PRD Therapeutics Announces Initiation of First-in-Human Study for PRD001

TOKYO--( BUSINESS WIRE )-- PRD Therapeutics, Inc., a clinical stage company focused on the development of novel lipid metabolism regulators targeting homozygous familial hypercholesterolemia (HoFH) and metabolic dysfunction associated fatty liver disease (MASH/MASLD), today announced that the company recently initiated dosing in a First-in-Human (FIH) clinical trial of PRD001, a first-in-class SOAT2 (formerly known as ACAT2) selective inhibitor.

“We are excited to initiate dosing in this clinical trial of PRD001. Many clinical trials have been conducted on SOAT1/2 dual or SOAT1 selective inhibitors, but this is the first clinical trial of an SOAT2 selective inhibitor” said Kanji Hosoda, Ph.D., CEO and co-founder of PRD Therapeutics. “Several results with SOAT1 or 2 knockout mice have been published, suggesting that knocking out or inhibiting only SOAT2 is crucial to demonstrate safety and efficacy. PRD001 is the world's first and only SOAT2-selective inhibitor and is expected to exhibit safety and efficacy in humans as well. Our preclinical animal models (LDL-R KO mice; HoFH model, and high-fat diets induced MASH model mice) have shown that PRD001 lowers blood and liver lipids and suppresses the progression of fatty liver and atherosclerosis with no adverse events. PRD001 has the potential to be the first-in-class effective and safe oral therapy for HoFH patients suffering from no or extremely low LDL receptor activity.”

This FIH Phase 1 study is designed to evaluate the safety, tolerability, pharmacokinetics, and the signs of early efficacy (LDL-C lowing effect, and liver fat quantitative value using MRI-PDFF) of PRD001 in adult healthy volunteers. More information can be found at NCT07034183 .

About PRD001

PRD001 is a first-in-class, orally available small molecule SOAT2-selective inhibitor. It uniquely controls three key pathways of lipid metabolism; cholesterol synthesis in the liver, cholesterol absorption in the small intestine, and uptake of blood LDL-C with a single drug, leading to a potent reduction in a blood LDL-C level independent of the LDL receptor.

This research and development is supported by Japan Agency for Medical Research and Development (AMED) under Strengthening Program for Pharmaceutical Startup Ecosystem (Project title: " Development of First-in-class oral lipid metabolism regulator PRD001 and POC obtained for lipid metabolism disorder ").

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