Dec. 8 at 11:41 AM
$ARCT For Class 1 cystic fibrosis (CF) patients, they normally have severe disease, heavy mucus burden, chronic infections and fixed or severe lung damage. Existing cystic fibrosis therapies are generally ineffective for them due to mutations. Class 1 CF patients remain one of the most difficult to treat in the CF populations because the normal CFTR fixing drugs don’t apply.
In the second cohort of phase 2 study, dosing with Arcturus’ CF 032 therapy with 10mg for only 28 days, the study was designed to investigate safety, tolerability, and to help determine the right dose level for further studies. The treatment was generally safe and well tolerated. An important result from this study was the reductions in mucus plugs and volume in 4 of the 6 Class 1 patients.
Personally i think the cystic fibrosis 032 therapy of Arcturus "works" and is effective as shown in the evidence from the 4 out of the 6 Class 1 patients.
