Protalix Announce Positive Results Alidornase Alfa Phase II Trial

Protalix BioTherapeutics, Inc.,a biopharmaceutical company, announced positive results from the company's phase II clinical trial of alidornase alfa for the treatment of cystic fibrosis (CF). Sixteen patients were enrolled in the study, all of whom completed the study. alidornase alfa is a plant cell-expressed, chemicallymodified recombinant DNase enzyme resistant to inhibition by actin, which the company has specifically designed to enhance the enzyme's efficacy in CF patients.

The phase II trial is a 28-day switchover study to evaluate the safety and efficacy of alidornase alfa in CF patients previously treated with Pulmozyme (currently the only commercially available DNase therapy). Participation in the trial was preceded by a two-week washout period from Pulmozyme before treatment with alidornase alfa via inhalation. The primary efficacy results show that treatment with alidornase alfa resulted in clinically meaningful lung function improvement, as demonstrated by a mean absolute increase in the percent predicted forced expiratory volume in one second (ppFEV1) of 3.4 points from baseline. Moreover, a mean absolute increase in ppFEV1 of 2.8 points was also observed in patients participating in the trial when compared to measurements taken from patients at initiation before the switch from Pulmozyme to alidornase alfa.

A commercially available small molecule CFTR modulator for the treatment of CF has reported a mean absolute increase in ppFEV1 of 2.5 from baseline in its registration clinical study. This score was achieved while 74% of the patients participating in the trial of the CFTR modulator were also treated with the modulator on top of Pulmozyme. While this marketed CFTR addresses a certain mutation applicable to less than 50% of CF patients, alidornase alfa is being developed to treat all CF patients.