Rege Nephro Begins Phase II Trial Enrollment for ADPKD Drug Tamibarotene

Rege Nephro CO., Ltd. has recently commenced patient enrollment for the second phase of its Phase II clinical trial of tamibarotene, intended for treating autosomal dominant polycystic kidney disease (ADPKD). The initiative, under the code RN-014, began enrolling patients in April 2024 following a thorough safety review of data from the first phase, which started in December 2023. The company is aiming to complete this enrollment by the end of 2024.

ADPKD is a prevalent hereditary condition characterized by the development and growth of multiple cysts in the kidneys, which gradually leads to renal dysfunction. The disease can also manifest with cysts in other organs such as the liver and pancreas and can cause various vascular abnormalities including high blood pressure, cerebral aneurysms, and heart valve issues. Currently, the only approved treatment for ADPKD is tolvaptan, which primarily aims to inhibit cyst growth. However, it is not a curative treatment, which underscores the need for new therapeutic options.

Tamibarotene, the drug under investigation by Rege Nephro, is a retinoic acid receptor (RAR) agonist that has shown promise in inhibiting cyst formation and enhancing renal function. The clinical trial dose, determined through various mouse model experiments, is set at 4mg per day, and prior studies have confirmed its safety. Following guidance from the Pharmaceuticals and Medical Devices Agency (PMDA), the Phase II trial commenced in December 2023. This trial is structured into a pharmacokinetic (PK) phase and a randomization phase, with stringent safety protocols in place. These safety measures include phased patient enrollment, regular independent safety monitoring, strict criteria for dose reduction and discontinuation of the drug, and conditions for the potential halting of the entire trial.

Rege Nephro CO., Ltd., a biotech firm in the clinical stage based on induced pluripotent stem cell (iPSC) technology from the Center for iPS Cell Research and Application (CiRA) at Kyoto University, is at the forefront of developing treatments and cell therapies for diseases of the kidney, liver, and pancreas. The company aims to leverage its advanced scientific research to address the unmet medical needs in these areas.

This trial is part of a larger effort within the pharmaceutical community to find new treatments for ADPKD, following the limited success of previous trials with other drugs. The fact that many of these have been discontinued due to inefficacy highlights the critical need for effective new therapies. RN-014 represents the most advanced stage of development for any new drug currently in the pipeline for ADPKD patients.

The trial information is accessible through jRCT (Japan Registry of Clinical Trials) and ClinicalTrials.gov, which are public platforms for the submission and dissemination of clinical research data. This transparency is crucial for the ongoing development of tamibarotene and for fostering trust and collaboration within the medical and patient communities.

In summary, Rege Nephro's ongoing clinical trial holds promise for advancing the treatment landscape for ADPKD. The company’s innovative approach and rigorous safety protocols are designed to ensure the trial’s success and contribute valuable insights into the potential of tamibarotene as a treatment for this challenging condition.