相关临床试验
9
0 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
2013
已完成
6
66.7%
招募中
1
11.1%
终止
2
22.2%
暂无批准数据
- Eloxx Pharmaceuticals completed a $66.0 million public offering and uplisted to Nasdaq, ending Q2 2026 with $62.0 million in cash and no outstanding debt. - The company plans to initiate a Phase 2b trial of exaluren in nonsense mutation Alport syndrome in Q3 2026, with topline data expected by mid-2027. - The European Commission granted orphan medicinal product designation for exaluren in autosomal dominant polycystic kidney disease (ADPKD) in July 2026. - Cash runway is expected to fund operations through data readouts for both Alport syndrome and ADPKD, extending into mid-2028.
- The Alport syndrome market across the 7MM was valued at USD 27 million in 2025, with the United States accounting for approximately 88% of the total market share. - No therapies are currently approved specifically for Alport syndrome; standard care relies on ACE inhibitors, ARBs, and kidney transplantation for end-stage disease. - ENYO Pharma's vonafexor demonstrated positive Phase II ALPESTRIA-1 results in January 2026, showing slowed kidney function decline, with a Phase III trial planned for 2H 2026. - The pipeline includes diverse mechanisms such as ELX-02 for nonsense mutations, setanaxib (NOX1/4 inhibitor), and BAY3401016 (anti-Sema3A monoclonal antibody), with total prevalent cases estimated at ~203,800 in the 7MM in 2025.
