Advanced Programs

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.

Mid-Stage Programs

PEG-PAL for PKU: BioMarin initiated the Phase II trial in September 2009.  The Phase II clinical trial is an open-label, multi-center study to be conducted in approximately 35 patients in a series of dose-escalating cohorts.  The primary treatment period of eight once weekly injections at a fixed dose will be followed by eight weeks of dose and frequency optimization and an extension period.  Results from the Phase II PEG-PAL trial are expected in 3Q 2010. BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy: BioMarin initiated the Phase I trial in the first quarter of 2010 and expects to report results in the third quarter of 2010.  BMN-195 is an orally available small molecule which may upregulate utrophin, a potential substitution for the missing dystrophin protein in DMD patients.  Assuming a successful Phase I trial, BioMarin expects to initiate a Phase II trial in Q1 2011.

Preclinical Programs

BMN-185 - IgA protease for IgA nephropathy: BioMarin is completing early preclinical work and expects to move to the next phase of research in the first half of 2010.  IgA proteases have been shown to cleave IgA complexes, the deposition of which causes IgA nephropathy, an orphan kidney disorder with few treatment alternatives. BMN-673 (PARP Inhibitor) and additional early development candidates:  BioMarin is working on multiple early development opportunities, including the recently acquired BMN-673 from LEAD.  The company expects to file an IND for BMN-673 by the end of 2010.Undisclosed programs: Two additional undisclosed biologics are advancing toward IND-enabling decisions.SOURCE BioMarin Pharmaceutical Inc.