Oct. 28, 2009 (PR Newswire) -- NOVATO, Calif., Oct. 28 /PRNewswire-FirstCall/ --

Financial Highlights ($ in millions, except per share data)

Item                   Q3 2009                Q3 2008 Comparison
Total BioMarin
Revenue               $80.8                  11% increase
Total Net Product
Revenue               $78.4                  16% increase
Naglazyme Net
Product Revenue       $42.1                  26% increase
Aldurazyme BioMarin
Net Product Revenue   $14.6                  $20.7 ($5.6 net inventory
transfer)
Kuvan Net Product
Revenue               $21.7                  57% increase
GAAP Net Income        $6.6                   $0.8
GAAP Net Income
per share             $0.07 (basic),         $0.01 (basic and diluted)
$0.06 (diluted)
Non-GAAP Net Income    $15.5                  $8.2
Non-GAAP Net Income
per share             $0.16 (basic),         $0.08 (basic and diluted)
$0.13 (diluted)

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced financial results for the third quarter ended September 30, 2009. GAAP net income was $6.6 million ($0.06 per diluted share) for the third quarter of 2009, compared to GAAP net income of $0.8 million ($0.01 per diluted share) for the third quarter of 2008. Non-GAAP net income was $15.5 million ($0.13 per diluted share) for the third quarter of 2009, compared to non-GAAP net income of $8.2 million ($0.08 per diluted share) for the third 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 nine months ended September 30, 2009 was $5.2 million ($0.05 per diluted share), compared to GAAP net income of $6.3 million ($0.06 per diluted share) for the nine months ended September 30, 2008. Non-GAAP net income was $33.9 million ($0.33 per diluted share) for the nine months ended September 30, 2009, compared to non-GAAP net income of $21.9 million ($0.21 per diluted share) for the nine months ended September 30, 2008.

As of September 30, 2009, BioMarin had cash, cash equivalents and short and long-term investments totaling $491.8 million.

"During the quarter, we continued to advance our clinical pipeline as we completed enrollment for the Phase I/II study of GALNS for MPS IVA and initiated the Phase II trial of PEG-PAL for PKU. On the commercial front, we were issued patents covering stable tablet formulation and the once daily dosing regimen for Kuvan, and received claims covering the approved administration of Kuvan with food. We believe that these patents are significant in extending protection beyond orphan drug market exclusivity," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "Based on our performance to date, we have narrowed the guidance range on a few items to reflect increased visibility into the year, including improved expectations for the bottom line. Our commercial products are performing well, and we continue to carefully manage expenses. We are also carefully evaluating both internal pipeline programs and external product opportunities to maximize long-term value for both the company and our shareholders. Also, as announced earlier this week, we acquired Huxley Pharmaceuticals, which is developing a proprietary form of 3,4-DAP for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome. A positive opinion was issued by the EMEA last week, and we expect to launch this product in the EU in the first quarter of 2010. This low-risk deal leverages our EU commercial infrastructure, fits our orphan drug strategy with a specialist oriented and potentially high priced therapeutic and has the potential for near-term value creation."

Net Product Revenue

Net product revenue from Naglazyme (galsulfase), an enzyme replacement therapy for mucopolysaccharidosis VI (MPS VI), was $42.1 million for the third quarter of 2009, an increase of 26.0 percent compared to Naglazyme net product revenue of $33.3 million for the third quarter of 2008. Net product revenue from Naglazyme for the nine months ended September 30, 2009 was $124.3 million, an increase of 29.2 percent from net product revenue of $96.2 million for the nine months ended September 30, 2008. Changes in foreign currency rates, net of hedges caused a negative impact to Naglazyme sales of $1.3 million and $5.0 million in the three and nine months ended September 30, 2009, respectively.

Net sales of Aldurazyme (laronidase), an enzyme replacement therapy for mucopolysaccharidosis I (MPS I) recorded by Genzyme, were $40.3 million for the third quarter of 2009, an increase of 5.5 percent compared to net sales of Aldurazyme by Genzyme of $38.2 million for the third quarter of 2008. Net sales of Aldurazyme recorded by Genzyme for the nine months ended September 30, 2009 were $116.4 million, compared to $113.7 million for the nine months ended September 30, 2008. Changes in foreign currency rates caused a negative impact to Aldurazyme sales by Genzyme of $1.5 million and $8.7 million in the three and nine months ended September 30, 2009, respectively. In the third quarter of 2009, Aldurazyme unit volume increased 11.6 percent compared to the third quarter of 2008 as the number of patients on therapy worldwide continues to grow.

Net product revenue to BioMarin related to Aldurazyme was $14.6 million for the third quarter of 2009, compared to net product revenue to BioMarin of $20.7 million for the third quarter of 2008. The timing of inventory transfers to Genzyme reduced net product revenue reported by BioMarin in the third quarter of 2009. During the third quarter of 2009, 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. During the third quarter of 2008, Genzyme's Aldurazyme inventory levels increased, which resulted in BioMarin recorded net product revenue that was higher than the royalty earned on Genzyme third party sales. Net product revenue to BioMarin related to Aldurazyme was $53.4 million for the nine months ended September 30, 2009, compared to $58.1 million for the nine months ended September 30, 2008.

Net product revenue from Kuvan (sapropterin dihydrochloride) Tablets, a product for the treatment of phenylketonuria (PKU), was $21.7 million for the third quarter of 2009, compared to $13.8 million for the third quarter of 2008. Net product revenue from Kuvan for the nine months ended September 30, 2009 was $54.1 million, compared to net revenue of $31.6 million for the nine months ended September 30, 2008. The quantity of commercial tablets dispensed to patients in the U.S., the best metric to track true patient demand, increased 8.5 percent in the third quarter of 2009 compared to the second quarter of 2009.

2009 Guidance

Revenue Guidance ($ in millions)

Item                           2009 Guidance       Previous 2009 Guidance
Total BioMarin Revenues        $313 to $327        $311 to $336
Total Net Product Revenues     $306 to $320        $304 to $329
Naglazyme Net Product Revenue  $165 to $170        $165 to $175
Kuvan Net Product Revenue      $72 to $76          $70 to $80
Aldurazyme Net Product
Revenue to BioMarin           Unchanged           $69 to $74

Selected Income Statement Guidance ($ in millions)

Item                           Guidance            Previous 2009 Guidance
Cost of Sales (% of
Total Revenue)                19% to 20%          19% to 21%
Selling, General and
Administrative Expense        $120 to $125        $120 to $130
Research and Development
Expense*                      $117 to $121        $118 to $128
Interest Income                $5                  $5 to $7
Impairment Loss on La Jolla
and Summit Investments**      Unchanged           $5.9

GAAP Net Income (Loss)         $(8) to $(4)        $(12) to $(6)
Stock Compensation Expense     $35                 $34
Non-GAAP Net Income***         $39.8 to $43.8      $35.4 to $41.4

*    Includes upfront research and development expenses of $8.8 million
associated with the La Jolla Pharmaceutical Company transaction.
**   Represents impairment losses on investments in La Jolla
Pharmaceutical Company of $4.5 million and Summit plc of $1.4 million
during the first quarter of 2009.
***  Non-GAAP net income excludes non-cash stock compensation expense,
nonrecurring material items and the tax effect of the adjustments.
Please see the table provided at the end of this press release for a
full reconciliation between GAAP and non-GAAP expected net income.

Anticipated Upcoming Milestones

1Q10: Launch of amifampridine phosphate in the EU

1Q10: Initiation of Phase I trial for BMN-195 for DMD

1Q10: American College of Medical Genetics (ACMG) Meeting - Possible data from study of Kuvan in institutionalized PKU patients

2Q10: Initiation of Kuvan neurocognitive outcome study

1H10: Announcement of next IND candidate

1H10: Results from Phase I/II trial for GALNS for MPS IVA

Mid-2010: Results from PEG-PAL Phase II trial

Late 2010/ Early 2011: Initiation of pivotal Phase III trial for GALNS for MPS IVA

1H11: 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.

--  GALNS for MPS IVA: BioMarin initiated the Phase I/II trial in mid-April
2009 and completed enrollment in mid-July.