Summarized by Masters of Longevity from PR Newswire.
A biotech press release describing a finalized 52-week clinical trial design for a readthrough therapy in nonsense-mutation ADPKD and a new chief medical officer joining the program.
Key Takeaways
- The EXPRESS Phase 2 will randomize ~60 adults with PKD1 or PKD2 nonsense mutations 1:1 to exaluren or placebo for 52 weeks.
- Primary endpoint is annualized change in height-adjusted total kidney volume measured by MRI with scans at baseline and Weeks 28, 36, and 52.
- Eloxx reported peer-reviewed preclinical data showing exaluren restores full-length polycystin-1 in cells with PKD1 nonsense mutations.
