Data From Phase 2 Trials of ELX-02 Expected This Year

Marisa Wexler MS avatar

by Marisa Wexler MS |

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Eloxx Pharmaceuticals announced it expects to share data later this year from the first four treatment groups of a Phase 2 program evaluating the investigational therapy ELX-02 in people with cystic fibrosis (CF) who have at least one G542X mutation.

“Given the substantial efforts of our clinical team, we have made considerable progress in enrolling patients in our ongoing Phase 2 clinical program to explore the potential of ELX-02 to treat cystic fibrosis. Given this progress, we are positioned to present data from the first four treatment arms of the study in the fourth quarter of this year,” Sumit Aggarwal, Eloxx’s president and CEO, said in a press release.

CF is caused by mutations in the CFTR gene. ELX-02 is designed to treat people with CF caused by certain types of mutations, called nonsense mutations. G542X is the most common CF-causing nonsense mutation.

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A nonsense mutation causes a “stop” signal in the middle of a gene — sort of like a period in the middle of a sentence. When the cell’s protein-making machinery “reads” the gene, it stops at the early signal, producing a truncated protein that is rapidly degraded by the cell. ELX-02 is designed to allow the cell’s protein-making machinery to “read through” the erroneous stop signal and make a full-length protein, improving the transport of water and chloride ions in and out of cells.

ELX-02 is being evaluated in CF patients who have at least one G542X mutation in two open-label Phase 2 clinical trials, EL-004 (NCT04126473) and EL-012 (NCT04135495), both sponsored by Eloxx. In open-label trials, the participants and researchers know what treatments are being given, so no placebo is used.

Participants in both trials are being given four escalating daily doses of ELX-02, from 0.3 mg/kg to as high as 3 mg/kg. The medication is administered by subcutaneous (under-the-skin) injection.

The studies’ main goals are to assess the investigational medicine’s safety profile and pharmacological properties. The effect of treatment on participants’ lung function and sweat chloride levels also will be assessed.

So far, data have shown no serious adverse side effects related to treatment with ELX-02, according to Eloxx.

The two trials are actively recruiting participants at sites in the U.S. and in Australia, Germany, and Israel.

Eloxx recently announced the addition of a fifth treatment group to the program, which will be assessing ELX-02 in combination with the approved therapy Kalydeco (ivacaftor) for patients with amenable mutations.


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