KYOTO, Japan, May 28, 2024 /PRNewswire/ -- Rege Nephro CO., Ltd. (https://www.regenephro.co.jp/en) has announced that patient enrollment began in April for the second stage of its Phase II clinical trial of tamibarotene as a developmental product for the treatment of autosomal dominant polycystic kidney disease (ADPKD) under the code RN-014.

日本京都，2024年5月28日/PRNewswire/--Rege Nephro CO.，Ltd(https://www.regenephro.co.jp/en)已宣布，患者登记于4月开始进行他米巴罗汀II期临床试验的第二阶段，该试验是一种开发产品，用于治疗常染色体显性多囊肾病（ADPKD），代码为RN-014。

After reviewing all available safety data from the first stage, which started in December of last year, the decision was made to advance to the second stage. The company aims to complete patient enrollment by the end of 2024..

在审查了去年12月开始的第一阶段的所有可用安全数据后，决定进入第二阶段。该公司计划在2024年底前完成患者登记。。

Public Clinical Trial Information

公开临床试验信息

jRCT (Japan Registry of Clinical Trials): A system for the submission and publication of clinical research (https://jrct.niph.go.jp/latest-detail/jRCT2011230055)

jRCT（日本临床试验登记处）：临床研究提交和发表的系统(https://jrct.niph.go.jp/latest-detail/jRCT2011230055)（笑声）

ClinicalTrials.gov: A public website provided by the U.S. National Library of Medicine (https://clinicaltrials.gov/study/NCT06289998)

ClinicalTrials.gov：美国国家医学图书馆提供的公共网站(https://clinicaltrials.gov/study/NCT06289998)（笑声）

Tolvaptan is the only approved drug for the treatment of ADPKD. Since the launch of tolvaptan, pharmaceutical companies worldwide have been developing numerous clinical trials of new drugs to treat ADPKD, but most of these drugs have already been discontinued due to inefficacy, highlighting the strong need for new treatment options.

托伐普坦是唯一被批准用于治疗ADPKD的药物。自托伐普坦推出以来，世界各地的制药公司一直在开发许多治疗ADPKD的新药临床试验，但大多数这些药物由于无效而已经停止使用，突显出对新治疗方案的强烈需求。

RN-014 is currently at the most advanced stage of development among the drugs in development for patients with ADPKD..

RN-014目前处于ADPKD患者正在开发的药物中最晚期的开发阶段。。

About ADPKD

关于ADPKD

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary cystic kidney disease. It is characterized by the gradual development and enlargement of multiple cysts in both kidneys, leading to progressive renal dysfunction. Cysts can also occur in the liver and pancreas, and vascular abnormalities throughout the body are common, including high blood pressure, cerebral aneurysms, and heart valve abnormalities.

常染色体显性遗传性多囊肾病（ADPKD）是最常见的遗传性囊性肾病。它的特征是两个肾脏中多个囊肿的逐渐发展和扩大，导致进行性肾功能不全。囊肿也可能发生在肝脏和胰腺中，全身血管异常很常见，包括高血压，脑动脉瘤和心脏瓣膜异常。

Currently, tolvaptan, which inhibits cyst growth, is the only approved treatment for ADPKD, but there is no curative treatment available..

目前，抑制囊肿生长的托伐普坦是唯一被批准的ADPKD治疗方法，但尚无治愈方法。。

About RN-014

关于RN-014

Tamibarotene, under development for ADPKD, is a retinoic acid receptor (RAR) agonist expected to potently inhibit cyst formation and improve renal function. The clinical trial dose identified in multiple mouse model experiments is 4mg/day, and its safety has been confirmed in past studies. With advice from the Pharmaceuticals and Medical Devices Agency (PMDA), the Phase II clinical trial began in December 2023.

正在开发用于ADPKD的塔米巴罗汀是一种视黄酸受体（RAR）激动剂，有望有效抑制囊肿形成并改善肾功能。在多个小鼠模型实验中确定的临床试验剂量为4mg/天，其安全性已在过去的研究中得到证实。在药品和医疗器械管理局（PMDA）的建议下，第二阶段临床试验于2023年12月开始。

This trial includes a pharmacokinetic (PK) phase and a randomization phase. Various safety measures have been established, including phased patient enrollment, periodic independent safety monitoring, strict criteria for dose reduction and discontinuation of the investigational drug, and criteria for discontinuation of the entire trial..

该试验包括药代动力学（PK）阶段和随机化阶段。已经制定了各种安全措施，包括分阶段患者登记，定期独立安全监测，严格的剂量减少和停用研究药物标准，以及停止整个试验的标准。。

About Rege Nephro CO., Ltd.

关于Rege Nephro CO.，Ltd。

Rege Nephro CO., Ltd. is a clinical-stage biotech company utilizing induced pluripotent stem cell (iPSC)-based technology from the Center for iPS Cell Research and Application (CiRA), Kyoto University. It is developing therapeutics and cell therapy for kidney, liver, and pancreas diseases.

Rege Nephro CO.，Ltd.是一家临床阶段生物技术公司，利用京都大学iPS细胞研究与应用中心（CiRA）基于诱导多能干细胞（iPSC）的技术。它正在开发治疗肾脏、肝脏和胰腺疾病的疗法和细胞疗法。