Protalix BioTherapeutics, Inc. (NYSE-Amex: PLX), announced today positive top-line results from the Company's pivotal Phase III clinical trial of UPLYSO (taliglucerase alfa, previously referred to as prGCD) in treatment naive patients diagnosed with Gaucher disease.
The trial met its primary endpoint, mean reduction in spleen volume after nine months compared with baselines, in both 60 U/kg dose and in the lower 30 U/kg dose treatment groups (P<0.0001). The primary endpoint was stipulated in the Special Protocol Assessment (SPA) agreed on by the Company and the U.S. Food and Drug Administration (the “FDA”) prior to the commencement of the trial. Additionally, the primary endpoint was observed already after six months of treatment in both treatment groups.
Statistically significant improvements compared with baselines were also observed in the secondary endpoints, including increase in hemoglobin level, decrease in liver size and increase in platelet count at the 60 U/kg dose.
Statistically significant improvements compared with baselines were observed in hemoglobin level and liver size and significant nominal reduction in platelet count in the lower dose of 30 U/kg.
The safety analysis for both doses showed that UPLYSO was well tolerated and no serious adverse events were reported. Only 6% of patients in the trial developed antibodies to UPLYSO during the study. None of the patients in the trial developed neutralizing antibodies to UPLYSO. Only 6% of the patients in the trial experienced hypersensitivity.
Most adverse events were mild in intensity and not drug related and were transient in nature.
The Company plans to present more comprehensive results in the near future, and at upcoming medical meetings.
“Clinical studies with UPLYSO to date demonstrate very encouraging efficacy results with a high safety profile and a low rate of antibody formation,” said Professor Ari Zimran, M.D. Director of the Gaucher Clinic at Shaare Zedek Medical Center in Jerusalem and Coordinating Investigator of the Company’s pivotal Phase III trial of UPLYSO. “The results of this pivotal trial suggest that UPLYSO has the potential to become an attractive treatment alternative for Gaucher disease patients.”
"We are extremely pleased with the top line Phase III safety and efficacy results for UPLYSO for the treatment for Gaucher disease,” said Dr. David Aviezer, President and Chief Executive Officer. “We expect to complete the ongoing rolling New Drug Application (NDA) submission for marketing clearance with FDA before the end of this year. In addition, these results further validate our ProCellEx platform for the expression of safe and efficacious human therapeutic proteins in plant cell cultures, and sets the ground for many other lucrative opportunities.”