Date: 2014-06-18
Type of information: Initiation of patient enrollment
phase: 2a
Announcement: enrollment of the first patient
Company: Protalix BioTherapeutics (Israel)
Product: PRX-112 (oral glucocerebrosidase)
Action
mechanism: PRX-112 is an enzyme replacement therapy (ERT). The compound is a plant cell expressed form of the glucocerebrosidase enzyme (GCD) that is naturally encapsulated within carrot cells and administered orally.
Disease: Gaucher disease
Therapeutic area: Rare diseases - Genetic diseases
Country:
Trial
details: The phase IIa trial is a 28 day open-label, sequential dose escalation study to evaluate the safety of oral PRX-112 and study the dose dependant pharmacokinetics of Oral GCD in 10 naive 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.
Latest
news: * On June 18, 2014, Protalix BioTherapeutics announced that the first Gaucher patient has been enrolled in the Company\'s phase IIa clinical trial of oral glucocerebrosidase (GCD), or oral GCD (PRX-112), for the treatment of Gaucher disease. The Company expects the trial to be completed during the third quarter of 2014.
In a phase I 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.
