Clinical Trials
16
1 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
N/A
Active, not recruiting
1
6.3%
Completed
9
56.3%
Recruiting
5
31.3%
Terminated
1
6.3%
No approval data available
- 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.
- ENYO Pharma's Phase 2 Alpestria-1 study demonstrated that vonafexor reversed historical kidney function decline from -6.4 mL/min/1.73 m²/yr to a mean functional gain of +4.8 mL/min/1.73 m²/yr in Alport syndrome patients. - The FXR agonist showed durable effects with 73% of patients maintaining albuminuria reduction three months after treatment cessation, suggesting disease-modifying potential. - The company plans to advance vonafexor to Phase 3 development in Alport syndrome, with FDA Fast Track designation submission planned for January 2026.
• The non-alcoholic steatohepatitis (NASH) market is poised for substantial growth, projected to expand significantly by 2032 across the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan. • This growth is fueled by the introduction of novel therapies targeting NASH, with several drugs in Phase II and Phase III clinical trials showing promise for improving treatment outcomes. • Key players such as Madrigal Pharmaceuticals, Intercept Pharmaceuticals, and Novo Nordisk are actively developing innovative treatments, contributing to a dynamic and competitive market landscape. • The increasing prevalence of NASH and the growing understanding of its pathophysiology are driving the demand for effective therapies, creating opportunities for pharmaceutical companies.
