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标题: Promethera Biosciences在AASLD 2019上展示了针对HepaStem™治疗急性慢 [打印本页]

作者: StephenW    时间: 2019-11-13 12:19     标题: Promethera Biosciences在AASLD 2019上展示了针对HepaStem™治疗急性慢


Promethera Biosciences Presents Updated Clinical Results at AASLD 2019 for World’s First Stem Cell Therapy Trial using HepaStem™ in Acute-on-Chronic Liver Failure

3-Month Follow-up Data from the Fully Recruited Phase 2a Trial with a Total of 24 Patients Confirms Safety and Early Efficacy Trends and Dosing Regimen for Subsequent Trials
November 11, 2019 02:00 AM Eastern Standard Time

MONT-SAINT-GUIBERT, Belgium--(BUSINESS WIRE)--Promethera Biosciences SA, a global innovator in cell-based medicines and liver diseases, published updated clinical data from the ongoing phase 2a study (HEP101) of its lead product candidate HepaStem™ in patients with Acute-on-Chronic Liver Failure (ACLF) or acute decompensation (AD) at high risk of developing ACLF. The data were presented in an oral presentation at the Annual Meeting of the American Association for Study of Liver Diseases (AASLD) on November 10, 2019, in Boston, by Promethera’s principal investigator Prof. F. Nevens, KULeuven, Belgium. The data set confirmed earlier findings presented at The International Liver Congress™ - ILC 2019 in April.

Currently, liver transplant is the only rescue treatment for patients suffering from ACLF and to a lesser degree for patients with acute decompensation of the liver (AD). There are no other existing therapies that can rescue the end-stage-liver failure. Promethera is developing HepaStem™ as an alternative to transplant; it consists of liver derived stem cells that are obtained from ethically donated healthy human organs and expanded in GMP culture conditions. These liver derived signaling stem cells migrate through the blood stream to reach the liver, where they support the tissue regeneration via their potent secretome.

In the trial HEP101, HepaStem has been intravenously infused in single or repeated injections in multiple ascending doses in patients to assess the tolerability and the safety profile of the treatment. The trial which recruited a total of 24 patients, including 16 patients with ACLF and 8 patients with AD has met its primary endpoint.

With one or two repeated doses up to 1.2 million cells per kilogram of body weight, no adverse events related to HepaStem occurred and no clinically significant changes were shown in platelet count, fibrinogen levels, and coagulation factors following HepaStem infusion. The demonstrated safety of two infusions of 1.2 million cells per kilogram of body weight provide solid guidance for conducting the subsequent trials. In addition to the positive safety profile, the study has shown trends in efficacy with improvement in three indicators of liver disease severity; Model for End Stage Liver Disease score (MELD), Child-Pugh score and bilirubin levels, 28 days and three months after treatment initiation. Promethera plans to demonstrate the efficacy of HepaStem™ at 1.0 million cells per kilogram of body weight in ACLF with a large multicentric double blind placebo-controlled trial (HEP102). The study HEP102 is expected to start end 2019.

“We continue to pioneer the use of liver stem cells for the treatment of severe liver diseases and the updated status from the world’s first clinical trial evaluating a cell-based therapy in ACLF gives us confidence about the safety aspects and the broad therapeutic potential of our therapy,” said Etienne Sokal, M.D., Ph.D., Promethera’s Group Chief Medical Officer. “As a potential first tangible alternative to organ transplantation for an ever-growing patient population, HepaStem will advance into a larger efficacy trial in ACLF focusing on a reduced need for transplantation and a decreased mortality.”

Prof. Nevens added, “With the data set having evolved since we last presented results at this years’ ILC Congress, the Phase 2a trial provides a strong rationale for further clinical evaluation of the therapeutic concept and has provided a safe dosing regimen which will guide future studies.”

About Promethera Biosciences

Promethera Biosciences is a global innovator in liver therapeutics whose mission is to bring life-saving treatments to reduce the need for liver transplantation. Our lead clinical program, derived from our patented cell technology platform HepaStem, is designed to benefit from its immune-modulatory and anti-fibrotic properties. In addition to our cell-based pipeline we develop antibody technologies, such as the antiTNF-R1 antibody Atrosimab, to complement and diversify our therapeutic options. We are a team of international experts operating out of facilities in Mont-Saint-Guibert, Belgium, Durham, NC, USA, Tokyo, Japan and Basel, Switzerland.

Promethera®, HepaStem™, H2stem®, are all registered trademarks of the PROMETHERA group.

Contacts

Promethera Biosciences SA
Alexandra Schiettekatte
[email protected]
Web: www.promethera.com
作者: StephenW    时间: 2019-11-13 12:19

Promethera Biosciences在AASLD 2019上展示了针对HepaStem™治疗急性慢性肝衰竭的全球首例干细胞疗法试验的最新临床结果

全面招募的2a期试验(共24例患者)的3个月随访数据证实了安全性和早期疗效趋势以及后续试验的用药方案
十一月11,2019 02:00东部标准时间

比利时蒙圣-吉伯特-(美国商业资讯)-细胞药物和肝脏疾病的全球创新者Promethera Biosciences SA发布了正在进行的主要候选药物HepaStem的2a期研究(HEP101)的最新临床数据。 ™急性肝功能衰竭(ACLF)或急性代偿失调(AD)且罹患ACLF的风险很高。数据由Promethera首席研究员F.Nevens教授(比利时库鲁芬,比利时)于2019年11月10日在波士顿的美国肝病研究协会(AASLD)年会上进行了口头陈述。数据集证实了4月在国际肝病大会-ILC 2019上发表的早期发现。

目前,对于患有ACLF的患者,肝移植是唯一的抢救方法,对于急性肝失代偿(AD)的患者,肝移植的治疗程度较小。没有其他现有的疗法可以挽救末期肝功能衰竭。 Promethera正在开发HepaStem™,以替代移植。它由肝脏衍生的干细胞组成,这些细胞得自道德上捐赠的健康人体器官,并在GMP培养条件下扩增。这些肝脏衍生的信号干细胞通过血流迁移到达肝脏,在那里它们通过有效的分泌组支持组织再生。

在试验性HEP101中,已以多次递增剂量对患者进行单次或重复静脉注射HepaStem,以评估治疗的耐受性和安全性。该试验共招募了24位患者,其中包括16位ACLF患者和8位AD患者。

一次或两次重复给药最多可产生每千克体重120万个细胞,没有发生与HepaStem相关的不良事件,并且输注HepaStem后血小板计数,纤维蛋白原水平和凝血因子均未显示出临床显着变化。两次输注每公斤体重120万个细胞的安全性被证明为进行后续试验提供了坚实的指导。除了积极的安全性,该研究还显示出随着肝病严重性三个指标的改善疗效的趋势。治疗开始后28天和3个月的终末期肝病评分(MELD),Child-Pugh评分和胆红素水平模型。 Promethera计划通过一项大型多中心双盲安慰剂对照试验(HEP102)证明ACFA中HepaStem™在每公斤体重100万个细胞中的功效。这项研究HEP102预计将于2019年底开始。

“我们继续率先使用肝干细胞治疗严重的肝脏疾病,全球首个评估ACLF中基于细胞的疗法的临床试验的最新状态使我们对我们的安全性和广泛的治疗潜力充满信心疗法”,Promethera集团首席医学官医学博士Etienne Sokal说。 “对于不断增长的患者群体,HepaStem作为器官移植的潜在第一个切实可行的替代品,将进入ACLF的一项更大的功效试验,重点在于减少移植需求和降低死亡率。”

Nevens教授补充说:“自从我们上次在今年的ILC大会上发表结果以来,数据集不断发展,因此2a期临床试验为进一步对治疗概念进行临床评估提供了有力的依据,并提供了安全的给药方案,可指导未来的研究。”

关于普罗米修拉生物科学

Promethera Biosciences是肝脏疗法的全球创新者,其使命是带来挽救生命的疗法以减少对肝移植的需求。我们的领先临床计划源自我们已获专利的细胞技术平台HepaStem,旨在从其免疫调节和抗纤维化特性中受益。除了基于细胞的产品线外,我们还开发抗体技术,例如抗TNF-R1抗体Atrosimab,以补充和多样化我们的治疗选择。我们是一支由国际专家组成的团队,其业务遍及比利时的Mont-Saint-Guibert,美国北卡罗来纳州达勒姆,日本东京和瑞士巴塞尔。

Promethera®,HepaStem™,H2stem®是PROMETHERA集团的注册商标。

联络人

Promethera Biosciences SA
亚历山德拉(Alexandra Schiettekatte)
[email protected]
网址:www.promethera.com




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