Protalix Biotherapeutics Inc (NYSEMKT:PLX) reported positive data from its Phase 2 clinical study of alidornase alfa for the cure of Cystic Fibrosis. Sixteen subjects were enrolled in the trial, all of whom closed the trial. Alidornase alfa has been specifically designed to improve the enzyme’s efficacy in CF subjects.
Protalix Biotherapeutics reported that the Phase 2 study is a 28-day switchover trial to assess the efficacy and safety of alidornase alfa in CF subject previously cured with Pulmozyme®. Participation in the study was preceded by a 2-week failure period from Pulmozyme® before cure with alidornase alfa through inhalation.
The primary efficacy data represent that treatment with alidornase alfa led in clinically significant lung function development, as shown by a mean absolute jump in the ppFEV1 of 3.4 points over baseline. Also, a mean absolute jump in ppFEV1 of 2.8 points was seen in subjects participating in the study as against measurements taken from subjects at start before the switch to alidornase alfa from Pulmozyme®.
A commercially accessible small molecule CFTR modulator for CF treatment has recorded a mean absolute jump in ppFEV1 of 2.5 over baseline in registration clinical trial. This score was recorded while 74% of the subjects participating in the study of the CFTR modulator were treated with the modulator on Pulmozyme®. While this commercialized CFTR addresses specific mutation pertinent to less than 50% of patients with CF, alidornase alfa is being advanced to cure all CF patients.
Sputa accessible DNA samples were assessed for almost half of the subjects. A mean decline of more than 70% in DNA content over baseline was noted, and a mean decline of more than 90% from baseline was seen for sputa visco-elasticity. There was correlation between improvement in pulmonary function and sputa parameters. Protalix Biotherapeutics reported that PK analysis performed showed alidornase alfa is not taken into a patient’s circulatory system, indicating higher levels of alidornase alfa continues to be available in the subject’s lungs.