BioMarin Pharmaceutical Inc.
Excluding non-cash expenses related to the acquisition of the Ascent Pediatrics business, BioMarin's Pro Forma Adjusted net loss was $18.0 million ($0.28 per share) for the quarter and $37.9 million ($0.59 per share) for the six months ended June 30, 2004. The following table reconciles BioMarin's GAAP net loss to BioMarin's Pro Forma Adjusted net loss for the quarter and six months ended June 30, 2004:
Three Months Six Months Ended June Ended June 30, 2004 30, 2004 Net loss $(55,598) $(75,543) Non-GAAP adjustments: Acquired in-process research and development 35,444 35,444 Amortization of acquired intangible assets 819 819 Imputed interest expense 1,108 1,108 Fair value inventory adjustment 252 252 Pro Forma Adjusted net loss $(17,975) $(37,920) Pro Forma Adjusted net loss per share, basic and diluted $(0.28) $(0.59) Weighted average common shares outstanding, basic and diluted 64,339 64,282
BioMarin's net loss in the second quarter of 2004 was due primarily to: non-cash expenses of $37.6 million related to the acquisition of the Ascent Pediatrics business from Medicis Pharmaceutical Corporation; $8.2 million in ongoing research and development expenses related to Aryplase(TM) for the treatment of mucopolysaccharidosis VI (MPS VI); BioMarin's 50 percent share of the loss from BioMarin/Genzyme LLC of $1.7 million related to Aldurazyme(R); and research and development expenses of $1.6 million related to the advancement of the company's phenylketonuria (PKU) clinical development program.
BioMarin recorded $4.6 million in net sales of Orapred(R) for the second quarter ended June 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. According to IMS Health (source IMS: MPA), prescriptions of Orapred increased approximately 10 percent for the first half of 2004 over prescriptions for the same period in 2003.
Net sales of Aldurazyme recorded by BioMarin/Genzyme LLC were $9.2 million for the second quarter ended June 30, 2004 compared to $1.1 million for the second quarter ended June 30, 2003 (the product was launched by Genzyme in the United States in May 2003 and in European Union in June 2003). Net sales for the six months ended June 30, 2004 were $16.6 million compared to $1.5 million for the same period in 2003.
Louis Drapeau, Chief Financial Officer of BioMarin stated, "The integration of the Ascent Pediatrics business is proceeding as planned and we are on track to complete the transition from Medicis to BioMarin within three months of the closing of the transaction." Mr. Drapeau continued, "In the second half of the year, we look forward to filing for regulatory approval of Aryplase(TM) for MPS VI in the United States and the European Union based on the positive Phase 3 results we recently announced, and to initiating an additional clinical trial with Phenoptin(TM) in PKU patients."
Second Quarter Program Highlights Positive Clinical Data on 6R-BH4 for PKU
On July 20, 2004, BioMarin announced positive results from a clinical study of 6R-BH4 in PKU patients. 6R-BH4 is the active agent in Phenoptin, an investigational small molecule therapeutic for the treatment of PKU. The results demonstrated that 6R-BH4 reduced blood phenylalanine levels by at least 30 percent in 45 percent of the patients in the study, with a dose of 10 mg/kg for seven days. When the dose was increased to 20 mg/kg, Phe levels dropped by at least 30 percent in 61 percent of the patients. The average reduction in blood Phe in these patients was 50 percent and 64 percent, respectively. No serious side effects of 6R-BH4 were observed. The company also announced that it has received orphan drug designation for Phenoptin in both the United States and European Union. BioMarin expects to initiate an additional clinical study with Phenoptin in PKU patients by the end of 2004.
Positive Phase 3 Clinical Data on Aryplase for MPS VI
On June 3, 2004, BioMarin announced positive results from its Phase 3 clinical trial of Aryplase, an investigational enzyme replacement therapy for the treatment of MPS VI. The clinical trial demonstrated a statistically significant improvement in the primary endpoint of endurance (p=0.025) and one of the secondary endpoints, urinary glycosaminoglycan reduction (p<0.001), in patients receiving Aryplase compared to patients receiving placebo. In the three-minute stair climb endurance test, also a secondary endpoint, patients receiving Aryplase demonstrated an improvement (p=0.053) over patients receiving placebo. BioMarin expects to file for marketing authorization for Aryplase in both the United States and European Union in the fourth quarter of 2004. BioMarin has identified approximately 250 patients with MPS VI, an increase of approximately 25 percent over the previously reported number of approximately 200 patients.
Successful Integration of the Ascent Pediatrics Business
On May 18, 2004, BioMarin completed the acquisition of the Ascent Pediatrics business from Medicis Pharmaceutical Corporation. The transaction provides BioMarin a sales force of approximately 70 people that call on pediatricians located throughout the United States. The sales force details Orapred, the leading product in its class for the treatment of acute exacerbations of asthma in children. The integration of the Ascent Pediatrics business is proceeding rapidly without interruption to the sales and marketing activities, as evidenced by the continued growth in Orapred prescriptions. The Orapred sales and marketing team will soon augment existing precommercialization efforts associated with the company's additional product candidates, beginning with Aryplase.
Increasing Sales of Aldurazyme for MPS I
Sales of Aldurazyme by BioMarin/Genzyme LLC were $9.2 million for the second quarter of 2004. Aldurazyme sales have increased steadily since its launch in the second quarter of 2003. Sales were $1.1 million, $3.4 million, $6.7 million, and $7.4 million and $9.2 million for the five consecutive quarters since launch. Previously announced sales guidance for the 12 months ending December 31, 2004 provided by Genzyme Corporation, BioMarin's joint venture partner responsible for sales and marketing of Aldurazyme, remains unchanged at between $40 million and $44 million. BioMarin and Genzyme recently received marketing authorization for Aldurazyme in Canada and Australia.
Updated Financial Guidance
BioMarin expects that net sales of Orapred will be approximately $42 million for the full year of 2004, of which BioMarin will realize the net sales recorded since the close of the deal with Medicis on May 18, 2004. The company expects that its Pro Forma Adjusted net loss for 2004, excluding the $37.6 million in non-cash expenses related to the Ascent Pediatrics transaction, will be approximately $66 million to $68 million, which is less than the previously projected $68 million to $70 million. On a GAAP basis, BioMarin expects that the net loss for 2004 will be approximately $112 million to $114 million. BioMarin expects to end 2004 with approximately $85 million to $90 million in cash, cash equivalents, and restricted cash.
Upcoming Presentations at Medical Conferences
Investigators will present data from clinical studies of Vibrilase for serious burns, Aldurazyme for MPS I, Aryplase for MPS VI, and Phenoptin for PKU at several medical conferences in the coming months. The following is a list of the abstracts submitted for presentation:
The 12th Congress of the International Society for Burn Injuries, August 22 - August 26, 2004, Yokohama, Japan
-- Clinical Development of Vibrilase(TM) (vibriolysin) for Burn Wound Debridement: Evaluation of Safety in a Phase 1a Study of Irritation and Contact Sensitization, and a Phase 1b Study of Partial-Thickness Burn Wound Debridement
The Society for Inborn Errors of Metabolism (SSIEM) 41st Annual Symposium, August 31 - September 3, 2004, Amsterdam, the Netherlands
-- A Five Year Study of Aldurazyme for Treatment of MPS I -- Enzyme Replacement in Two Patients with MPS I -- Monthly Intrathecal Enzyme Therapy for Canine MPS I -- Update on Clinical Trial Results in MPS VI -- Multiple Dose Study of Tetrahydrobiopterin (BH4) in Phenylketonuria
The American Academy of Pediatrics 2004 National Conference and Exhibition, October 9 - 13, 2004, San Francisco, CA
-- The Important Role of Pediatricians in Recognizing Symptom Clusters in Patients with Mucopolysaccharidosis I
American Society of Human Genetics Annual Meeting 2004, October 26 - 30, 2004, Toronto, Canada
-- A Five Year Study of Aldurazyme for Treatment of MPS I -- The MPS I Registry: Centralized Data Collection to Delineate the Natural History and Treatment of MPS I -- Monthly Intrathecal Enzyme Replacement Therapy Improves Lysosomal Storage in the Brain and Spinal Cord in Canine MPS I -- A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational Clinical Study of Recombinant Human N-acetylgalactosamine 4-Sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI Upcoming Corporate Presentations
BioMarin will present an overview of its business and product development programs at the following healthcare and biotechnology investment conferences:
-- UBS Global Life Sciences Conference, September 27 - 30, 2004, New York City, New York -- Rodman & Renshaw 6th Annual Healthcare Conference, October 26 - 28, 2004, New York City, New York -- CIBC World Markets 15th Annual Healthcare Conference, November 8 -10, 2004, New York City, New York
BioMarin will host a conference call and webcast to discuss second quarter financial results today at 12:00 p.m. EDT (18:00 CEST). This event can be accessed on the BioMarin website at: http://investor.biomarinpharm.com/ .
Date: August 10, 2004 Time: 12:00 p.m. EDT (18:00 CEST) U.S. & Canada Toll-free Dial in #: 1-800-915-4836 International Dial in #: 973-317-5319 Replay Toll-free Dial in #: 1-800-428-6051 Replay International Dial in #: 973-709-2089 Replay Code #: 363459
BioMarin Pharmaceutical Inc. develops innovative biopharmaceuticals and commercializes therapeutics for serious pediatric diseases.
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: the successful integration of the sales force, sales operations and the Orapred business into BioMarin's organization; 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; 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.
BioMarin's press releases and other company information are available online at www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.
NOTE: Aldurazyme(R) is a registered trademark of BioMarin/Genzyme LLC. All rights reserved.
Orapred(R) is a registered trademark of Medicis Pediatrics, Inc., and is used under license.
CONTACT: Joshua A. Grass, Sr. Manager, Investor and Financial Relations, +1-415-506-6777, or Susan Ferris, Manager, Corporate Communications, +1-415-506-6701, both of BioMarin Pharmaceutical Inc.
BioMarin Pharmaceutical Inc. and Subsidiaries Consolidated Balance Sheets (In thousands, except share and per share data) December 31, June 30, 2003 (1) 2004 (unaudited) Assets Current assets: Cash and cash equivalents $121,406 $48,247 Short-term investments 84,951 80,549 Restricted cash -- 25,124 Accounts receivable -- 4,612 Inventory -- 2,074 Investment in and advances to BioMarin/Genzyme LLC 16,058 18,675 Other current assets 2,854 2,306 Total current assets 225,269 181,587 Property and equipment, net 25,154 27,000 Acquired intangible assets and goodwill -- 133,090 Other assets 5,917 5,739 Total assets $256,340 $347,416 Liabilities and Stockholders' Equity Current liabilities: Accounts payable and accrued liabilities $10,098 $20,650 Current portion of acquisition obligation, net of discount -- 58,228 Other current liabilities 2,717 2,392 Total current liabilities 12,815 81,270 Convertible debt 125,000 125,000 Equipment and facility loan -- 3,778 Long-term portion of acquisition obligation, net of discount -- 93,474 Other long-term liabilities 672 704 Total liabilities 138,487 304,226 Stockholders' equity: Common stock, $0.001 par value: 150,000,000 shares authorized; 64,156,285 and 64,366,988 shares issued and outstanding December 31, 2003 and June 30, 2004, respectively 64 64 Additional paid-in capital 414,110 420,577 Warrants 5,219 85 Deferred compensation (145) (58) Accumulated other comprehensive loss (17) (557) Accumulated deficit (301,378) (376,921) Total stockholders' equity 117,853 43,190 Total liabilities and stockholders' equity $256,340 $347,416 (1) December 31, 2003 balances were derived from the audited consolidated financial statements. BioMarin Pharmaceutical Inc. and Subsidiaries Consolidated Statements of Operations For the Three and Six Months Ended June 30, 2003 and 2004 (In thousands, except per share data, unaudited) Three Months Ended Six Months Ended June 30, June 30, 2003 2004 2003 2004 Net product sales $-- $4,563 $-- $4,563 Milestone revenue 12,100 -- 12,100 -- Total revenue 12,100 4,563 12,100 4,563 Operating expenses: Cost of sales -- 613 -- 613 Research and development 11,731 12,292 22,722 26,179 Selling, general and administrative 4,460 7,539 7,259 11,228 Amortization of acquired intangible assets -- 819 -- 819 Acquired in-process research and development -- 35,444 -- 35,444 Equity in the loss of BioMarin/ Genzyme LLC 5,386 1,699 12,139 3,458 Total operating expenses 21,577 58,406 42,120 77,741 Loss from operations (9,477) (53,843) (30,020) (73,178) Interest income 593 663 1,006 1,424 Interest expense (213) (2,418) (343) (3,789) Net loss from continuing operations (9,097) (55,598) (29,357) (75,543) Gain on disposal of discontinued operations -- -- 577 -- Net loss $(9,097) $(55,598) $(28,780) $(75,543) Net loss per share, basic and diluted: Net loss from continuing operations $(0.14) $(0.86) $(0.49) $(1.18) Gain on disposal of discontinued operations -- -- 0.01 -- Net loss $(0.14) $(0.86) $(0.48) $(1.18) Weighted average common shares outstanding, basic and diluted 63,494 64,339 60,247 64,282 Pro forma adjusted net loss (1) $(9,097) $(17,975) $(28,780) $(37,920) Pro forma adjusted net loss per share $(0.14) $(0.28) $(0.48) $(0.59) (1) Pro Forma Adjusted net loss excludes non-cash expenses related to the acquisition of the Ascent Pediatrics business totaling $37.6 million. The non-cash expenses include in-process research and development, amortization of acquired intangible assets, imputed interest expense and a fair value inventory adjustment. Please refer to the body of the press release dated August 10, 2004 for discussion of Pro Forma Adjusted net loss as a non-GAAP financial measure.
SOURCE: BioMarin Pharmaceutical Inc.
CONTACT: Joshua A. Grass, Sr. Manager, Investor and Financial Relations,
+1-415-506-6777, or Susan Ferris, Manager, Corporate Communications,
+1-415-506-6701, both of BioMarin Pharmaceutical Inc.
Web site: http://www.bmrn.com/