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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: 21 Apr 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; Rett syndrome; Hereditary nephritis; Cystinosis
  • 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

  • 13 Apr 2026 Eloxx Pharmaceuticals plans a phase II trial for Hereditary nephritis (In children, In adolescents, In adults) in the USA and the United kingdom(SC) in May 2026 , (NCT07523581)
  • 12 Nov 2024 Pharmacodynamics data from Phase-II trial in Hereditary nephritis released by Eloxx Pharmaceuticals
  • 11 Jul 2024 Eloxx Pharmaceuticals completes pre-Investigational New Drug Application (PIND) meeting with the US FDA in USA to initiate a phase-II clinical trial for Hereditary nephritis

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