ELX-02 + ivacaftor

**ELX-02 + ivacaftor** is a combination of ELX-02, a synthetic aminoglycoside designed to induce translational readthrough of premature stop codons (nonsense mutations) in mRNA, and ivacaftor, a small molecule potentiator of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The combination was developed for the treatment of cystic fibrosis (CF) patients carrying class I nonsense mutations in the CFTR gene, particularly the G542X mutation. ELX-02 works by enabling ribosomal readthrough of premature termination codons, potentially restoring the synthesis of full-length, functional CFTR protein. Ivacaftor increases the open probability (gating) of the CFTR chloride channel, improving chloride transport in responsive mutant proteins. The combination aims to restore both the presence and function of CFTR in patients with nonsense mutations. The primary developer is Eloxx Pharmaceuticals. In clinical trials, the combination was well tolerated but did not achieve statistical significance for key efficacy endpoints in phase 2 studies of class I CF patients; activity was observed in specific subgroups. The combination has been studied via subcutaneous administration of ELX-02 and oral ivacaftor[1][3][5][7].

Other names

ELX-02/ivacaftor