SERONO AND BIOMARIN REPORT PHENOPTIN DATA
Serono and BioMarin Pharmaceutical's investigational oral small molecule, Phenoptin for the treatment of the genetic disorder phenylketonuria, has posted positive results in a Phase III trial.
Results confirm that all pre-specified primary and secondary endpoints were met and data from the Phase III study demonstrate a statistically significant reduction at six weeks in blood Phe levels in patients receiving Phenoptin, compared with those receiving placebo.
The study enrolled 89 patients with elevated blood Phe levels aged eight years and above at 29 sites in the U.S., Europe and Canada. All patients had demonstrated a reduction in blood Phe levels, approximately 30 percent or more, following treatment with Phenoptin in a Phase II screening study.