Protalix BioTherapeutics Announces Positive Interim Results from Phase II Clinical Trial of AIR DNase™ for the Treatment of Cystic Fibrosis

Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced today positive interim results from the Company’s phase II clinical trial of alidornase alfa for the treatment of Cystic Fibrosis (CF) for the first 13 CF patients enrolled in the study.  Fifteen patients have been enrolled in, and are expected to complete, the study.  alidornase alfa is a plant cell expressed, chemically modified 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 switch-over study to evaluate the safety and efficacy of alidornase alfa in CF patients previously treated with Pulmozyme®.  Participation in the trial is preceded by a two-week washout period from Pulmozyme® before treatment with alidornase alfa via inhalation. The initial primary efficacy result shows that alidornase alfa improves lung function as demonstrated by a mean absolute increase in the percent predicted forced expiratory volume in one second (ppFEV1) of 4.1 points from baseline.  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 Pulmozyme® on top of the modulator.  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.Sputa available DNA samples were analyzed for approximately half of the patients.  A mean reduction of approximately 60% in DNA content from baseline was observed, and a mean reduction of approximately 90% from baseline was observed for sputa visco-elasticity.  This data provides further supportive evidence of improved lung function after treatment with alidornase alfa, as demonstrated by the increase in ppFEV1.No serious adverse events were reported, and all adverse events that occurred during the study were mild and transient in nature.“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,” commented Moshe Manor, Protalix’s President and Chief Executive Officer.  “We are looking forward to reporting full results from the study before the end of the first quarter of 2017.”“The preliminary efficacy results of alidornase alfa are very encouraging, even when compared to past trials of approved drugs for the treatment of CF.  Although the study was performed on a small number of patients, the data is very encouraging since it shows clinically meaningful results,” said Professor Eitan Kerem, Chairman of Pediatrics, Head of The Cystic Fibrosis Center, Hadassah University Hospital.  “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.”
About Protalix BioTherapeutics, Inc.Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®.  Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration (FDA) in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights.  Protalix’s development pipeline includes the following product candidates: PRX-102, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; PRX-106, an orally delivered anti-inflammatory treatment; PRX-110, a chemically modified DNase I for the treatment of Cystic Fibrosis; and others.
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Marcy Nanus
The Trout Group, LLC
646-378-2952
mnanus@troutgroup.com