Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients

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Eloxx Pharmaceuticals Announces Final Data Assessment from Phase 2 Combination Clinical Trial of ELX-02 in Class 1 Cystic Fibrosis Patients

Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX ), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the final data assessment from the Phase 2 clinical trial of ELX-02 in combination with ivacaftor in Class 1 CF patients with at least one nonsense mutation. In the final assessment, ELX-02 demonstrated clinically relevant improvement in ppFEV1. The final data assessment includes a reanalysis using change in ppFEV1 from Day 1 instead of baseline, as multiple patients experienced disease progression between screening and treatment. Initial topline results from this trial were reported in September 2022.

“We believe that the clinical improvements with ELX-02 observed in a trial of CF patients with severe illness, coupled with remission observed in one patient in the ongoing Phase 2 trial of ELX-02 in Alport syndrome patients with nonsense mutations, reinforces our belief in the disease-modifying potential of ELX-02 and warrants further development. Similar to the patients in this CF trial, participants in our Phase 2 Alport syndrome trial also had highly progressive autosomally recessive disease, making a remission in even one patient highly clinically significant. The body of data strongly validate our decision to advance ELX-02 into a pivotal study in Alport syndrome,” said Sumit Aggarwal, President and CEO of Eloxx.

Mr. Aggarwal continued, “The high concentrations of ELX-02 observed in the kidney in those with Alport Syndrome make the disease an ideal target for further study, as the ELX-02 exposure in the Alport study is at least 25-fold higher than in the CF trial.”

Final Assessment of ELX-02 Phase 2 Combination Trial in Class 1 CF Patients

The Phase 2 combination clinical trial of ELX-02 was designed to evaluate safety and assess biological activity in G542X nonsense mutation Class 1 CF patients as monotherapy and in combination with ivacaftor after 5 weeks of treatment. Results from the final analysis of 13 patients evaluable, versus 11 at time of initial assessment are summarized below:

  • 6 of 13 patients entered trial from monotherapy arm (after average 463 days) and had a decrease in lung function (annualized -4.26% reduction in ppFEV1) due to disease progression.
  • Treatment with ELX-02 stabilized disease overall and resulted in a clinically relevant increase in ppFEV1 in six of thirteen patients based on change in ppFEV1 at the end of treatment at Day 35 compared to the start of treatment at Day 1.
Number or patientsChange in ppFEV1 (%) at end of treatment at Day 35 vs Day 1Change in ppFEV1 (%) at Safety follow up vs Day 35
Overall, n=13-0.31%-2.69%
Responders, n=6+2.83%-5.83%

  • Topline data previously confirmed biological activity. Patients with higher baseline sweat chloride levels had increased responses to treatment as indicated by sweat chloride concentration (p=0.00013 at Day 35).
  • ELX-02 was generally well tolerated in the trial, with no treatment-related serious adverse events noted.

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