Eloxx Pharmaceuticals, Inc.

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company that develops new, small molecule drug candidates. These candidates are designed to modify the ribosome and encourage the ribosome to "read through" premature stop codons caused by nonsense mutations, allowing for the production of full-length proteins. This approach of targeting ribosome subunits offers a way to treat various genetic diseases. Nonsense mutations are responsible for approximately 10% to 12% of patients with a specific genetic disease. There are over 7,000 inherited genetic diseases affecting 350 million people globally. The company's immediate priority is to advance the clinical development of its main product candidate, exaluren, for rare kidney diseases. Additionally, through a collaboration with Almirall, S.A., it is developing ZKN-013 for rare skin diseases. Exaluren is a new eukaryotic ribosome-selective glycoside (ERSG) candidate designed to induce premature termination codon (PTC) readthrough in the presence of nonsense mutations, aiming to restore full-length proteins. Eloxx Pharmaceuticals believes this mechanism could be applicable to all genetic diseases caused by nonsense mutations. Initially, exaluren is being developed to induce full-length Collagen IV protein for the treatment of Alport syndrome with nonsense mutations (NMAS). NMAS is a genetic kidney disorder, a subset of Alport syndrome, caused by the loss of one of three Collagen IV subtypes: Collagen IV alpha 3 (COL4A3), alpha 4 (COL4A4), or alpha 5 (COL4A5), leading to the loss of the Collagen IV alpha 3,4,5 trimer. This trimer is produced in podocyte cells and is essential for the kidney's filtration structure, which includes the glomerular basement membrane (GBM), foot processes (FPs), and slit diaphragms. Eloxx Pharmaceuticals estimates that NMAS affects approximately 6% to 7% of Alport syndrome patients, or about 5,000 patients in the United States and approximately 14,000 patients in developed countries and China. NMAS is characterized by blood (hematuria) and protein (proteinuria) in the patient's urine, leading to progressive kidney disease, hearing loss, and eye abnormalities.