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Exaluren - Eloxx Pharmaceuticals

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Drug Profile

Exaluren - Eloxx Pharmaceuticals

Alternative Names: ELX-02; NB-124; Synthetic aminoglycoside

Latest Information Update: 19 Aug 2026

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At a glance

  • Originator Technion - Israel Institute of Technology
  • Developer Eloxx Pharmaceuticals
  • Class Amines; Aminoglycosides; Antifibrotics; Small molecules
  • Mechanism of Action Ribosomal protein modulators
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Cystic fibrosis; Mucopolysaccharidosis I; Hereditary nephritis; Rett syndrome; Cystinosis; Autosomal dominant polycystic kidney disease
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II Cystinosis; Hereditary nephritis
  • Preclinical Cystic fibrosis
  • No development reported Duchenne muscular dystrophy; Mucopolysaccharidosis I; Rett syndrome

Most Recent Events

  • 12 Aug 2026 Exaluren - Eloxx Pharmaceuticals receives Orphan Drug status for Hereditary nephritis in European Union
  • 12 Aug 2026 USFDA approves IND application for Exaluren in Hereditary nephritis
  • 12 Aug 2026 Eloxx Pharmaceuticals plans a phase IIb trial for Nonsense mutation alport syndrome in third quarter of 2026

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