Currently, our lead investigational drug, ELX-02, is a eukaryotic ribosomal selective glycoside (ERSG) designed to restore production of functional proteins, suppress nonsense mutations, and overcome nonsense-mediated decay without the limiting toxicities of aminoglycosides. ELX-02 is in Phase 2 clinical trials in cystic fibrosis patients with diagnosed nonsense mutations on one or both alleles. There is a high-unmet medical need among these patients, as they have a high burden of disease, and few, if any, treatment options available.
Our library of novel small molecule eukaryotic ribosomal selective glycoside (ERSG) compounds has been designed to treat rare and ultra-rare premature stop codon diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins, a process known as translation. We believe these molecules hold the potential to be disease-modifying therapies that may change the course of hundreds of genetic diseases and improve the lives of patients with premature stop codon diseases caused by the nonsense mutations.
Our research and development strategy targets rare or ultra-rare diseases where: a high unmet medical need exists, identified nonsense mutation-bearing patient population is established, preclinical read-through can be established in predictive personalized medicine models, and a defined path through Orphan Drug development, regulatory approval, patient access and commercialization is identifiable. We believe patient advocacy to be an important element of patient focused drug development and seek opportunities to collaborate with patient advocacy groups throughout the discovery and development process. Our investigational drug product candidate, ELX-02, is in Phase 2 proof of concept clinical trials in cystic fibrosis.