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Rege Nephro突破遺傳性腎病難題 RN-014 有望改寫 ADPKD 治療現況

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日期2025-07-10
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突破遺傳性腎病難題,RN-014 有望改寫 ADPKD 治療現況。

RN-014:常染色體顯性多囊腎病(ADPKD) 是最常見的遺傳性且難治性疾病,其特徵為腎臟內逐漸形成大量囊腫,最終導致末期慢性腎衰竭。

本公司利用疾病專一性 iPS 細胞所建立的人類疾病模型,成功發現針對 ADPKD 的小分子治療藥物。
 
目前唯一獲准治療 ADPKD 的藥物為 tolvaptan,儘管副作用嚴重,其年銷售額仍高達 15 億美元。

基於此,我們的產品具有成為「同類首創(first-in-class)且首線治療(first-line)」藥物的潛力。

預計將根據正在進行中的第二期臨床試驗結果,於 2025 年底前獲得有關本候選藥物療效的實質證據。

RN-014: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common hereditary and intractable disease characterized by the progressive formation of numerous renal cysts, leading to end-stage chronic kidney failure.

A small-molecule therapeutic for ADPKD was discovered through a human disease model using disease-specific iPS cells. Despite severe side effects, the only treatment option, tolvaptan, generates sales of $1.5 billion.

Given this, our product has the potential to be a first-in-class and first-line therapy.

We aim to obtain evidence of our therapeutic candidate's efficacy by the end of 2025, based on results from our ongoing Phase 2 clinical trial.