Protalix Top Line Results (AMEX:PLX)

On Thursday, shares of Protalix BioTherapeutics moved upwards after they announced the positive results from their Phase 3 clinical trial of UPLYSO drug for the treatment of Gaucher disease. The company announced that the trial has met the desired endpoints and that they were able to get mean reduction in the spleen volume after nine months compared with baselines. The primary endpoints were mentioned in the Special Protocol Assessment which were agreed by the company itself and the Food and Drug Administration before the trial was initiated. Currently the shares of Protalix BioTherapeutics are 5.52 percent above from the previous closing.

The Phase 3 clinical trial for UPLYSO was a nine months, randomized, dose-ranging safety and efficacy study in patients suffering from Gaucher disease. Patients were randomized to receive 60 units/kg and 30 units/kg of UPLYSO administered intravenously once a fortnight. The Food and Drug Administration panel had already granted UPLYSO orphan product designation and fast track development status and was under the SPA. Protalix BioTherapeutics is currently developing UPLYSO for Gaucher disease patients in the United States and in other European countries under the Expanded Access Program (EAP) which has been approved by the FDA.

The safety analysis for both the doses showed that UPLYSO does not react sharply and has no serious adverse events reported so far. Additionally, significant improvements were observed even in the secondary endpoints, including increase in hemoglobin levels, decrease in liver size and increase in platelet count at the 60 units/kg dose. Though some adverse effects were registered but they were very mild and not drug related and transient in nature. Only six percent of the patients in the trial experienced hypersensitivity and none of the patients developed neutralizing antibodies to UPLYSO. Protalix BioTherapeutics have announced that they plan to present more comprehensive results in the future.