Protalix Biotherapeutics, Inc. (NYSE-Amex: PLX), announced the completion of its pivotal Phase III trial for prGCD, the Company’s proprietary plant-cell expressed recombinant form of glucocerebrosidase (GCD) for the treatment of Gaucher disease. The trial enrolled a total of 31 patients across Europe, North America, South America, Israel and South Africa. No serious adverse events were reported in this trial. The Company plans to announce top-line results from this study in October 2009 and expects to complete its rolling New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) before the end of the year.
The pivotal Phase III clinical trial of prGCD is a multi-center, randomized, double-blind, parallel group, dose-ranging trial to assess the safety and efficacy of prGCD in treatment naive patients diagnosed with Gaucher disease. In the trial, patients were selected randomly for one of two dosing arms and received IV infusions every two weeks for nine months. The primary endpoint of the study is the reduction in spleen volume from baseline, as measured by MRI.
Protalix also announced that UPLYSO™ is the proposed brand name for its Gaucher disease drug candidate prGCD. The generic name for the compound is taliglucerase alfa. Trademark applications for UPLYSO have been submitted world wide.
“We are pleased to announce the completion of our Phase III pivotal trial with UPLSYO (prGCD) and look forward to reviewing and announcing the results in October,” said Dr. David Aviezer, President and CEO of Protalix. “We expect that the results from this trial will further validate our technology for manufacturing human recombinant protein drugs cost-effectively and safely through our proprietary plant- cell based expression system. We believe our technology can provide a competitive and appealing option for patients world-wide.”