Cystic fibrosis digest - 2021

Cystic fibrosis digest - 2021

Eloxx Pharmaceuticals enrollment update

ELX-02 (Eukaryotic ribosomal selective glycoside) – Eloxx Pharmaceuticals

  • Trial details: Eloxx Phase 2 program, Cystic Fibrosis

NCT04135495

NCT04126473

N=8

N=16

Locations: US

Locations: Australia, Germany, Israel

Arm 1: ELX-02 0.3 mg/kg

Arm 2: ELX-02 0.75 mg/kg

Arm 3: ELX-02 1.5 mg/kg

Arm 4: ELX-02 up to 3 mg/kg

Arm 5: ELX-02 + Kalydeco (ivacaftor)

  • Based on enrollment to date, Eloxx expects to present data from the first four treatment arms of the study in Q4’21
  • ELX-02 received US Orphan Drug Designation from FDA and EMA for cystic fibrosis (For full story click here)

Eloxx to present data from the first four treatment arms from its ELX-02 Phase 2 program in Q4’21

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CI Scientists Commentary:

  • About ELX-02: It is a eukaryotic ribosomal selective glycoside (ERSG) designed to increase the read-through activity in patients with nonsense mutations and enable the production of sufficient amounts of full-length functional protein to restore activity
  • Currently, there are no approved therapies to treat CFTR nonsense mutations. G542X Nonsense allele frequency accounts for 37%
  • Expanding cystic fibrosis pipeline, Eloxx is developing macrolide-based ribosome modulating agents (RMAs) in patients with nonsense mutations ensuring a broad approach for all nonsense allele mutations

– Dr. Kowndinya, CI Scientists

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