PEG PAL: Phase III amended

BioMarin disclosed in its 1Q14 financial results that it now expects to report data from the double-blind Phase III 165-302 trial of PEG PAL (BMN 165) to treat PKU about a year later than previously expected. The trial is designed to enroll patients receiving a stable dose of PEG PAL in the open-label Phase III 165-301 trial. BioMarin is now modifying the inclusion criteria of the 165-302 trial to specify an undisclosed threshold for phenylalanine reduction in 165-301 trial. The inclusion criteria previously only said patients needed to have received PEG PAL for at least 14 weeks and be on a stable dose for 28 days. The 165-301 trial is enrolling patients who have not been previously treated with PEG PAL.

BioMarin said it is changing the criteria to include patients who had "better" phenylalanine reductions, which the company said will improve the likelihood of "hitting the neurocognitive endpoints" in the trial. PKU is a genetic disease caused by a deficiency of phenylalanine hydroxylase (PAH) enzyme, leading to the accumulation of phenylalanine in the blood and brain. This results in a variety of symptoms, including brain damage, seizures, tremors and cognitive problems. ...