Eloxx Pharmaceuticals Inc. (NASDAQ: ELOX) stock declined by 14.77% in the current market trading. Eloxx Pharmaceuticals, Inc. is working on developing new ribosome modulating agents (RMAs) using its TURBO-ZM chemical technology platform and its portfolio of Eukaryotic Ribosome Selective Glycosides. ELX-02, Eloxx’s primary investigational drug candidate, is a small molecule therapeutic candidate that aims to restore full-length functional protein synthesis.
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What is happening?
Eloxx Pharmaceuticals released an enrollment update for the current Phase 2 study of ELX-02 in individuals having at least one G542X allele for cystic fibrosis (CF). Eloxx intends to release data from the first four treatment arms in Q4 of 2021, based on enrolment thus far.
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Eloxx has enlisted a significant number of patients to determine the biological activity of ELX-02 as of the end of June 2021. ELX-02 is now being tested in CF patients with nonsense mutations in the CFTR gene in Phase 2 clinical studies.
Subsequently, a fifth treatment arm was added to the study to ensure the adequacy of ELX-02 in conjunction with Kalydeco (ivacaftor) which is an FDA-approved CFTR potentiator for cystic fibrosis in patients with at least one CF gene mutation susceptible to ivacaftor. The program is primarily funded by the Cystic Fibrosis Foundation. The US Food and Medication Administration has designated ELX-02 as an orphan drug for the treatment of cystic fibrosis.
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Sumit Aggarwal, President and Chief Executive Officer stated,
They have made significant progress in enrolling patients in their current Phase 2 clinical study to investigate the efficacy of ELX-02 to treat cystic fibrosis, due to the hard work of their clinical team. As a result of this accomplishment, they expect to present results from the study’s first four treatment arms in the fourth quarter of this year.