Biopharmaceuticals Protalix Biotherapeutics, together with its partner in the development and commercialization of Casey’s rare diseases division, today released positive topline results from the BRIGHT Phase III clinical study examining alpha-pegoniglassidase (PRX-102), at a dose of 2 mg / kg Body weight given every four weeks as a potential treatment for Fabry disease.
PRX-102 is a company-developed drug, made from a recombinant protein, alpha-galactosidase-A (cross-linked α-galactosidase-), extracted from a plant cell culture that has undergone chemical pegylation after extraction.
The BRIGHT study is a 12-month Phase III clinical trial in open indication and switch-over therapy designed to evaluate the safety, efficacy, and pharmacokinetics of PRX-102 therapy, for up to 30 patients with Fabry disease previously treated with enzyme replacement therapy (ERT). Commercially available for at least three years and in a stable dose given every two weeks.
The topline results indicate that PRX-102 given at a dose of 2 mg per kg of body weight by intravenous infusion every four weeks was well tolerated by the patients who received it and clinical stability was maintained which was maintained in adult Fabri patients. None of the new patients developed antibodies to the drug as a result of the treatment (ADA) following the switch to PRX-102.
“We are excited to publish the results of the BRIGHT study, which is our third consecutive clinical trial in PRX-102 that ends in positive results, after Phase I / II and BRIDGE clinical trials. The results indicate that this experimental drug is well tolerated and has the potential to effectively treat patients. Adults with Fabri’s disease, “said Dr. Einat Brill Almon, senior vice president and chief development officer of Protelix.
“We are encouraged to note that all patients who completed this study chose to join the long-term follow-up study. To date, 80% of patients enrolled in the BRIGHT study have been treated with this treatment regimen for two years. We look forward to furthering the study and evaluating the results once again.”
“The results illustrate the potential of PRX-102 to become an important therapeutic alternative for the Fabri patient community and that a 2 mg PRX-102 treatment regimen every four weeks may offer significant benefits to both patients and physicians. The attending physicians will be happy with a therapeutic regimen Another potential and well-tolerated one that they could offer to Fabry patients subject to the approval of the PRX-102, “said Dror Bashan, president and CEO of Protelix.
“We are pleased with our solid balance sheet that supports the development efforts and look forward to the implementation of a year rich in milestones that will contribute to bearing the value of the company.”
The study included 30 cow patients (24 men and 6 women). The most common symptoms and manifestations of Fabri’s disease included circumcision, fever intolerance, angiocartomas and hypohydrosis. All 30 patients received at least one dose of PRX-102 and 29 patients completed a 12-month study.
Of these 29 patients, 28 received the designated drug regimen of 2 mg / kg every four weeks throughout the study and one patient was transferred to a 1 mg dose of PRX-102 per kg every two weeks according to the protocol. One patient withdrew from the study after the first transfusion due to a car accident.
After screening tests, patients were recruited and switched from the treatment they received to an intravenous infusion of 2 mg per kg of PRX-102 every four weeks for 52 weeks (14 infusions in total). The first infusion of PRX-102 was given under controlled conditions at the study site .
Based on the criteria outlined in the protocol, patients were able to receive the PRX-102 transfusions in home care after the researcher and the Medical Monitor agreed that performing an infusion under such conditions was safe. The safety and efficacy research objectives were tested throughout the 52 weeks of the experiment.
“Patients participating in the BRIGHT study expressed satisfaction with the treatment regimen every four weeks,” said Dr. John Burnett of the University of Iowa and lead researcher in the BRIGHT study. “An infusion of 2 mg / kg every four weeks may allow patients to maintain their status. Clinically reduced by half the number of treatments. “
Patients ‘responses to the survey conducted with a quality of life questionnaire indicate that patients’ self-perception of health remained high and stable during the 52-week study. Approximately 75% of study participants felt an improvement or lack of change in the average severity of pain at week 52 compared to baseline. The influencing factors appearing in the questionnaire also remained stable throughout the study. Pain-related results indicate that there was no increase or return in pain. No clinical events related to Fabry disease have been reported during the study.
“Of the 30 patients who participated, 20 remained negative for neutralizing antibodies throughout the course of treatment. Of the 10 patients who were initially positive for antibodies to the drug, four became negative for neutralizing antibodies at 12 months, a fact that suggests the development of tolerance (toleration) of these patients,” D added. Mr. Almon. These immunogenicity data are highly encouraging and support the PRX-102 benefit-risk profile.
“On behalf of Casey’s staff, we thank patients, their families, and researchers for their time and research participation,” said Giacomo Casey, who heads Casey’s Rare Diseases Division. “Their dedication has helped advance this Phase III study and the topline data are another important milestone in our joint effort to make PRX-102 available to Fabry patients as soon as possible.”