉，并且在发生出血并发症时能够迅速逆转其活性。公司主要候选药物包括BB-031和BB-025。BB-031采用核酸适配体（RNA aptamer）技术，通过靶向血管性血友病因子（vWF）治疗血栓形成，具有起效快、持续时间短的特点。多个缺血性中风的小型和大型动物的非临床模型研究结果表明，BB-031能够在中风发生后长达6小时的时间内重新打开脑部被血栓阻塞的动脉。BB-025则是一种能快速中和BB-031的溶栓活性的互补寡核苷酸。柯君医药宣布完成亿元B轮融资1月31日，柯君医药宣布完成亿元B轮融资。柯君医药致力于创新小分子和核酸药物的研发。公司目前已构建靶向前药技术平台和核酸药物技术平台，正在推进新一代抗血小板药物和多项抗病毒药物管线的临床开发。公司在研管线CG-0255是P2Y12受体拮抗剂类的抗血小板药物，针对急性冠状动脉综合征、心肌梗死、脑卒中等多种适应症。此外，公司CG2021V项目针对慢性乙肝功能性治愈适应症，利用创新的mRNA技术并联合siRNA产品，实现对机体的免疫调节作用，达到清除乙肝病毒的效果。据悉，本轮融资资金将主要用于推进柯君医药心脑血管核心管线产品CG-0255的关键临床开发，并推动管线中其他核心品种加速向IND推进。Resalis Therapeutics完成1000万欧元融资1月4日，Resalis Therapeutics公司宣布完成了1000万欧元（约1100万美元）的A轮融资。此轮融资所得款项将用于启动和完成其主要产品RES-010在肥胖症领域的首个人体1期临床试验，并为2期临床试验做好准备。RES-010是一种基于非编码RNA的ASO，靶向在调节脂质代谢和能量消耗中起核心作用的miR-22。RES-010旨在为肥胖症提供一种疾病调节方法，能更持久地减轻体重，并能与GLP-1受体激动剂等已获批准的治疗药物联合使用，延长治疗的持久性。Senisca完成370万英镑种子轮融资3月18日，Senisca宣布完成370万英镑种子轮融资。Senisca是一家生物技术公司，专注于调节RNA生物学以治疗与年龄相关的疾病。公司正在开发基于寡核苷酸的药物，以重置剪接因子水平并重新编程衰老细胞。具有促炎特征和功能受损的衰老细胞正在成为多种与年龄相关的疾病的致病因素，通过RNA剪接调节来针对与年龄相关的疾病，可减弱衰老来实现细胞重编程和疾病缓解。据悉，本轮融资将资助治疗管线项目的持续临床前开发。安龙生物完成数千万元A+轮融资1月23日，安龙生物宣布完成数千万元A+轮融资。安龙生物致力于基因治疗及小核酸药物的研发。安龙生物搭建了ANOG小核酸药物技术平台，基于Gal-Mola载体可将药物靶向肝外组织，目前应用于乙型肝炎、高血压等领域的药物研发。根据公司官网介绍，安龙生物拥有自主知识产权的肝外载体，靶向脑部、眼部、肺部、脂肪、肌肉、肿瘤等组织，可实现局部给药，有望实现更长周期给药。该公司已与两家上市公司（凯因科技、信立泰）进行合作，分别针对乙肝、高血压疾病开发寡核苷酸药物。Bolden Therapeutics获融资1月9日，Bolden Therapeutics获得新一轮投资。Bolden Therapeutics致力于开发治疗中枢神经系统疾病的疗法，以促进神经细胞再生，并改善神经系统疾病的预后。公司的科学联合创始人Justin Fallon博士和Ashley Webb博士已经确定了刺激神经干细胞以促进成人大脑中新神经元产生的关键分子通路，目前正在研究针对这一基因验证靶点的反义寡核苷酸，候选药物已准备好进行体内有效性测试。本轮投资或将用于促进神经再生的反义寡核苷酸药物研发和临床测试。Alys Pharmaceuticals完成1亿美元种子融资2月13日，投资公司Medicxi将六家专注于皮肤适应症的生物科技公司结合，推出具有多种平台技术与研发管线的Alys Pharmaceuticals，并同时以1亿美元种子融资支持其于免疫皮肤疾病领域当中创新疗法的开发。这笔资金将帮助Alys实现到2026年底前完成7至10项概念验证（POC）以及至少一个项目进入临床注册研究的目标。Alys由Medicxi和几位知名的皮肤病学和科学专家（包括诺贝尔奖得主Craig Mello博士）共同创立，致力于重新定义皮肤病治疗的前景。该公司结合了6家公司的管线和平台，其中包括开发皮肤疾病siRNA疗法的Aldena Therapeutics，预防放射疗法对健康皮肤的副作用的Graegis Pharmaceuticals等。目前，Alys共拥有十多个活跃项目，凭借其尖端平台，其管线将可能进一步增长，并涵盖包含炎症性疾病、自身免疫性疾病、癌症患者和罕见皮肤病等适应症。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐点击“在看”，分享创鉴汇健康新动态