Biopharmaceutical company Protalix BioTherapeutics has treated the first patient in a Phase I study of PRX-112 (oral GCD), an orally-administered enzyme for the treatment of Gaucher disease.
The open-label safety and pharmacokinetic study will assess the delivery of prGCD, the company's approved injectactble form of the glucocerebrosidase enzyme, after oral administration of Oral GCD in 12 patients.
Study patients will be orally administered with re-suspended carrot cells once in the first cohort of the trial and three times successively each day during the second cohort of the trial.
Gaucher disease is a genetic lysosomal storage disease, characterised by a deficiency in the enzyme glucocerebrosidase, where glucosylceramide collects around major organs.
Shaare Zedek Medical Center, Jerusalem, Israel, the Gaucher Clinic director and lead clinical investigator Professor Ari Zimran said Oral GCD enables the delivery of active enzyme into the patient's blood stream using the natural characteristics of plant cells.
"We believe oral delivery of GCD has the potential to improve patients' quality of life without compromising the efficacy or safety of the treatment," Zimran said.
The company expects to complete the study during the third quarter of 2013.
The stability of the enzyme in the carrot cell, and the cellulose wall's capacity to protect the enzyme against degradation in the digestive tract in an in-vitro model of the stomach and intestines. were established in pre-clinical studies.
Enzyme levels in the plasma and active enzyme gathering in target organs such as the spleen and liver have been demonstrated in both rats and pigs fed with PRX-112.
Protalix product development senior vice president Dr Einat Brill Almon said; "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."