ELOX Eloxx Pharmaceuticals

Eloxx Announces FDA IND Clearance for Exaluren in Nonsense-Mutation ADPKD; EXPRESS Phase 2 First Patient 3Q 2027

FDA cleared the IND for exaluren in nonsense-mutation ADPKD, enabling U.S. site activation for the global Phase 2 EXPRESS trial. The trial will randomize ~60 patients 1:1 to exaluren or placebo for 52 weeks with annualized htTKV growth as primary endpoint; first patient dosing is planned for 3Q 2027 and topline data in 1H 2029. Pre-IND FDA feedback noted that substantial durable htTKV effect could support Breakthrough Therapy Designation and that htTKV is a potential surrogate for accelerated approval.

Eloxx Finalizes EXPRESS Phase 2 Design in Nonsense-Mutation ADPKD

Eloxx finalized the design of the global EXPRESS Phase 2 trial in nonsense-mutation ADPKD: 52-week dosing of ~60 patients (1:1 exaluren vs placebo) with annualized htTKV growth rate as primary endpoint assessed by MRI. First patient dosing is expected in 3Q 2027 and 52-week topline data in 1H 2029. The company also received US orphan drug designation for ADPKD and noted that substantial durable htTKV effect could support Breakthrough Therapy Designation or accelerated approval.

exaluren · NCT07523581 registry update

countries: {'total_sites': 12, 'us_sites': 8} → {'total_sites': 11, 'us_sites': 7}

Eloxx: EC grants orphan designation for exaluren in ADPKD

The European Commission granted orphan medicinal product designation for exaluren in ADPKD. This designation adds to the existing FDA orphan drug designation for Alport syndrome and supports the planned Phase 2 trial initiation in nonsense mutation ADPKD in 2027.