Serono metabolic drug posts positive trial results

Phenylketonuria (PKU) affects approximately 50,000 diagnosed patients in the developed world and is caused by a deficiency of the enzyme phenylalanine hydroxylase which is required for the metabolism of phenylalanine (Phe), an essential amino acid found in most protein-containing foods.

If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and brain, resulting in a variety of complications including severe mental retardation and brain damage, mental illness, seizures and tremors, and cognitive problems.

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 US, Europe and Canada. All patients had demonstrated a reduction in blood Phe levels, approximately 30% or more, following treatment with Phenoptin in a phase II screening study.

“These encouraging results represent an important milestone in Serono’s strategy to bring to market novel treatments for serious metabolic diseases,” said Dr Susan Herbert, head of reproductive health and metabolic endocrinology global product development at Serono.