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」）。
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