Cerevance Achieves Key Endpoints in Phase 1 Clinical Trial of Novel Parkinson's Disease Drug CVN424


2019-05-15 20:36:00 Drugs


Cerevance, a clinical stage biopharmaceutical company advancing new medicines for brain diseases, has successfully completed its Phase 1 clinical trial of CVN424, the company’s first-in-class, orally-delivered compound in development for the treatment of Parkinson’s disease. The clinical study successfully met its primary endpoint of safety in healthy volunteers. The study included 64 healthy volunteers who received either single doses or seven daily doses of CVN424, ranging from 1 mg to 225 mg, or placebo. There were no serious or severe adverse events or clinically significant changes in vital signs, ECG or laboratory values. The drug was rapidly absorbed orally and had a half-life that will support once-daily dosing. “CVN424’s excellent safety profile helps validate Cerevance’s approach to CNS target selection and drug discovery,” said David H. Margolin, MD, PhD, Senior Vice President for Clinical and Translational Medicine at Cerevance. “CVN424 acts through a target that is selectively expressed by neurons important in controlling movement and was engineered to achieve excellent CNS penetration with minimal effects on other organs.” CVN424 acts on a novel, non-dopaminergic target protein present specifically in dopamine receptor D2-expressing medium spiny neurons in the indirect pathway of the basal ganglia. The compound modulates the D2-dependent indirect pathway, but not the D1-dependent direct pathway. This selective targeting is designed to produce the positive therapeutic effects of current treatments while avoiding side effects such as dyskinesia associated with dopaminergic therapies. “We are pleased with the Phase 1 trial results for CVN424 and are eager to advance into Phase 2,” said Brad Margus, Chief Executive Officer of Cerevance. “Our NETSseq technology platform uses human post-mortem brain tissue to identify targets that are selectively expressed in specific cell types and circuits or changed in disease states, allowing us to discover new therapeutics for neurodegenerative diseases. Beyond CVN424, we look forward to advancing a broad pipeline of therapies acting on targets that have not yet been explored for the treatment of CNS disorders.” Based on the positive Phase 1 results, a Phase 2 study to demonstrate the clinical benefits of CVN424 will be initiated later this year.
塞力万斯,一家临床阶段的生物制药公司,致力于脑病新药的开发,已经成功完成了 CVN424的一期临床试验, CVN424是公司在开发中用于治疗帕金森病的第一个口服复方制剂。临床研究成功地满足了健康志愿者的主要安全终点。 这项研究包括64名健康志愿者,他们接受单剂量或每天7次 CVN424剂量,范围从1毫克到225毫克,或安慰剂。生命体征、心电图或实验室值无严重或严重不良事件或临床显著变化。该药物口服吸收迅速,半衰期将支持每日一次的剂量。 “ CVN424卓越的安全性档案有助于验证 Cervance 对 CNS 目标选择和药物发现的方法,” Cervance 临床和转化医学高级副总裁 David H . Margolin 博士说。“ CVN424通过一个目标发挥作用,该目标由控制运动的重要神经元选择性表达,并被设计成实现出色的中枢神经系统渗透,对其他器官的影响最小。” CVN424在基底神经节间接通路中作用于多巴胺受体 D2表达的中枢神经中特异存在的新型非多巴胺能靶蛋白。该化合物调节 D2依赖的间接途径,而不是 D1依赖的直接途径。这一选择性靶向治疗的目的是产生积极的治疗效果,目前的治疗,同时避免副作用,如运动障碍相关的多巴胺治疗。 “我们对 CVN424的第1阶段试验结果感到满意,并渴望进入第2阶段,” Cerevance 首席执行官布拉德•马吉斯( Brad Margus )表示。“我们的 NETSseq 技术平台使用人类死后脑组织来识别在特定细胞类型和回路中选择性表达或在疾病状态中改变的目标,使我们能够发现神经退行性疾病的新疗法。除了 CVN424外,我们期待着推进一系列广泛的治疗方法,这些治疗方法针对的目标尚未被探索用于治疗中枢神经系统疾病。” 基于第1期的阳性结果,将于今年晚些时候启动第二期研究,以证明 CVN424的临床益处。