Rege Nephro CO., Ltd. Announces Patient Enrollment for Phase II Clinical Trial of Tamibarotene for ADPKD

KYOTO,Japan,May 28,2024 -- 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 tamibaroteneas a developmental product for the treatment of autosomal dominant polycystic kidney disease (ADPKD) under the code 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.

Public Clinical Trial Information

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

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

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

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

About 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. 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.

About 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.

Media Inquiries:

Katsuhisa Yamaguchi,CFO

Rege Nephro CO.,Ltd.

TEL: 075-744-6858

E-mail: [email protected]