Potential Treatment for Cystic Fibrosis Displays Encouraging Interim Data

According to a story from Drug Development Technology, the drug developer Translate Bio has recently reported interim results from its phase 1/2 clinical trial. This trial is testing the company’s investigational product candidate MRT5005, which is being developed as a treatment for cystic fibrosis, a rare disorder that affects breathing. The drug is designed to address the underlying disease mechanism regardless of which mutation the patient has.

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.

The Trial So Far

The clinical trial included a single ascending dose portion which measured patient response to 8mg, 16mg, and 24mg doses. This portion included results from a total of 12 patients.

As far as safety was concerned, MRT5005 was generally found to be well tolerated, particularly at the low to medium range of doses. There were no serious adverse events reported during the study. The most common negative side effects were headaches and coughing. The most notable efficacy findings appeared in the 16mg dose group. These three patients saw a mean increase maximum of 15.7 percent from baseline forced expiratory volume. In the 24mg group, one patient saw a 21.4 percent increase but they others experienced no meaningful changes.

The next portion of the trial will be multiple dose ascending and will include both 12mg and 20mg dose levels based on results from the single dose portion.

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