Protalix BioTherapeutics Inc. announced that the first Gaucher patient has been enrolled in the company’s Phase 2a clinical trial of oral glucocerebrosidase (GCD), or oral GCD (PRX-112), for the treatment of Gaucher disease. Oral GCD is the company’s proprietary plant cell expressed form of an active glycosylated glucocerebrosidase enzyme (GCD) that is naturally encapsulated within carrot cells and administered orally. The company expects the trial to be completed during the third quarter of 2014.
The Phase 2a trial is a 28 day open-label, sequential dose escalation study to evaluate the safety of oral PRX-112 and study the dose dependent pharmacokinetics of Oral GCD in 10 naïve adult Gaucher patients. Subjects will receive once daily oral administrations of oral GCD for five consecutive days at each dose, with a two-day washout period between doses.
In a Phase 1 study conducted year end 2013, oral GCD was found to be safe and well tolerated in all 16 patients across all of the three doses tested. There were no drug-related serious adverse reactions reported, and no patient discontinued the study prematurely. Presence of the GCD enzyme was detected in patients’ blood circulation and the enzyme demonstrated biological activity. With a daily oral administration of oral GCD, the company expects to achieve a steady state level of active GCD enzyme in the blood circulation of patients similar to the physiological state in healthy individuals.
“Following the completion of our Phase 1 trial of oral GCD last year, we were encouraged to see the clinical proof of concept demonstrated in the study and we look forward to continuing the development of an oral enzyme treatment for Gaucher patients,” commented Dr. David Aviezer, Protalix’s president and chief executive officer. “We believe oral delivery of GCD has the potential to improve patients’ quality of life and possibly clinical maintenance while maintaining the efficacy and safety of an enzyme treatment.”
Date: June 18, 2014
Source: Protalix BioTherapeutics
Filed Under: Drug Discovery