Eloxx Resumes Enrollment in Phase 2 Trial of ELX-02 in Europe, Israel

Eloxx Pharmaceuticals has resumed enrollment in its Phase 2 clinical trial evaluating ELX-02 in cystic fibrosis (CF) patients in Israel and Europe, after it was temporarily halted due to the COVID-19 pandemic. 

“We are pleased that enrollment in our Phase 2 clinical trial in cystic fibrosis has been resumed in Israel and Europe, however, our U.S. trial remains temporarily paused due to the COVID-19 pandemic as the health and safety of our employees, healthcare workers, and patients must be our highest priority during this crisis,” Gregory Williams, PhD, CEO of Eloxx, said in a press release. 

ELX-02 is an investigational therapy targeting nonsense mutations in the CFTR gene, which cause CF. These mutations create a premature stop signal in the gene, leading to the production of a shorter CFTR protein that is quickly degraded by the cell.

ELX-02 works by instructing the ribosome — structures in the cell responsible for synthesizing proteins — to skip over the stop signal. Theoretically, this enables the production of sufficient amounts of full-length CFTR protein.

Before the suspension, Eloxx had been enrolling patients with at least one copy of the G542X mutation, the most common nonsense mutation in CF patients, in its two open-label, dose-ascending Phase 2 trials called EL-004 (NCT04126473) and EL-012 (NCT04135495). 

EL-004 aimed to recruit up to 16 participants in Germany and Israel, while EL-012 was recruiting up to eight participants in the U.S. The goal of the trials is to evaluate the safety and tolerability of multiple-ascending doses of ELX-02 in CF patients with the G542X mutation. 

ELX-02’s pharmacokinetics and pharmacodynamics — how the therapy moves through and is processed by the body — are also being assessed in these trials. 

Earlier this year, enrollment for the two Phase 2 trials was nearly complete. However, in light of the COVID-19 pandemic, Ellox decided to temporarily pause trial enrollment in March 2020. The decision was made to avoid unnecessary exposure of CF patients, who are more at risk of severe illness if they contract COVID-19.

Now, enrollment for the EL-004 trial in Germany and Israel has reopened.

“We have thoughtfully re-engaged with our clinical sites and investigators in Europe and Israel, and we continue to work closely with our clinical sites and investigators in the U.S.,” Williams said.

Eloxx initially anticipated reporting top-line data from the trials during the first half of 2020.

“We remain committed to completing enrollment in our Phase 2 proof of concept clinical trial program and reporting top line data as soon as feasible,” Williams added. “We will provide further updates as they become available.”