Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results For Oral GCD In Gaucher Disease Patients

CARMIEL, Israel, Oct. 14, 2013 (GLOBE NEWSWIRE) -- Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced today initial positive results from its phase I clinical trial of oral glucocerebrosidase (GCD), or Oral GCD (PRX-112), in patients with Gaucher disease. In the trial, Oral GCD was well-tolerated, and active enzyme was detected in patients' blood circulation. Oral GCD is an orally available form of the plant cell-expressed enzyme, glucocerebrosidase or GCD, which is the same active substance as the Company's approved enzyme replacement therapy, ELELYSO™. Oral GCD is an active form of human glucocerebrosidase which is naturally encapsulated within the carrot cells in which it is produced. 

The phase I clinical trial is an open label safety and pharmacokinetic study designed to assess the delivery of prGCD after oral administration of Oral GCD in Gaucher patients. Patients received one of three doses of re-suspended carrot cells in a single oral administration during the first segment of the trial and three consecutive daily administrations during the second segment of the trial. The study is being conducted in two sites; at Shaare Zedek Medical Center, Jerusalem, Israel, and at the Rambam Medical Center, Haifa, Israel.

Overall, oral GCD was found to be safe and well tolerated in all 12 patients across all of the three doses tested. There were no serious adverse reactions reported and no patient discontinued the study prematurely. Presence of an enzyme was detected in patients' blood circulation and the enzyme demonstrated biological activity. In addition, some of the patients who suffered from thrombocytopenia and had low platelet counts demonstrated a meaningful improvement in platelet count. Accordingly, the trial has been extended to enroll and evaluate additional Gaucher patients with low platelet counts. The amended study is expected to conclude during the fourth quarter of 2013. Additional data from the phase I study will be presented at the 10 th Annual WORLD Symposium, February 10-13, 2014 in San Diego, CA.

"Oral administration of proteins has been a long time challenge for the biopharmaceutical industry due to the early degradation of proteins in the digestive track. The results of this phase I trial provide compelling proof of concept data for the oral delivery of enzymes using plant cells as a delivery platform," commented Prof. Ari Zimran, Associate Professor and Director of the Gaucher Clinic at Shaare Zedek and Principal Investigator. "Oral delivery of GCD has the potential to dramatically change the way Gaucher patients are treated—not only improving patients' quality of life by eliminating the need for bi-weekly infusions, but perhaps also having clinical benefit due to the steady maintenance of enzyme levels in patients' blood circulation."

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