Israeli pharmaceutical company Protalix BioTherapeutics has announced 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, GCD, which is the same active substance as the company's approved enzyme replacement therapy, ELELYSO.
Oral GCD is an active form of human GCD 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 10th Annual WORLD Symposium, 10 February to 13 February 2014 in San Diego, California.