BioMarin Pharmaceutical reports GAAP net income of $4.7M for fourth-quarter 2009

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced financial results for the fourth quarter and year ended December 31, 2009. GAAP net income was $4.7 million ($0.05 per diluted share) for the fourth quarter of 2009, compared to GAAP net income of $24.5 million ($0.21 per diluted share) for the fourth quarter of 2008, which included a $30.0 million payment from Merck Serono related to the approval of Kuvan in the EU.  Non-GAAP net income was $13.5 million ($0.13 per diluted share) for the fourth quarter of 2009, compared to non-GAAP net income of $8.0 million ($0.08 per diluted share) for the fourth quarter of 2008.  Non-GAAP net income excludes non-cash stock compensation expense, certain nonrecurring material items and the tax effect of the adjustments.  The reconciliation of the non-GAAP measures to the estimated GAAP net income is detailed in the table provided at the end of the press release.

GAAP net loss for the year ended December 31, 2009 was $0.5 million ($0.00 per diluted share), compared to GAAP net income of $30.8 million ($0.29 per diluted share) for the year ended December 31, 2008.  Non-GAAP net income was $47.1 million ($0.46 per diluted share) for the year ended December 31, 2009, compared to non-GAAP net income of $30.0 million ($0.29 per diluted share) for the year ended December 31, 2008.

As of December 31, 2009, BioMarin had cash, cash equivalents and short and long-term investments totaling $470.5 million.  

"Robust product sales of Naglazyme and Kuvan drove profitability in the fourth quarter and a strong finish to 2009.  Commercially, we continue to aggressively pursue expansion of the Naglazyme and Kuvan markets and look forward to launching Firdapse in the EU in late March," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.  "We also have much to look forward to in terms of clinical milestones in 2010, including results from the Phase I/II trial of GALNS for MPS IVA in the second quarter, results from the Phase II PEG-PAL trial in the third quarter of 2010, results from the Phase I trial of BMN 195 for DMD in the third quarter of 2010 and the IND filing for the recently acquired PARP inhibitor from LEAD Therapeutics.  Even after factoring in the Huxley and LEAD acquisitions, we expect to be slightly cash flow positive in 2010."

Net Product Revenue

Net product revenue from Naglazyme (galsulfase), an enzyme replacement therapy for mucopolysaccharidosis VI (MPS VI), was $44.4 million for the fourth quarter of 2009, an increase of 21.6 percent compared to Naglazyme net product revenue of $36.5 million for the fourth quarter of 2008.  Net product revenue from Naglazyme for the year ended December 31, 2009 was $168.7 million, an increase of 27.1 percent from net product revenue of $132.7 million for the year ended December 31, 2008.  Changes in foreign currency rates, net of hedges, caused an increase to Naglazyme sales of $0.6 million in the three months ended December 31, 2009 and a decrease of $4.4 million for the year ended December 31, 2009.  

Net sales of Aldurazyme (laronidase), an enzyme replacement therapy for mucopolysaccharidosis I (MPS I) recorded by Genzyme, were $38.7 million for the fourth quarter of 2009, an increase of 2.9 percent compared to net sales of Aldurazyme by Genzyme of $37.6 million for the fourth quarter of 2008.  Net sales of Aldurazyme recorded by Genzyme for the year ended December 31, 2009 were $155.1 million, compared to $151.3 million for the year ended December 31, 2008.  Changes in foreign currency rates caused an increase to Aldurazyme sales by Genzyme of $2.4 million in the three months ended December 31, 2009 and a decrease of $6.3 million for the year ended December 31, 2009.

Net product revenue to BioMarin related to Aldurazyme was $16.8 million for the fourth quarter of 2009, compared to net product revenue to BioMarin of $14.4 million for the fourth quarter of 2008.  During the fourth quarter of 2009, BioMarin transferred more inventory to Genzyme compared to units shipped to third party customers by Genzyme, which resulted in an increase in BioMarin net product revenue from the royalty payable to BioMarin by Genzyme. During the fourth quarter of 2008, BioMarin transferred less inventory to Genzyme compared to units shipped to third party customers by Genzyme, which resulted in a reduction in BioMarin net product revenue from the royalty payable to BioMarin by Genzyme.  Net product revenue to BioMarin related to Aldurazyme was $70.2 million for the year ended December 31, 2009, compared to $72.5 million for the year ended December 31, 2008.

Net product revenue from Kuvan (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), was $22.7 million for the fourth quarter of 2009, compared to $15.1 million for the fourth quarter of 2008.  Net product revenue from Kuvan for the year ended December 31, 2009 was $76.8 million, compared to net revenue of $46.7 million for the year ended December 31, 2008.  The quantity of commercial tablets dispensed to patients in the U.S., the best metric to track true patient demand, increased 17.8 percent in the fourth quarter of 2009 compared to the third quarter of 2009.

Firdapse Launch Update

BioMarin is on track to launch Firdapse for LEMS in the EU on a country by country basis beginning in late March.  Firdapse pricing has been filed in Germany at 23 Euros per tablet.  Since dosages can range from 15 mg to 60 mg a day, the annual cost of therapy can vary widely from patient to patient.  BioMarin estimates that the annual cost will range be between 10,000 and 50,000 Euros per year.  

Anticipated Upcoming Milestones March 2010: Launch of Firdapse in the EU March 2010: American College of Medical Genetics (ACMG) Meeting – data from multiple investigator-sponsored Kuvan trials June 2010: Initiation of Kuvan neurocognitive outcomes study 2Q 2010: Results from Phase I/II trial for GALNS for MPS IVA 3Q 2010: Results from PEG-PAL Phase II trial 3Q 2010: Results from Phase 1 trial for BMN 195 for DMD 4Q 2010/1Q 2011: Initiation of pivotal Phase III trial for GALNS for MPS IVA Late 2010: File IND for BMN-673 (PARP Inhibitor) 1H 2011: Availability of blood Phe monitor

Research and Development Programs

BioMarin continues to make significant investments in research and development to ensure continued growth of the company.  The current pipeline includes programs which are in various stages of development and are focused on treating a range of unmet medical needs.  BioMarin is making significant investments in manufacturing and laboratory facilities to support the advancement of these programs.

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.