Eloxx Pharmaceuticals Reports Positive Biopsy Results, ELX-02 Shows Promise in Alport Syndrome Treatment
Eloxx Pharmaceuticals Reports Positive Biopsy Results, ELX-02 Shows Promise in Alport Syndrome Treatment
Eloxx Pharmaceuticals, Inc. (NASDAQ:ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported results from an assessment of patient biopsies by NIPOKA GmbH (Nipoka). They have developed a highly accurate method for the quantification of podocyte foot process morphology. These results confirm previously reported positive biopsy results from the proof-of-concept Phase 2 open-label clinical trial (NCT05448755) of ELX-02 for the treatment of Nonsense Mutation Alport syndrome patients. Analysis of formalin-fixed paraffin-embedded (FFPE) biopsy samples by Nipoka show ELX-02 treatment improved podocyte foot process morphology with lower effacement in all three patients at the end of the 8-week study period.
亞洲網加利福尼亞州聖約瑟10月23日電罕見疾病核糖體核糖體靶向基因療法的領先企業Eloxx製藥公司(納斯達克代碼：ELOX)今天公佈了NIPOKA公司(NIPOKA)(NIPOKA)對患者活組織檢查的評估結果。他們開發了一種高精度的足細胞足突形態量化方法。這些結果證實了先前報道的ELX-02治療無義突變Alport綜合徵患者的概念驗證第二階段開放標籤臨床試驗(NCT05448755)的陽性活檢結果。Nipoka對福馬林固定石蠟包埋(FFPE)活檢樣本的分析表明，ELX-02治療改善了所有三名患者在8周研究期結束時足細胞足突的形態，並降低了消失率。