GPR6

BOSTON, Oct. 21, 2024 (GLOBE NEWSWIRE) -- Cerevance, a clinical-stage biopharmaceutical company advancing multiple cell type-specific therapies for the treatment of neurodegenerative, psychiatric, and central nervous system-controlled metabolic disorders, has published the full results from the Phase 2 clinical trial of solengepras, in eClinicalMedicine , a peer-reviewed journal published by The Lancet Discovery Science Suite . The results demonstrated that solengepras, a potentially first-in-class, oral, once-daily, non-dopaminergic, GPR6 inverse agonist, significantly reduced OFF time in individuals with Parkinson’s disease and was generally well-tolerated. The Phase 2 clinical trial met the key efficacy endpoint and demonstrated a reduction in average daily OFF time from baseline at Day 27, as recorded over two days using a self-reported patient motor diary. On Day 27, the change from baseline in daily OFF time was -1.6 hours (p 5%) were headache and nausea which were generally mild and transient. “We are excited to share the results of this important Phase 2 study with the broader scientific community through this publication in eClinicalMedicine,” said chief executive officer, Craig Thompson. “The data demonstrated solengepras’ potential to be a meaningful therapeutic option for individuals with Parkinson’s disease, addressing not only motor symptoms but also overall quality-of-life.” For more information on the Phase 2 clinical trial and to access the full publication, visit About Solengepras (CVN424) Solengepras is designed to provide a potentially novel approach to the treatment of Parkinson’s disease. Unlike dopaminergic therapies, which primarily act by replenishing, enhancing, or mimicking dopamine, solengepras is designed to selectively address the indirect pathway by modulating the GPR6 receptor. By inhibiting GPR6, solengepras aims to restore both motor and non-motor function without directly affecting dopamine levels or signaling, improving the relative balance between the direct and indirect pathways, and potentially reducing the risk of common side effects associated with dopaminergic therapies, such as dyskinesias and motor fluctuations. Solengepras is currently being evaluated as a once-daily, oral treatment for use as both a monotherapy (Phase 2 ASCEND clinical trial) and as an adjunctive therapy to levodopa and other anti-Parkinsonian medication (Phase 3 ARISE clinical trial). About Parkinson’s Disease Parkinson’s disease is a progressive neurodegenerative disorder that is characterized by both motor symptoms, such as tremor, rigidity, and bradykinesia/akinesia, and non-motor symptoms, such as mood changes, apathy, and cognitive deficits. Globally, Parkinson’s disease is the fastest growing neurological disorders, affecting more than ten million people worldwide and approximately one million people in the United States. The current standard of care has primarily relied on dopaminergic therapies, which lose effectiveness over time and are associated with side effects that result in challenging risk-benefit pro About Cerevance Cerevance is focused on advancing cell type-specific therapies for the treatment of neurodegenerative, psychiatric and central nervous system (“CNS”)-controlled metabolic disorders. Our proprietary platform, N uclear E nriched T ranscript S ort seq uencing (NETSseq), allows us to identify targets that are expressed at very low levels, that are present in rare cell types, or that change over time as a disease progresses. Our most advanced product candidate, solengepras (CVN424), is currently in Phase 3 development and has the potential to be a first-in-class, oral non-dopaminergic therapy for both the motor and non-motor symptoms of Parkinson’s disease. Our second product candidate, CVN766, is designed to be a highly selective oral antagonist of the orexin 1 receptor that we plan to evaluate in a Phase 2 study for the potential treatment of binge eating disorder and schizophrenia. Our third product candidate, CVN293, is a highly selective investigational oral inhibitor targeting potassium two pore domain channel subfamily K member 13 (KCNK13) and represents a potentially novel intervention point for neurodegenerative disorders by reducing neuroinflammation. We plan to evaluate CVN293 in a Phase 2 study for the potential treatment of frontotemporal dementia. Contacts Cerevance: Johnna Simões, ir@cerevance.com Media: Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。 据全球临床试验收录网站和中国临床试验登记平台统计，8月共17款创新药进入III期阶段，其中6款来自国内药企。这些在研药物覆盖靶点不乏HER2、IL-4Rα、TYK2等成熟靶点，也包括GPR6和LEPR这类还在探索成药性的靶点。本文筛选其中3款国内药企重点产品加以介绍。 01 TQB2102；适应症：HER2低表达乳腺癌 TQB2102是正大天晴自主研发的一种靶向HER2两个非重叠表位ECD2及ECD4的ADC，由人源化HER2 IgG1双抗通过连接子与拓扑异构酶I抑制剂偶联而成。TQB2102静注入血后，抗体部分与HER2阳性肿瘤细胞表面ECD2、ECD4结合，ADC复合物经细胞内吞并转运到溶酶体，连接子经酶切后释放出小分子药物，导致DNA损伤和细胞死亡。 截图来源：药融云全球药物研发数据库 目前，全球共37款临床在研HER2 ADC，其中3款为HER2双抗ADC，包括TQB2102、JSKN-003（康宁杰瑞）和KM501（康明百奥）。JSKN-003是最早进入III期阶段的HER2双抗ADC，其III期研究于2023年12月启动。 02 泰莱替尼；适应症：非小细胞肺癌 泰莱替尼（taletrectinib）是葆元医药与信达生物合作开发的一款ROS1/Trk抑制剂，已于2023年11月凭借II期TRUST-II研究的积极结果申报上市。TRUST-II研究结果显示，在初治ROS1阳性NSCLC患者（n=25）中，经确认的客观缓解率（cORR，标准为肿瘤体积缩小）达到了92%；在既往接受过1种ROS1-TKI抑制剂治疗的ROS1阳性NSCLC患者（n=21）中，cORR为57%。 截图来源：药融云全球药物研发数据库 此外，泰莱替尼对存在脑转移的患者（n=8）也有效，cORR为62.5%。初治患者的无进展生存期（PFS）尚未达到，经治患者的PFS约为11.7个月。泰莱替尼的总体耐受性良好，安全性与既往研究一致。最常见的治疗期间不良事件（TEAE）为肝酶升高和胃肠道相关不良事件，其中大多数为1级或2级。神经系统TEAE的发生率低，最常见的AE是头晕（13%），其中大部分是1级。ROS1基因重排是NSCLC的致癌驱动基因，发生于2%-3%的晚期NSCLC患者；NTRK基因融合是多种实体瘤的致癌驱动基因，发生于0.5%的多种晚期实体瘤患者。已上市的克唑替尼、恩曲替尼和恩莎替尼是此类患者的常用治疗选择。葆元医药在III期研究中也选择了克唑替尼作为泰莱替尼的挑战对象。 03 HRS-8427 ；复杂性尿路感染（包括急性肾盂肾炎） HRS-8427是头孢地尔衍生，是恒瑞抗感染领域的 1 类新药。该药进入 Ⅲ 期临床，单药用于治疗复杂性尿路感染。 截图来源：药融云全球药物研发数据库 HRS-8427通过抑制细胞壁的生物合成发挥抑菌杀菌作用，临床前显示其在多个标准菌株和临床分离菌株构建的小鼠感染模型中发挥抗菌药效，安全性良好。HRS-8427曾于2022年7月被批准针对尿路感染的临床试验，彼时恒瑞医药透露该产品的相关项目累计已投入研发费用约3307万元。 END 近期热门资源获取 后台回复关键词“报告”，获取数十篇药融云出品独家报告 后台回复关键词“2023首仿”，获取2023年首仿药物获批名单 后台回复“GLP-1深度报告”，获取完整《GLP-1产业现状与未来发展》PDF版。 后台回复“中国 I 类新药”，获取完整《中国 I 类新药靶点》PDF版。 后台回复“NAFLD/NASH”，获取完整《NAFLD/NASH报告》PDF版。 后台回复“AI+制药”，获取完整《中国AI制药企业白皮书》PDF版。 后台回复“XXG”，获取完整《中国心血管系统药物分析报告》PDF版。 后台回复“FZZJ”，获取完整《基于剂型改良的复杂注射剂分析》PDF版。 后台回复“药品进出口”，获取完整《中国药品进出口白皮书》深度报告-PDF版。 联系我们，体验药融云更多专业服务 会议 合作 园区 服务 数据库 咨询 定制 服务 媒体 合作 点击阅读原文，申请药融云企业版免费试用！

BOSTON, May 09, 2024 (GLOBE NEWSWIRE) -- Cerevance, a company focused on developing precision novel therapeutics for central nervous system (CNS) diseases, will present an oral presentation at the upcoming International Association of Parkinsonism and Related Disorders (IAPRD) XXIX World Congress on Parkinson’s Disease and Related Disorders, taking place in Lisbon, Portugal from May 19-22, 2024. Presentation details Title: CVN424, a Novel GPR6 Inverse Agonist Demonstrates Efficacy in an Adjunctive Parkinson’s Disease Phase 2 Clinical TrialOverview: In a Phase 2 clinical trial, CVN424, a novel, non-dopaminergic, GPR6 inverse agonist, demonstrated a significant reduction in OFF time and was safe and well tolerated with low rates of mostly mild and moderate adverse events. Patients also reported enhanced motor functionality in daily activities, as assessed by the UPDRS Part II. These findings enhance the confidence in CVN424's potential to address both motor and non-motor symptoms of Parkinson’s disease, consistent with observations in pre-clinical models, potentially heralding a transformative non-dopaminergic therapy.Presenter: Karl Kieburtz, MD, MPHSession Date and Time: Monday, May 20; 9:35 – 10:20am Session Title: PD Therapy I About CerevanceCerevance is focused on the development of precision treatments for central nervous system (CNS) disorders, including chronic neurodegenerative conditions such as Alzheimer's disease, Parkinson's disease, frontotemporal dementia, and amyotrophic lateral sclerosis. Utilizing a large and growing repository of over 15,000 human brain tissue samples, Cerevance is generating an unprecedented level of expression and epigenetic data thereby enabling the company to identify the most promising targets for the next generation of treatments for CNS disorders. The company uses its proprietary Nuclear Enriched Transcript Sort sequencing (NETSseq) platform and advanced machine learning techniques to uncover the gene expression profiles of select cell types to identify novel targets that are uniquely expressed in relevant circuits affected by diseases or are altered in disease states. With the information obtained from its research, combined with the expertise of its team of scientists and drug developers, Cerevance is advancing multiple therapeutics through clinical development, with CVN424, CVN766, and CVN293 being the furthest along in the pipeline. CVN424 is a first-in-class non-dopamine therapy that shows promise in improving both motor and non-motor symptoms of Parkinson's disease and may also have disease-delaying effects. CVN766 is a potent and highly selective antagonist of the orexin 1 receptor which may benefit various psychiatric conditions including schizophrenia, anxiety/panic, binge eating/obesity, substance use disorder, and Prader-Willi Syndrome. CVN293 is a novel blocker of potassium efflux in glia, regulating the inflammasome in individuals living with neurodegenerative diseases. Cerevance’s robust pipeline aims to transform the lives of patients affected by CNS diseases. Contacts Cerevance: Johnna Simoes, ir@cerevance.com Media: Andrew Mielach, amielach@lifescicomms.com, +1-646-876-5868