Protalix Biotherapeutics, Inc. continues to make headlines with its innovative compound, a recombinant form of glucocerebrosidase (prGCD). Last week, the company released that its compound had received the FDA’s Orphan Drug Donation. Yesterday, the company announced that they have completed the Phase III trial for prGCD. The compound is designed to treat Gaucher disease, a rare and serious lysosomal storage disorder in humans with debilitating symptoms.
Protalix plans to release top-line results from this study in October 2009, but has shed light on a few details; the trial enrolled 31 patients across Europe, North America, South America, Israel and South Africa. Most importantly, the company is pleased to report that no serious adverse events occurred in this trial. Protalix expects to complete its rolling New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) by the end of 2009.
The pivotal Phase III clinical trial of prGCD is a randomized, double-blind, parallel group, dose-ranging trial designed to assess the safety and efficacy of prGCD in 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, measured by MRI exams.
Protalix also announced that UPLYSO™ is the proposed brand name for its treatment candidate; the generic name for the compound is taliglucerase alfa. The company has already begun filing trademark applications for UPLYSO.
President and CEO of Protalix, Dr. David Aviezer, commented, “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. 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.”