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.
罕见病核糖体RNA靶向基因疗法的领导者Eloxx Pharmicals, Inc.（纳斯达克股票代码：ELOX）今天公布了NIPOKA GmbH（Nipoka）对患者活检的评估结果。他们开发了一种高度准确的方法来量化足细胞足部过程形态。这些结果证实了先前报告的用于治疗胡说八道突变Alport综合征患者的 ELX-02 2期开放标签临床试验（NCT05448755）的活检阳性结果。Nipoka对福尔马林固定石蜡包裹（FFPE）活检样本的分析表明，在为期8周的研究期结束时，ELX-02 治疗改善了足细胞足部过程形态，所有三名患者的流失率都较低。