Latest news with #PRD001
Business Wire
16 hours ago
- Health
- Business Wire
PRD Therapeutics宣布啟動PRD001的首例人體研究
東京--(BUSINESS WIRE)--(美國商業資訊)-- PRD Therapeutics, Inc.是一家臨床階段公司，專注于開發靶向純合子家族性高膽固醇血症(HoFH)和代謝功能障礙相關脂肪性肝病(MASH/MASLD)的新型脂質代謝調節劑。該公司今日宣布，近期已啟動PRD001的首例人體(FIH)臨床試驗給藥。PRD001是一款同類首創的SOAT2（原稱ACAT2）選擇性抑制劑。 PRD Therapeutics執行長兼共同創辦人Kanji Hosoda博士表示：「我們很高興啟動PRD001臨床試驗的給藥。此前已展開過多項針對SOAT1/2雙重抑制劑或SOAT1選擇性抑制劑的臨床試驗，但這是第一個針對SOAT2選擇性抑制劑的臨床試驗。多項關於SOAT1或2基因敲除小鼠的研究結果已發表，顯示僅敲除或抑制SOAT2對證明安全性和有效性至關重要。PRD001是全球第一個且唯一的SOAT2選擇性抑制劑，可望也在人體中展現安全性和有效性。我們的臨床前動物模型（LDL-R KO小鼠；HoFH模型，以及高脂飲食誘導的MASH模型小鼠）顯示，PRD001可降低血液和肝臟脂質水準，抑制脂肪肝和動脈粥樣硬化的進展，且無不良事件。對於LDL受體活性缺失或極低的HoFH病患，PRD001可望成為同類第一個有效且安全的口服療法。」 該FIH 1期研究旨在評估PRD001在成年健康志願者中的安全性、耐受性、藥物動力學及早期療效跡象（LDL-C降低效應，以及使用MRI-PDFF檢測的肝臟脂肪量化值）。更多資訊可在 NCT07034183 查詢。 關於PRD001 PRD001是同類首創的口服小分子SOAT2選擇性抑制劑。它透過單一藥物獨特控制脂質代謝的三大關鍵途徑：肝臟膽固醇合成、小腸膽固醇吸收以及血液LDL-C攝取，從而強效降低血液LDL-C水準，且不依賴LDL受體。 本研發專案由日本醫療研究開發機構(AMED)透過 製藥初創企業生態系統強化計畫 提供支援（專案名稱：「同類首創口服脂質代謝調節劑PRD001的開發及脂質代謝紊亂的POC驗證」）。 免責聲明：本公告之原文版本乃官方授權版本。譯文僅供方便瞭解之用，煩請參照原文，原文版本乃唯一具法律效力之版本。
Business Wire
20 hours ago
- Health
- Business Wire
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 ").
