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BioMarin Announces Third Quarter 2004 Financial Results
Conference Call and Webcast to be Held Today at 12:00 PM EST (18:00 CET)
Nov 2, 2004
PRNewswire-FirstCall
NOVATO, Calif.

BioMarin Pharmaceutical Inc. (Nasdaq and SWX: BMRN) today announced financial results for the third quarter of the fiscal year 2004. The company incurred a net loss of $29.5 million ($0.46 per share) for the third quarter ended September 30, 2004 compared to $21.3 million ($0.33 per share) for the third quarter ended September 30, 2003. The net loss was $105.0 million ($1.63 per share) for the nine months ended September 30, 2004 compared to $50.1 million ($0.81 per share) for the nine months ended September 30, 2003.

BioMarin's net loss for the quarter ended September 30, 2004 included $3.8 million in non-cash charges related to the acquisition of the Ascent Pediatrics business. The non-cash charges related to the acquisition for the nine months ended September 30, 2004 were $41.4 million.

Louis Drapeau, Acting Chief Executive Officer of BioMarin commented, "The Board of Directors and senior management team have set specific goals for improving our financial position and strengthening our business without slowing the progress of our key product development programs. First, we are intensely focused on product outlicensing activities designed to bolster our cash position and reduce our development expenses while retaining the rights to market our products in the United States. Secondly, we are initiating strategic marketing campaigns for Orapred(R) in anticipation of new generic competition. Lastly, we have implemented company-wide cost reduction strategies that will reduce expenses in 2005."

Mr. Drapeau continued, "In the next two months, we anticipate passing two important product development milestones. We expect to file for approval of Aryplase(TM) in the United States and European Union. Additionally, we plan to initiate a Phase 2 study of Phenoptin(TM) in phenylketonuria (PKU) patients and to discuss key elements of our PKU clinical development plan with the FDA. On a final note, the Board of Directors has interviewed several highly qualified candidates for the permanent CEO position at BioMarin."

BioMarin recorded $0.2 million in net sales of Orapred(R) (prednisolone sodium phosphate oral solution) for the third quarter and $4.7 million for the nine months ended September 30, 2004. BioMarin began recording sales of Orapred after the close of the acquisition of the Ascent Pediatrics business from Medicis Pharmaceutical Corporation, which occurred on May 18, 2004. Prescriptions of Orapred for the first nine months of the 2004 increased 6 percent over the same period in 2003 (IMS; NPA).

Net sales of Aldurazyme(R) (laronidase) recorded by BioMarin/Genzyme LLC were $10.3 million for the third quarter ended September 30, 2004 compared to $3.4 million for the third quarter ended September 30, 2003. Aldurazyme was launched by Genzyme Corporation in the United States in May 2003 and in the European Union in June 2003. Net sales recorded by BioMarin/Genzyme LLC for the nine months ended September 30, 2004 were $27.0 million compared to $4.9 million for the same period in 2003. BioMarin's 50 percent share of the loss from BioMarin/Genzyme LLC for the third quarter ended September 30, 2004 was $0.5 million.

BioMarin's net loss in the third quarter of 2004 included: $12.8 million in selling, general and administrative expenses; $3.8 million in non-cash expenses related to the acquisition of the Ascent Pediatrics business from Medicis Pharmaceutical; $5.7 million in ongoing research and development expenses related to Aryplase(TM) (galsulfase) for the treatment of mucopolysaccharidosis VI; and research and development expenses of $2.6 million related to the advancement of the company's PKU clinical development program.

Third Quarter Program Highlights

Investigational New Drug Application (IND) Filed With the U.S. Food and Drug Administration (FDA) for Phenoptin(TM) (6R-BH4) for PKU

On August 24, 2004, BioMarin announced it had filed an IND with the FDA for Phenoptin, a proprietary oral formulation of 6R-BH4. The company expects to initiate a Phase 2 clinical trial of Phenoptin based on this IND by the end of 2004.

Positive Phase 1b Clinical Data on Vibrilase(TM) (Vibriolysin) for Serious Burns

On August 25, 2004, BioMarin announced positive results from its Phase 1b clinical trial for Vibrilase, a topical enzyme for the treatment of serious burns. Data from the trial suggest that treatment with Vibrilase is generally safe and well-tolerated, and effective in debriding partial-thickness burns.

BioMarin will host a conference call and webcast to discuss third quarter financial results today at 12:00 PM EST (18:00 CET). This event can be accessed on the BioMarin website at: http://investor.biomarinpharm.com/ .

   Date:  November 3, 2004
   Time:  12:00 PM EST (18:00 CET)
   U.S. & Canada Toll-free Dial in #:  800-706-7741
   International Dial in #:  617-614-3471
   Participant Pass Code:  34306670
   Replay Toll-free Dial in #:  888-286-8010
   Replay International Dial in #:  617-801-6888
   Replay Code #:  98459011

BioMarin Pharmaceutical Inc. develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the performance of BioMarin's product Orapred and BioMarin/Genzyme LLC's product Aldurazyme; the financial performance of the company as a whole; the continued development and commercialization of Aldurazyme, Aryplase and Phenoptin; and actions by regulatory authorities. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: our joint venture partner's success in continuing the commercialization or Aldurazyme; results and timing of current and planned preclinical studies and clinical trials; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning each of the described products; the market for each of these products and particularly Aldurazyme and Orapred; actual sales of Aldurazyme and Orapred; the possible development of competing products; the effect on sales of Orapred following the recent approval of a generic product that is therapeutically equivalent to Orapred; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Factors That May Affect Future Results" in BioMarin's 2003 Annual Report on Form 10-K and the factors contained in BioMarin's reports on Forms 10-Q and 8-K. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation, to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

NOTE: Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC. All rights reserved.

Orapred(R) and Ascent(R) are registered trademarks of Medicis Pediatrics, Inc., and are used under license.

   Contacts:

   Joshua A. Grass
   Sr. Manager, Investor & Financial Relations
   BioMarin Pharmaceutical Inc.
   415-506-6777

   Susan Ferris
   Manager, Corporate Communications
   BioMarin Pharmaceutical Inc.
   415-506-6701


                       Consolidated Balance Sheets
             (In thousands, except share and per share data)

                                                December 31,  September 30,
                                                    2003           2004
                                                               (unaudited)
  Assets
  Current assets:
    Cash and cash equivalents                     $121,406        $34,981
    Short-term investments                          84,951         51,570
    Restricted cash                                     --         25,198
    Accounts receivable                                 --            119
    Inventory                                           --          3,090
    Investment in and advances to
    BioMarin/Genzyme LLC                            16,058         22,650
    Other current assets                             2,854          3,079

      Total current assets                         225,269        140,687

  Property and equipment, net                       25,154         35,620
  Acquired intangible assets, net                       --         99,123
  Goodwill                                              --         32,250
  Other assets                                       5,917          4,550

      Total assets                                $256,340       $312,230

  Liabilities and Stockholders' Equity
  Current liabilities:
    Accounts payable and accrued liabilities       $10,098        $24,541
    Current portion of acquisition obligation,
     net of discount                                    --         48,747
    Other current liabilities                        2,717          2,427

      Total current liabilities                     12,815         75,715

  Convertible debt                                 125,000        125,000
    Equipment and facility loan                         --          6,986
    Long-term portion of acquisition obligation,
     net of discount                                    --         90,076
    Other long-term liabilities                        672            290

      Total liabilities                            138,487        298,067

  Stockholders' equity:
    Common stock, $0.001 par value: 150,000,000
     shares authorized; 64,156,285 and
     64,388,138 shares issued and outstanding
     December 31, 2003 and September 30, 2004,
     respectively                                       64             64
    Additional paid-in capital                     414,110        420,758
    Warrants                                         5,219             --
    Deferred compensation                             (145)            --
    Accumulated other comprehensive loss               (17)          (260)
    Accumulated deficit                           (301,378)      (406,399)

      Total stockholders' equity                   117,853         14,163

      Total liabilities and stockholders' equity  $256,340       $312,230


                  Consolidated Statements of Operations
     For the Three and Nine Months Ended September 30, 2003 and 2004
             (In thousands, except per share data, unaudited)

                              Three Months Ended      Nine Months Ended
                                  September 30,         September 30,
                               2003        2004        2003        2004

  Net product sales              $--        $181         $--      $4,744
  Milestone revenue               --          --      12,100          --

    Total revenue                 --         181      12,100       4,744

  Operating expenses:
    Cost of sales (excludes
     amortization of
     developed product
     technology)                  --          35          --         648
    Research and
     development              14,211      11,806      36,784      37,670
    Selling, general and
     administrative            1,956      12,761       9,364      24,304
    Amortization of acquired
     intangible assets            --       1,694          --       2,513
    Acquired in-process
     research and development     --          --          --      35,444
    Equity in the loss of
     BioMarin/Genzyme LLC      4,503         507      16,642       3,965

      Total operating
       expenses               20,670      26,803      62,790     104,544

  Loss from operations       (20,670)    (26,622)    (50,690)    (99,800)
  Interest income                781         573       1,787       1,997
  Interest expense            (1,402)     (3,429)     (1,745)     (7,218)

  Net loss from continuing
   operations                (21,291)    (29,478)    (50,648)   (105,021)
  Gain on disposal of
   discontinued operations        --          --         577          --

    Net loss                $(21,291)   $(29,478)   $(50,071)  $(105,021)

  Net loss per share,
   basic and diluted:
    Net loss from
     continuing operations    $(0.33)     $(0.46)     $(0.82)     $(1.63)
    Gain on disposal
     of discontinued
     operations                   --          --        0.01          --

    Net loss                  $(0.33)     $(0.46)     $(0.81)     $(1.63)

  Weighted average
   common shares
   outstanding, basic
   and diluted                63,815      64,384      61,450      64,316

SOURCE: BioMarin Pharmaceutical Inc.

CONTACT: Joshua A. Grass, Sr. Manager, Investor & Financial Relations,
+1-415-506-6777, or Susan Ferris, Manager, Corporate Communications,
+1-415-506-6701, both of both of BioMarin Pharmaceutical Inc.


BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

Palynziq®
Brineura®
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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