Protalix BioTherapeutics’ AIR DNase (alidornase alfa), developed to make mucus in the lungs of cystic fibrosis (CF) patients less sticky, showed remarkably good results in an early analysis of a small, proof-of-concept Phase 2 trial (NCT02722122).
The inhaled drug is a DNase, an enzyme that works to chop up DNA present in the mucus of CF patients. AIR DNase is a chemically altered enzyme that is harvested from plant cells.
The trial specifically enrolled only patients who had previously been treated with Pulmozyme (dornase alpha). All stopped using Pulmozyme two weeks before the 28-day study. In total, the trial has enrolled 15 people and the current analysis included the first 13 who completed the study.
Comparing trial data so far with reported data on a currently approved CFTR modulator, the new drug showed larger improvements in lung function, the company announced in a press release. The effects of the two modulators were not directly compared in this study, and Protalix did not identify which CFTR modulator it was referring to in these data comparisons.
Lung function was measured by percent predicted forced expiratory volume in one second (ppFEV1), which increased by 4.1 points from study start among the 13 patients. In comparison, the approved CFTR modulator reported improved lung function of 2.5 points in the clinical trial that formed the basis of its regulatory approval, a result achieved while 75 percent of patients were also treated with Pulmozyme.
Researchers also analyzed the DNA content of the mucus in about half the patients, and reported around 60 percent less DNA evident after treatment. The visco-elasticity, or thickness, of the mucus was reduced by 90 percent.
Protalix also pointed out that its AIR DNase is designed to treat all CF patients, regardless of underlying mutation.
“We are enthusiastic about the data generated in this trial as we were able to see meaningful improvements in efficacy in a way that have not been reported for a long time in the challenging CF space,” Moshe Manor, Protalix’s president and CEO, said in the release. “We are looking forward to reporting full results from the study before the end of the first quarter of 2017.”
Patients in the trial did not experience any serious adverse events, with those noted described as mild and brief.
“Although the study was performed on a small number of patients, the data is very encouraging since it shows clinically meaningful results,” said Eitan Kerem, a professor and chairman of Pediatrics, and head of The Cystic Fibrosis Center at Hadassah University Hospital in Israel. “I look forward to following the results of upcoming trials of alidornase alfa. If the data continues to be as positive, clearly alidornase alfa will be a key treatment for all CF patients.”