Topline results expected in first half of 2023

WATERTOWN, MA, USA I February 27, 2023 I Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the first patients have now been dosed in its Phase 2 study of ELX-02 for the treatment of Alport syndrome in patients with nonsense mutations. Topline results are expected in the first half of 2023.

“Patients with Alport syndrome with nonsense mutations lack disease modifying treatment options. We are pleased that we have taken learnings from our robust preclinical data and are exploring the potential of ELX-02 in a patient population that has been under served. With dosing initiated in our Phase 2 clinical trial, we remain poised to deliver topline clinical results from this trial in the first half of 2023,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.

This Phase 2 trial is targeting dosing of up to eight Alport syndrome patients with nonsense mutations in the COL4 gene. Patients will be dosed for two months with a three month follow-up. In addition to the primary endpoint of safety, the key secondary efficacy endpoint of proteinuria will be measured every two weeks. Treatment effect on proteinuria is a well-validated endpoint for several renal indications and a good predictor of treatment outcomes. For eligible patients, induction of COL4 will also be measured at the end of two months. Topline results are expected in the first half of 2023.

About Alport syndrome

Alport syndrome is a genetic disorder characterized by kidney disease with high levels of proteinuria, hearing loss and eye abnormalities caused by mutations in the genes (COL4A3, COL4A4, and COL4A5) needed for production of type 4 collagen. Approximately 6% to 7% of Alport syndrome patients, or approximately 9,400 to 12,750 individuals, are estimated to have nonsense mutations. These patients have significantly worse clinical outcomes than other Alport patients and have no disease modifying treatment options.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

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SOURCE: Eloxx Pharmaceuticals