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Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) is a clinical-stage biopharmaceutical company focused on the development of novel RNA-modulating drug candidates designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are genetic mutations that disrupt protein synthesis, leading to severe genetic disorders. With over 1,800 identified rare diseases caused by these mutations, Eloxx is at the forefront of innovative genetic therapies.
The company's lead product candidate, ELX-02, is a small molecule drug designed to restore production of full-length functional proteins. Currently in Phase 2 clinical development, ELX-02 has shown promise in treating Alport syndrome, a genetic disorder characterized by kidney disease, hearing loss, and eye abnormalities.
Recent advancements include confirmation of ELX-02's disease-modifying potential in Alport syndrome, with positive results from patient biopsies in clinical trials. Eloxx has also submitted an Investigational New Drug (IND) application to the U.S. FDA for ELX-02, aiming to include U.S.-based sites in its pivotal trials.
Additionally, Eloxx is developing ZKN-013, a TURBO-ZM™ based molecule for treating recessive dystrophic epidermolysis bullosa (RDEB) and other genetic conditions. The company has initiated strategic partnerships and aims to begin patient dosing by the end of 2023.
Financially, Eloxx reported a net loss of $3.6 million for Q3 2023 but has shown a decrease in overall R&D and administrative expenses compared to the previous year. The company is focused on improving its liquidity position and raising additional capital to fund operations through 2023 and beyond.
Eloxx continues to leverage its TURBO-ZM™ chemistry platform to develop novel Ribosome Modulating Agents (RMAs) and maintain its commitment to addressing the unmet medical needs of patients with rare genetic disorders. For the latest updates and detailed information, visit www.eloxxpharma.com.
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