According to a story from Cystic Fibrosis News Today, Eloxx Pharmaceuticals had just announced recently that it is officially re-opening enrollment for its phase 2 clinical study. This trial will be evaluating the company’s investigational therapy known as ELX-02, which is being developed as a treatment for cystic fibrosis, a rare lung disorder. Enrollment for the trial was brought to an abrupt halt earlier in the year because of the ongoing coronavirus/COVID-19 pandemic.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
A Comeback from COVID
Enrollment will proceed in both Israel and Europe. Representatives from Eloxx stated that enrollment for the US portion of the trial will continue to remain closed until further notice as the region remains more severely affected by the virus than Europe or Israel. The trial currently is aiming to recruit 16 participants from these areas.
ELX-02 is being designed to target the nonsense mutations present in the CFTR gene that are the most common cause of the disorder. The mechanism of action intervenes in the activity of the ribosome, a cellular organelle which plays a critical role in synthesizing different proteins. The mutation causes a shortened version of the CFTR protein to be created, which degrades too quickly; ELX-02 is supposed to restore the ability to produce the normal, full-length version.
There have been some significant advances in the treatment of this disorder in recent years. As more therapies become available, patients will have more opportunities than ever to manage their symptoms effectively.