Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates - Seite 3

Initiated Phase 1 Single Ascending Dose (SAD) study: Initiated Phase 1 SAD study in Australia with initial results expected in 2^nd half of 2024

Recently Published Paper on Autosomal Dominant Polycystic Kidney Disease (ADPKD) may Have Other Rare Kidney Disease Applications

A paper was published in April 2024, on ADPKD titled Genetics of cystogenesis in base-edited human organoids reveal therapeutic strategies for polycystic kidney disease, authored by Courtney E. Vishy, Chardai Thomas, Thomas Vincent, Daniel K. Crawford, Matthew M. Goddeeris, and Benjamin S. Freedman. Freedman and colleagues engineered base-edited human iPS cells to resolve the functional genetics of human PKD. They discovered that cyst formation in diseased organoids could be prevented with a single healthy gene copy. This insight enables the demonstration of both genetic and pharmacological therapeutic approaches for clinically relevant nonsense mutations and may have the potential to support other rare kidney diseases.

About Nonsense Mutation Alport Syndrome

Nonsense Mutation Alport syndrome (NMAS) is an ultra-rare Type IV Collagenopathy characterized by nonsense mutations in the genes (COL4A3, COL4A4, and COL4A5) that result in a less than full length (truncated) Type 4 Collagen. This disorder mostly affects children with a median age at diagnosis of 9 to 20 years. It is characterized by rapid and progressive damage to the kidneys, ear, and eyes, starting with worsening of kidney morphology to proteinuria and finally kidney failure, hearing loss and eye abnormalities. 90% NMAS patients progress to kidney failure and hearing loss before the age of 30. There are no approved therapies. It is estimated that there are approximately 11,000 NMAS patients in the US and >20,000 patients in US, Europe, Japan and China.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM 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. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations. For more information, please visit www.eloxxpharma.com.