Mixed Phase III PKU results for BioMarin's pegvaliase

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) lost $2.75 to $78.19 on Monday after its pegvaliase ( BMN 165) met the primary endpoint of the Phase III PRISM-2 study to treat phenylketonuria (PKU), but did not lead to significant improvements on key secondary endpoints. The company plans to submit a BLA to FDA by YE16 for the pegylated recombinant phenylalanine ammonia lyase ( PAL) enzyme.

BioMarin said pegvaliase met the study's primary endpoint of change in blood phenylalanine from baseline to week eight vs. placebo. PKU patients receiving pegvaliase had mean blood phenylalanine levels of 527.2 umol/L at week eight, compared to 503.9 umol/L at baseline. Patients receiving placebo had mean phenylalanine of 1,385.7 umol/L at week eight, compared to 536 umol/L at baseline (p<0.0001). ...