Versantis 将在 AASLD 上为失代偿期肝硬化患者提供 VS-01的阳性 1b

StephenW

Versantis 将在 AASLD 上为失代偿期肝硬化患者提供 VS-01的阳性 1b 期数据
2021 年 11 月 15 日，星期一，晚上 7:00·5 分钟阅读

VS-01进入针对慢性肝功能衰竭急性期患者的2a期研究

苏黎世，2021 年 11 月 15 日--(美国商业资讯)-- Versantis 是一家临床阶段的生物技术公司，为孤儿肝脏和儿科疾病开发新疗法，今天宣布其主要研究孤儿-指定产品 VS-01，用于 12 名失代偿性肝硬化住院患者，将在美国肝病研究协会 (AASLD) 年会上发表。数据显示 VS-01 在这些患者中是安全的且耐受性良好，并在该临床研究中显示出有希望的早期疗效迹象。 VS-01 是一种可能挽救生命的多器官支持疗法，旨在通过增强穿刺后氨和其他毒素的清除来及时逆转急性慢性肝衰竭 (ACLF)。

被选作口头报告的摘要如下：

口头报告标题：失代偿性肝硬化患者腹腔注射 VS-01 的安全性、初步疗效和药代动力学：一项首次人体、开放标签、1b 期临床试验
主讲人：Frank Erhard Uschner 博士，法兰克福歌德大学诊所内科 I 部转化肝病科，
会议日期/时间：2021 年 11 月 15 日，星期一，下午 12:30美东时间

由 AASLD 举办的年度肝脏会议汇集了来自世界各地的与会者，交流最新研究成果，讨论治疗的新进展，并与该领域的其他人建立联系。

这项单中心首次人体研究的主要目的是评估腹水和轻度肝性脑病肝硬化患者在单次和多次腹腔内给药后，在标准护理基础上腹腔给药 VS-01 的安全性和耐受性。次要目标是收集初步 PK、PD 和临床疗效数据。总共 12 名患者在法兰克福歌德大学 I 内科完成了治疗，并被分配接受单剂 VS-01（A 部分；n=9）或连续四剂（B 部分；n= 3）。在这些患者的腹水引流后，VS-01 然后通过现有的穿刺导管注入腹腔，并在 2-3 小时的停留时间后取出。没有报告与治疗相关的严重不良事件，也没有患者因不良事件而停止治疗。 VS-01 还展示了有希望的临床疗效结果，包括高且剂量依赖性的氨清除率、基于心理测试的肝性脑病的有希望改善，以及增加 ACLF 相关代谢物的腹膜清除率。

“数据显示 VS-01 在患有腹水和隐性（轻度）肝性脑病的肝硬化患者中是安全且耐受性良好的，非常有希望。我们能够通过治疗性穿刺导管使用标准医院设备对 VS-01 进行给药，这是我们相信可以很容易地纳入患者的护理标准中，”法兰克福歌德大学转化肝病学系首席研究员兼负责人 Trebicka 教授说。 “这些患者可用的治疗方法很少，而 VS-01 是对这些患者的补充，因此我们很高兴继续推进它的开发，并希望生成支持这种突破性临床方法的数据。”

“VS-01 代表了一种有前景的新疗法，可用于治疗腹水和肝硬化急性并发症的患者。这项早期研究的数据非常有希望，重要的是表明 VS-01 在这些患者中似乎是安全的且耐受性良好。氨和 ACLF 代谢物清除数据特别令人鼓舞，” Versantis 首席执行官兼联合创始人文森特福斯特补充道。 “这项研究的成功完成支持 VS-01 的未来发展。我们现在正准备在 ACLF 患者中启动一项多中心 2a 期研究，ACLF 是一种服务严重不足和资源不足的适应症。连同我们的创新产品管道，我们致力于为患有急性肝病的患者开发和商业化新的治疗方案。”
关于急性慢性肝衰竭 (ACLF)

ACLF 是一种医疗服务不足的疾病，尽管有最好的护理，但仍与高短期死亡率有关。 它的特征是先前存在的慢性肝病（例如，肝硬化）突然危及生命的恶化，导致肝脏和肝外器官衰竭迅速发展为昏迷和死亡。 在美国和欧盟，每年至少有 150,000 名患者因 ACLF 住院。 由于糖尿病、肥胖症、脂肪肝疾病和饮酒的患病率较高，发病率正在上升。 通过及时逆转 ACLF 和肝硬化引起的多器官并发症，VS-01 旨在改善这些患者的预后并减轻这种晚期肝病日益增加的健康和经济负担。

StephenW

Versantis to Present Positive Phase 1b Data at AASLD for VS-01 in Patients with Decompensated Cirrhosis
Mon, November 15, 2021, 7:00 PM·5 min read

VS-01 to Advance into Phase 2a study in Patients with Acute on Chronic Liver Failure

ZURICH, November 15, 2021--(BUSINESS WIRE)--Versantis, a clinical-stage biotechnology company developing novel therapies for orphan liver and pediatric diseases, today announced that positive Phase 1b clinical data from a study of its lead investigational, orphan-designated product, VS-01, in 12 hospitalized patients with decompensated liver cirrhosis will be presented at the American Association for the Study of Liver Diseases (AASLD) annual meeting. The data show that VS-01 was safe and well tolerated in these patients, and demonstrated promising early indications of efficacy in this clinical study. VS-01 is a potentially lifesaving, multi-organ support therapy that aims to timely reverse Acute-on-Chronic Liver Failure (ACLF) by enhancing the clearance of ammonia and other toxins following paracentesis.

The Abstract selected for oral presentation is as follows:

Oral Presentation Title: Safety and Preliminary Efficacy and Pharmacokinetics of Intraperitoneal VS-01 Infustions in Patients with Decompensated Liver Cirrhosis: A First-in-Human, Open-label, Phase 1b Clinical Trial
Presenter: Dr. Frank Erhard Uschner, Section for Translational Hepatology, Department of Internal Medicine I, Goethe University Clinic Frankfurt,
Session Date/Time: Monday, November 15, 2021, 12:30 p.m. EST

The annual Liver Meeting, held by AASLD, brings together attendees from around the world to exchange the latest research, discuss new developments in treatments, and network with others in the field.

The primary objective of this single-center first-in-human study was to evaluate the safety and tolerability of i.p.-administered VS-01 on top of standard of care in cirrhotic patients with ascites and mild hepatic encephalopathy following single and multiple intraperitoneal administrations. The secondary objectives were to gather preliminary PK, PD, and clinical efficacy data. In total, all 12 patients completed treatment in the Department of Internal Medicine I, Goethe University Frankfurt and were assigned to receive either a single dose of VS-01 (Part A; n=9) or four consecutive doses (Part B; n=3). Following the drainage of ascites in these patients, VS-01 was then infused into the peritoneal cavity via the existing paracentesis catheter and removed after a dwell time of 2-3 hours. No treatment-related serious adverse events were reported and no patient discontinued treatment due to an adverse event. VS-01 also demonstrated promising clinical efficacy results, including a high and dose-dependent ammonia clearance, promising improvement in hepatic encephalopathy based on psychometric tests, and increased peritoneal clearance of ACLF-related metabolites.

"The data show that VS-01 is safe and well tolerated in cirrhotic patients with ascites and covert (mild) hepatic encephalopathy, so very promising. We were able to administer VS-01 using standard hospital equipment via the therapeutic paracentesis catheter, which we believe can easily be incorporated into standard of care for patients," said Prof. Dr. Trebicka, the principal investigator and head of the Section of Translational Hepatology in Goethe University Frankfurt. "There are very few treatments available for these patients and VS-01 is complementary to those, so we are excited to continue advancing it’s development and hopefully generate the data supporting this ground-breaking clinical approach."

"VS-01 represents a promising new therapeutic for the potential treatment of patients with ascites and acute complications of cirrhosis. The data from this early study were very promising and importantly show that VS-01 appears to be safe and well tolerated in these patients. The amomonia and ACLF metabolites clearance data is particularly encouraging," added Vincent Forster, CEO and co-Founder of Versantis. "The successful completion of this study supports future development of VS-01. We are now preparing to initiate a multi-center Phase 2a study in patients with ACLF, a seriously underserved and under-resourced indication. Together with our pipeline of innovative products, we are committed to developing and commercializing new treatment options for patients suffering from acute liver diseases."
   About Acute-on-Chronic Liver Failure (ACLF)

ACLF is an underserved medical condition, which, despite best possible available care, is associated with high short-term mortality. It is characterized by an abrupt life-threatening worsening of a pre-existing chronic liver disease (e.g., cirrhosis) resulting in liver and extrahepatic organ failure rapidly progressing into coma and death. Every year at least 150’000 patients are hospitalized with ACLF in the US and EU. The incidence is growing due to a higher prevalence of diabetes, obesity, fatty liver diseases, and alcohol consumption. By timely reversing ACLF and the multi-organ complications arising from cirrhosis, VS-01 aims to improve outcomes in these patients and relieve the growing health and economic burden of this advanced liver disease.