ELX-02 received Fast Track and Orphan Drug Designations from the US Food and Drug Administration to treat cystic fibrosis patients with nonsense mutations. Credit: Jerry Nick, M.D. / commons.wikimedia.org.

Eloxx Pharmaceuticals has dosed the first participant in its Phase II clinical trial of ELX-02 to treat Alport syndrome patients with nonsense mutations.

Up to eight Alport syndrome patients with nonsense mutations in the COL4 gene are planned to be dosed in the Phase II clinical trial.

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In the study, participants will be dosed for two months and will have a three-month follow-up.

Induction of COL4 will also be measured for eligible patients at the end of two months.

The trial’s important secondary efficacy endpoint of proteinuria, along with its primary endpoint of safety, will be measured every two weeks.

The company expects to receive topline data from the trial in the first half of this year.

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Eloxx Pharmaceuticals president and CEO Sumit Aggarwal said: “Patients with Alport syndrome with nonsense mutations lack disease modifying treatment options.

“We are pleased that we have taken learnings from our robust preclinical data and are exploring the potential of ELX-02 in a patient population that has been under served.

“With dosing initiated in our Phase II clinical trial, we remain poised to deliver topline clinical results from this trial in the first half of 2023.”

An investigational small molecule drug candidate, ELX-02, has been designed for restoring the full-length functional proteins’ production.

It received Fast Track Designation and Orphan Drug Designation from the US Food and Drug Administration (FDA) to treat cystic fibrosis (CF) patients with nonsense mutations.

ELX-02 has also received orphan medicinal product designation from the European Commission.

The drug candidate is in clinical development, with a focus on cystic fibrosis.