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MiNA治疗学提出了在AASLD肝脏会议上证明MTL-CEBPA在肝衰竭动物 [复制链接]

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发表于 2016-12-13 13:45 |只看该作者 |倒序浏览 |打印
MiNA Therapeutics Presents Data Demonstrating Survival Benefit of MTL-CEBPA in Animal Model of Liver Failure at AASLD Liver Meeting
By
John Kent -
December 12, 2016

MiNA Therapeutics, the pioneer in RNA activation therapeutics, today announced the presentation of pre-clinical data on its MTL-CEBPA program in which the compound was shown to improve survival in a rat model of liver failure. MTL-CEBPA is the first development candidate to emerge from MiNA’s RNA activation platform and is currently being evaluated in a Phase I clinical study in patients with liver cancer.

The data were presented at the 2016 American Association for the Study of Liver Diseases (AASLD) Liver Meeting on November 14 in Boston, USA in a poster titled “The clinical candidate MTL-CEBPA leads to significant reduction in ascites and improvement in overall survival in a CCl4-induced liver failure model”. MTL-CEBPA is a SMARTICLES® liposomal formulation of a small activating RNA targeting the CEBPA gene.

“The clear and sustained survival benefit we see in the animal model of chronic liver failure illustrates the great potential of MTL-CEBPA to treat severe and late-stage liver diseases which represent significant medical burdens to both patients and society,” commented Robert Habib, CEO of MiNA Therapeutics. “Although the reported data are pre-clinical, we are currently in the clinic testing this compound in patients, many of whom have a background of liver disease. We intend to present the results of our Phase I study in 2017.”

In the experiments covered by the presentation, MTL-CEBPA was administered systemically for twelve weeks in male Sprague Dawley rats with CCl4-induced liver failure. In the treated animals, MTL-CEBPA attenuated hyper-ammonaemia and ameliorated ascites, two hallmarks of liver decompensation. These benefit were maintained in animals up to 14 weeks following completion of treatment. Treated animals demonstrated a very significantly improved survival rate compared to the control group at completion of the study.

The poster presented at the AASLD Liver Meeting is available on the Company’s website in the publication section under “Media”.

About MTL-CEBPA

MTL-CEBPA consists of a double stranded RNA formulated into a SMARTICLES® liposomal nanoparticle and is designed to activate the CEBPA gene. By restoring CEBPA expression to normal levels, MTL-CEBPA has been demonstrated to attenuate or reverse liver disease in a range of pre-clinical studies including models of liver cancer, liver cirrhosis, non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). MTL-CEBPA is currently under evaluation in OUTREACH, a first-in-human Phase I clinical study in patients with severe liver cancer. The multi-centre Phase I study will assess the safety and tolerability of MTL-CEBPA in patients with advanced primary or metastatic liver cancer who are ineligible or resistant to standard therapies. To learn more about the OUTREACH clinical study, please visit our listing at clinicaltrials.gov

About MiNA Therapeutics

Harnessing the innate mechanism of gene activation, MiNA Therapeutics’ platform enables the development of new medicines that restore normal function to patients’ cells. We are applying our technology and clinical know-how to transform the therapy landscape of severe liver diseases. Our initial product candidate will achieve clinical proof of concept in 2017.

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发表于 2016-12-13 13:46 |只看该作者
MiNA治疗学提出了在AASLD肝脏会议上证明MTL-CEBPA在肝衰竭动物模型中的生存​​获益的数据
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John Kent -
2016年12月12日

MiNA Therapeutics是RNA活化治疗的先驱,今天宣布介绍其MTL-CEBPA程序的临床前数据,其中该化合物显示改善肝衰竭大鼠模型的存活率。 MTL-CEBPA是从MiNA的RNA活化平台出现的第一个发展候选物,目前正在肝癌患者的I期临床研究中进行评估。

数据在11月14日在美国波士顿举行的2016年美国肝脏疾病研究协会(AASLD)肝脏会议上以标题为“临床候选物MTL-CEBPA导致腹水显着减少和总生存期改善的海报CCl4诱导的肝衰竭模型“。 MTL-CEBPA是靶向CEBPA基因的小活化RNA的脂质体制剂。

“我们在慢性肝衰竭动物模型中看到的明确和持久的生存获益说明了MTL-CEBPA治疗严重和晚期肝脏疾病的巨大潜力,这些疾病对患者和社会都是重大的医疗负担”,Robert Habib, MiNA治疗公司的首席执行官。 “虽然报告的数据是临床前的,我们目前正在临床测试这种化合物的患者,其中许多有肝脏疾病的背景。我们打算在2017年介绍我的第一阶段研究的结果。

在介绍的实验中,MTL-CEBPA在具有CCl4诱导的肝衰竭的雄性Sprague Dawley大鼠中全身施用12周。在治疗的动物中,MTL-CEBPA减轻高氨血症和改善腹水,肝失代偿的两个标志。在完成治疗后达14周的动物中保持这些益处。在研究完成时,与对照组相比,经处理的动物显示出非常显着改善的存活率。

在AASLD肝脏会议上提供的海报可在公司网站的“媒体”下的出版物部分中找到。

关于MTL-CEBPA

MTL-CEBPA由配制成脂质体纳米颗粒的双链RNA组成,并被设计用于激活CEBPA基因。通过将CEBPA表达恢复到正常水平,已经证明MTL-CEBPA在一系列临床前研究中减轻或逆转肝脏疾病,包括肝癌,肝硬化,非酒精性脂肪性肝病(NAFLD)和非酒精性脂肪性肝炎(NASH)。 MTL-CEBPA目前正在OUTREACH中进行评估,这是在严重肝癌患者中的第一人称I期临床研究。多中心I期研究将评估MTL-CEBPA在晚期原发性或转移性肝癌患者中的安全性和耐受性,这些患者不符合标准疗法或对标准疗法有抗性。要了解更多关于OUTREACH临床研究,请访问我们的上市在clinicaltrials.gov

关于MiNA治疗

利用基因激活的固有机制,MiNA治疗平台使得能够开发恢复患者细胞正常功能的新药。我们正在运用我们的技术和临床知识来改变严重肝脏疾病的治疗景观。我们的初始产品候选人将在2017年实现临床概念证明。
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