Date: 2013-10-14
Type of information:
phase: 1
Announcement: results
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
Country: Israel
Trial details: 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 12 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.
Latest
news: * On October 14, 2013, Protalix BioTherapeutics has announced initial positive results from its phase I clinical trial of oral glucocerebrosidase (GCD), PRX-112, in patients with Gaucher disease. In the trial, oral glucocerebrosidase was well-tolerated, and active enzyme was detected in patients' blood circulation. 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, February 10-13, 2014 in San Diego.* On April 2, 2013, Protalix BioTherapeutics has announced that the first patient has been treated in the phase I clinical trial of PRX-112, the Company's orally-administered enzyme product candidate for the treatment of Gaucher disease. Protalix expects the phase I trial to be completed during the third quarter of 2013.
* On March 7, 2013, Protalix BioTherapeutics has announced that it has received approval from the Israeli Ministry of Health to initiate a phase I clinical trial of PRX-112, or Oral GCD, the Company\'s orally-administered product candidate for the treatment of Gaucher disease. The company anticipates initiating this trial in two Israeli medical centers during the next month.
Protalix expects the phase I trial to be completed during the second quarter of 2013.
Pre-clinical studies of oral GCD demonstrate the stability of the enzyme in the carrot cell and the capacity of the cell\'s cellulose wall to protect the enzyme against degradation in the digestive tract in an in-vitro model of the stomach and intestines. Additionally, both rats and pigs fed with PRX 112, lyophilized carrot cells expressing GCD, have demonstrated enzyme levels in the plasma and accumulation of the active enzyme in target organs such as the spleen and liver.
