BioMarin Pharmaceutical (BMRN)

Firdpase: BioMarin is on track to launch Firdapse for LEMS in the EU on a country by country basis beginning in late March. The company also expects to meet with the FDA regarding the development strategy in the U.S. in the first half of 2010 and to explore additional possible indications.

GALNS for MPS IVA: The Phase I/II study is a 36-week, open-label, within-patient dose escalation trial followed by a treatment continuation phase. Encouraging preliminary results include: (1) decrease in keratan sulfate (KS) levels within a few weeks after the start of therapy; (2) improvements in 6-minute walk distance, 3-minute stair climb and pulmonary function at 24 weeks are consistent with those observed with clinical studies for MPS I, MPS II, and MPS VI; and (3) the frequency and severity of infusion reactions appear comparable to those observed with Naglazyme and Aldurazyme. The company expects to report full top-line results in the second quarter of 2010. Assuming positive results from the Phase I/II study, BioMarin expects to initiate a pivotal Phase III study by the fourth quarter of 2010 or first quarter of 2011.

Kuvan outcomes study/ Lifecycle development: BioMarin expects to initiate a randomized, placebo-controlled, 13-week Kuvan outcomes study in June 2010. Endpoints include clinically validated measures of neuropsychiatric symptoms. Several other programs are underway to expand and protect the market and to improve the ability of healthcare providers and patients to better manage PKU. These programs include a state-of-the-art handheld device to measure blood Phe levels in PKU patients. Human studies are planned for 2010. Regulatory approval and commercial availability of the handheld blood Phe monitor is expected in the first half of 2011.