Protalix Biotherapeutics, Inc. Treats First Gaucher Patient in Phase I Study With PRX-112, an Orally-Administered Enzyme Treatment of Gaucher Disease
Published: Apr 02, 2013
“With Oral GCD, we are using the natural characteristics of plant cells to deliver active enzyme into the patients blood stream,” said Professor Ari Zimran, M.D., Director of the Gaucher Clinic in Shaare Zedek Medical Center, Jerusalem, Israel and lead clinical investigator. “We believe oral delivery of GCD has the potential to improve patients’ quality of life without compromising the efficacy or safety of the treatment.”
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. Subjects receive re-suspended carrot cells in a single oral administration during the first cohort of the trial and three consecutive daily administrations during the second cohort of the trial. 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.
Dr Einat Brill Almon, the Company's Senior Vice President, Product Development, added: “We successfully demonstrated the ability of plant cells to act as an oral delivery mechanism for a number of therapeutic proteins in our animal studies. We believe that the results of our clinical trial of Oral GCD will provide additional support for our belief that this oral delivery mechanism can be developed for other proteins used to treat other indications.”
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell based expression system, ProCellEx®. Protalix's unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration (FDA) in May 2012, by Israel's Ministry of Health in September 2012 and by the Brazilian National Health Surveillance Agency (ANVISA) in March 2013. It also has been approved in Uruguay. Marketing applications for taliglucerase alfa have been filed in additional territories as well. Protalix has partnered with Pfizer Inc. for the worldwide development and commercialization of taliglucerase alfa, excluding Israel, where Protalix retains full rights. Protalix's development pipeline also includes the following product candidates: PRX-102, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; PRX-105, a pegylated recombinant human acetylcholinesterase in development for several therapeutic and prophylactic indications, a biodefense program and an organophosphate-based pesticide treatment program; an orally-delivered glucocerebrosidase enzyme that is naturally encased in carrot cells, also for the treatment of Gaucher disease; pr- antiTNF, a similar plant cell version of etanercept (Enbrel®) for the treatment of certain immune diseases such as rheumatoid arthritis, juvenile idiopathic arthritis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis; and others.
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Source: Protalix BioTherapeutics, Inc.