BioCentury
ARTICLE | Clinical News

Delay for BioMarin's PEG PAL

May 3, 2014 1:30 AM UTC

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) 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 phenylketonuria (PKU) about a year later than previously expected. The trial is designed to enroll patients who were on 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 that patients must have an undisclosed, pre-specified reduction in phenylalanine in the blood in the 165-301 trial. The inclusion criteria did not previously include the requirement and instead only said patients needed to have received PEG PAL for at least 14 weeks and have been on a stable dose for 28 days.

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. ...