Eloxx Pharmaceuticals, Inc.

Eloxx Pharmaceuticals, Inc. (OTC Expert Market: ELOX) is a clinical-stage biopharmaceutical company focused on developing novel small molecule product candidates designed to modulate the ribosome and promote readthrough of premature stop codons induced by nonsense mutations. The company's website is www.eloxxpharma.com.

The company's lead product candidate is exaluren, a novel eukaryotic ribosome-selective glycoside designed to induce premature termination codon readthrough and promote restoration of full-length proteins. Exaluren is being developed primarily for the treatment of Alport syndrome with nonsense mutations, a rare genetic kidney disorder affecting approximately five thousand patients in the United States. The company is also developing exaluren for autosomal dominant polycystic kidney disease in patients with nonsense mutations, with preclinical organoid and cellular models demonstrating increased protein expression and reduced cyst formation following treatment.

Through a collaboration with Almirall, S.A., Eloxx is developing ZKN-013, an oral ribosome modulating agent, for the treatment of rare dermatological diseases including recessive dystrophic epidermolysis bullosa and junctional epidermolysis bullosa caused by nonsense mutations. Under the Almirall License Agreement, the company received an upfront payment of three million dollars and a development milestone of three million dollars in 2024, with eligibility for up to approximately four hundred seventy million dollars in additional development, regulatory, and commercial milestone payments.

Exaluren has been tested in eight clinical trials involving one hundred forty-five subjects total, with no dose-limiting toxicities or serious adverse events attributed to the compound reported. The company conducted a Phase 2a proof-of-concept trial in three patients with autosomal recessive Alport syndrome, demonstrating improvements in kidney filtration architecture and Collagen IV protein restoration. Eloxx plans to initiate a Phase 2b clinical trial in the first half of 2026 with anticipated topline data by mid-2027.

The company's intellectual property portfolio includes sixty-six issued patents and one hundred one pending patent applications covering exaluren and over forty other read-through inducing compounds, with primary composition of matter coverage derived from an exclusive license with Technion Institute for Research and Development Ltd.