BioMarin Announces Second Quarter 2013 Financial Results

Total Revenue Growth Exceeds 10 Percent

Phase 3 Trial to Start in 3Q 2013

Conference Call and Webcast to be Held Today at 5:00 p.m. ET

SAN RAFAEL, Calif., July 25, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced financial results for its second quarter ended June 30, 2013. GAAP net loss was $21.5 million ($0.16 per diluted share) for the second quarter of 2013, compared to GAAP net loss of $32.0 million ($0.27 per diluted share) for the second quarter of 2012. GAAP net loss for the six months ended June 30, 2013 was $61.3 million ($0.46 per diluted share), as compared to GAAP net loss of $56.0 million ($0.48 per diluted share) for the six months ended June 30, 2012. Non-GAAP adjusted EBITDA was $0.0 million for the second quarter of 2013, compared to non-GAAP adjusted EBITDA loss of $7.3 million for the second quarter of 2012. Non-GAAP adjusted EBITDA was  a loss of $8.0 million for the six months ended June 30, 2013, as compared to a loss of $7.2 million for the six months ended June 30, 2012. The decreased GAAP net loss and the decreased non-GAAP adjusted EBITDA loss for the second quarter of 2013 compared to the second quarter of 2012 was primarily due to higher total revenue and a smaller increase in operating expenses.

As of June 30, 2013, BioMarin had cash, cash equivalents and short and long-term investments totaling $ 524.4 million, as compared to $525.7 million on March 31, 2013. 

"In the second quarter, we continued to execute on our development goals as we head into the second half of the year," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "We made good progress in a number of important areas including commercial, R&D and regulatory. Highlights included initiation of the Phase 3 PEG-PAL trial for PKU, confirmation of priority review status for Vimizim in the U.S., validation of the MAA by the EMA, submission of the Vimizim MA in Brazil and a positive update of the ongoing BMN 673 trial for genetically-defined cancers at the ASCO annual meeting. We are pleased with the momentum of the BMN 673 program because of maturing and improving responses, completed positive regulatory discussions and earlier Phase 3 timelines than originally anticipated. We remain on track to meet our upcoming goals for the remainder of the year."

Research and Development Programs

BioMarin continues to make significant progress in research and development to ensure a strong pipeline for the company. The current pipeline includes programs in various stages of development that focus on treating a range of rare and serious unmet medical needs.     

Programs Under Regulatory Review for Approval

• Vimizim for MPS IVA: During the second quarter, regulatory advancements were made worldwide. The FDA granted Vimizim priority review designation and assigned a PDUFA date of February 28, 2014. The EMA has validated the Vimizim MAA and has granted accelerated review status, which could lead to a CHMP opinion before year end. During the second quarter, the company also submitted the MA in Brazil. Ongoing production of bulk drug substance and drug product will allow for a global launch following approvals in multiple countries. Both the FDA and EMA have conducted or scheduled pre-approval inspections associated with the review of the marketing applications.

Advanced Clinical Programs

• PEG-PAL for PKU: The company initiated a pivotal Phase 3 study in the second quarter of 2013. The Phase 3 program includes (1) an open-label study to evaluate safety and blood Phe levels in naïve patients and (2) a randomized controlled study of the Phase 2 extension study patients and patients from the open label trial to evaluate blood Phe levels and neurocognitive endpoints, which is expected to be initiated in the third quarter of 2013. The company expects top-line results for the Phase 3 study in the fourth quarter of 2014.
   
• BMN 701 for Pompe Disease: The company plans to initiate a Phase 2/3 switching trial by the end of 2013 in late-onset Pompe patients who have previously been treated with alglucosidase alfa. Subject to discussions with health authorities, the proposed study design is a single arm trial, with treatment at 20 mg/kg administered every other week for 24 weeks and the respiratory parameter MIP as the primary endpoint. The company has completed a full scale production campaign using its new higher producing cell line and has verified the production yields. Characterization testing is currently ongoing, and it is expected that regulatory filings to support the introduction of this material into clinical studies will be submitted in the fourth quarter of 2013.
   
• BMN 673 (PARP inhibitor): As of July 24, the RECIST response rate from the ongoing trial is nine out of 18 breast cancer patients, or 50 percent, including one confirmed complete response. This response rate includes three additional confirmed responses since the last update at the ASCO Annual Meeting, and two new patients are yet to be confirmed. Four patients are ongoing with stable disease with potential for additional responses. All patients have been on treatment for at least twelve weeks. The study is still ongoing, and the company will provide additional updates later this year, including data in ovarian cancer, Ewing's sarcoma and small cell lung cancer. BioMarin now expects to initiate a Phase 3 trial for BMN 673 in deleterious gBRCA mutation metastatic breast cancer in late third quarter of 2013, earlier than previously announced at the ASCO Annual Meeting in early June.  

Early-Stage Clinical Programs

• BMN 111 for Achondroplasia: The company is modifying its clinical program based on a request from the Food and Drug Administration (FDA) for more clinical pharmacokinetic and safety data in children with the disease. BioMarin previously completed a Phase 1 study in adult healthy volunteers. Although the FDA acknowledges that the company has identified a range of doses that are relatively well-tolerated short–term, it has now requested additional PK and safety data in children with achondroplasia before proceeding to extended dosing in children. The FDA has placed the BMN 111 program on partial clinical hold. The company will work with the appropriate health authorities on the implications on the program. The company expects to initiate its first study in pediatric patients in the fourth quarter of 2013 or the first quarter of 2014.
   
• BMN 190 for LINCL (Batten disease): BioMarin filed a CTA in the first quarter of 2013 and expects to begin enrolling the study in the third quarter of 2013. 

Preclinical Programs

• Other early stage programs: BioMarin is working on multiple additional early development opportunities, including two new lead optimization programs gained through the acquisition of Zacharon Pharmaceuticals: inhibition of heparan sulfate synthesis for MPS III and inhibition of ganglioside synthesis for diseases such as Tay Sachs and Sandhoff. The company also has an ongoing Factor VIII gene therapy research program for Hemophilia A from University College London and St. Jude Children's Research Hospital.

Non-GAAP Adjusted EBITDA Reconciliation

The results for the three and six months ended June 30, 2013 and June 30, 2012 and financial guidance for the year ending December 31, 2013 are all determined in accordance with GAAP except for non-GAAP adjusted EBITDA which is determined on a non-GAAP basis. As used in this release, non-GAAP adjusted EBITDA is based on GAAP earnings before interest, taxes, depreciation and amortization (EBITDA) and further adjusted to also exclude certain non-cash stock compensation expense, non-cash contingent consideration expense and certain other nonrecurring material items (non-GAAP adjusted EBITDA). 

The following table presents the reconciliation of non-GAAP to GAAP financial metrics:

BioMarin believes that this non-GAAP information is useful to investors, taken in conjunction with BioMarin's GAAP information because it provides additional information regarding the performance of BioMarin's core ongoing business, Naglazyme, Kuvan, Aldurazyme and Firdapse and development of its pipeline. By providing information about both the overall GAAP financial performance and the non-GAAP measures that focus on continuing operations, the company believes that the additional information enhances investors' overall understanding of the company's business and prospects for the future. Further, the company uses both the GAAP and the non-GAAP results and expectations internally for its operating, budgeting and financial planning purposes.

Conference Call Details

BioMarin will host a conference call and webcast to discuss second quarter 2013 financial results today, Thursday, July 25, 2013 at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.

About BioMarin

BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company's product portfolio comprises four approved products and multiple clinical and pre-clinical product candidates. Approved products include Naglazyme® (galsulfase) for mucopolysaccharidosis VI (MPS VI), a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for mucopolysaccharidosis I (MPS I), a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Tablets, for phenylketonuria (PKU), developed in partnership with Merck Serono, a division of Merck KGaA of Darmstadt, Germany; and Firdapse® (amifampridine), which has been approved by the European Commission for the treatment of Lambert Eaton Myasthenic Syndrome (LEMS). Product candidates include Vimizim (N-acetylgalactosamine 6-sulfatase), formally referred to as GALNS, which successfully completed Phase III clinical development for the treatment of MPS IVA, PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase), which is currently in Phase III clinical development for the treatment of PKU, BMN-701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase I/II clinical development for the treatment of Pompe disease, BMN-673, a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase I/II clinical development for the treatment of genetically-defined cancers, and BMN-111, a modified C-natriuretic peptide, which is currently in Phase I clinical development for the treatment of achondroplasia. For additional information, please visit www.BMRN.com. Information on BioMarin's website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including, without limitation, statements about: the expectations of revenue and sales related to Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; our expectations regarding the progress and timing of BioMarin's clinical trials of PEG-PAL, BMN-673, BMN-701, BMN-111 and other product candidates; the continued clinical development and commercialization of Aldurazyme, Naglazyme, Kuvan, Firdapse, and its product candidates; our expectations regarding the timing of our regulatory filings for Vimizim and our other product candidates; and actions by regulatory authorities, particularly actions related to Vimizim. 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 success in the continued commercialization of Naglazyme, Kuvan, and Firdapse; Genzyme Corporation's success in continuing the commercialization of Aldurazyme; results and timing of current and planned preclinical studies and clinical trials, particularly with respect to PEG-PAL, BMN-673, BMN-701 and BMN-111; our ability to successfully manufacture our products and product candidates; the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities concerning Vimizim and each of the other described products and product candidates; the market for each of these products and particularly Aldurazyme, Naglazyme, Kuvan and Firdapse; actual sales of Aldurazyme, Naglazyme Kuvan and Firdapse; Merck Serono's activities related to Kuvan; and those risks that are discussed  in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, BioMarin's 2012 Annual Report on Form 10-K, and our periodic reports on Form 10-Q and Form 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.

Vimizim™ is our trademark, and BioMarin^®, Naglazyme^®, Kuvan^®, Firdapse^® are registered trademarks of BioMarin Pharmaceutical Inc.

Aldurazyme^® is a registered trademark of BioMarin/Genzyme LLC.