Enrollment Nearly Complete for Phase 2 Clinical Trials Testing ELX-02 in CF Patients

Enrollment Nearly Complete for Phase 2 Clinical Trials Testing ELX-02 in CF Patients
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Enrollment is nearing completion for two Phase 2 clinical trials testing the experimental treatment ELX-02 in people with cystic fibrosis (CF), according to Eloxx Pharmaceuticals, the company developing ELX-02.

“We expect full enrollment to be achieved during the first quarter of 2020, and to report topline results during the first half of 2020,” Eloxx stated in a press release.

ELX-02 is a eukaryotic ribosomal selective glycoside, a small molecule that works on ribosomes — the cellular machineries that translate messenger RNA into proteins.

CF is caused by mutations in the CFTR gene, which codes for a protein of the same name. Some of these mutations, called nonsense mutations, result in a stop codon within the gene. The stop codon causes ribosomes to stop making the complete CFTR protein, resulting in a half-finished protein that cannot carry out its function and is quickly degraded by the cell.

ELX-02 binds to ribosomes, and helps them read through these premature stop codons. Preclinical data suggest that this allows for the production of fully-functioning CFTR protein. Moreover, ELX-02 has shown a good safety profile in Phase 1 clinical trials in healthy volunteers (NCT02807961, NCT03292302, and NCT03309605).

The therapy is currently being tested in two Phase 2 clinical trials in CF patients who have at least one copy of the G542X allele, a nonsense mutation. A total of 24 participants are expected to be recruited in the EL-012 trial (NCT04135495) in Massachusetts, U.S., and in the EL-004 trial (NCT04126473) in Israel.

Both trials are funded by Eloxx. In addition, the U.S.-based EL-012 trial received funding from the Cystic Fibrosis Foundation.

The trials are open-label, meaning participants are aware that they will receive the therapy (and not a placebo). Each participant will receive four ascending doses of ELX-02, administered by subcutaneous (under-the-skin) injection. A low dose (0.3 mg/kg per day) will be administered first, followed by a dose increase per administration (0.75 and 1.5 mg/kg per day), up to 3.0 mg/kg per day.

The trials’ primary goal is to determine the safety of ELX-02 in people with CF, as assessed by the presence of adverse reactions. ELX-02’s pharmacokinetics and pharmacodynamics — how the therapy moves through and is processed by the body — will also be assessed.

Secondary outcomes include efficacy metrics, such as sweat chloride concentration and measurements of lung function.

More information about trial recruitment can be found here for EL-012, and here for EL-004.

In addition, Eloxx is also conducting clinical trials testing ELX-02 in other conditions that involve an aberrant stop codon, namely cystinosis.

Cystinosis is caused by mutations, including some nonsense mutations, in the CTNS gene. This leads to a buildup of the amino acid cysteine in the body, resulting in widespread toxicity.

A Phase 2 clinical trial (NCT04069260) being conducted in Canada is evaluating ELX-02 in people with nephropathic cystinosis, a form of the disease that affects the kidneys.

Preliminary data showed a decrease in cysteine levels in blood samples from some patients after ELX-02 treatment, and suggested that the therapy has a good safety profile, with the only reported adverse events being mild reactions at the injections site.

Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
Total Posts: 336

Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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Marisa, a science writer, holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.
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