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 ribosomes and encourage the ribosome to "read through" premature stop codons caused by nonsense mutations, allowing for the production of full-length proteins. This approach targets ribosome subunits 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 primary goal is to advance the clinical development of its lead product candidate, exaluren, for rare kidney diseases. Additionally, through a collaboration with Almirall, S.A., Eloxx is developing ZKN-013 for rare skin diseases. Exaluren is a new eukaryotic ribosome-selective glycoside product candidate engineered to induce premature termination codon readthrough in the presence of nonsense mutations, aiming to restore full-length proteins. This mechanism is believed to be applicable to all genetic diseases caused by nonsense mutations. Initially, Eloxx Pharmaceuticals is developing exaluren to induce full-length Collagen IV protein for the treatment of Alport syndrome with nonsense mutations (NMAS). NMAS is a genetic kidney disorder resulting from the loss of one of three Collagen IV subtypes (Collagen IV alpha 3, alpha 4, or alpha 5) and the subsequent loss of the Collagen IV alpha 3,4,5 trimer. This trimer is essential for the kidney's filtration structure. The company estimates that NMAS affects roughly 6% to 7% of Alport syndrome patients, or approximately 5,000 patients in the United States and approximately 14,000 patients in developed countries and China. NMAS is characterized by blood and protein in the urine, leading to progressive kidney disease, hearing loss, and eye abnormalities.