BioMarin Announces Fourth Quarter and Full Year 2014 Financial Results

Total BioMarin Revenue Increased 37% to $751 million in 2014

Rolling Submission of NDA for Drisapersen Underway; Acquisition of Prosensa Complete

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

SAN RAFAEL, Calif., Feb. 25, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced financial results for the fourth quarter and full year 2014, including full year revenues of $751.0 million, a 37% increase over 2013. For the quarter ended December 31, 2014, Non-GAAP net loss declined to $11.4 million ($0.08 per share, basic and diluted) for the fourth quarter of 2014, compared to non-GAAP net loss of $15.8 million ($0.11 per share, basic and diluted) for the fourth quarter of 2013. For the year ended December 31, 2014, non-GAAP net loss was reduced to $25.9 million ($0.18 per share, basic and diluted), compared to non-GAAP net loss of $40.4 million ($0.29 per share, basic and $0.30 per share diluted) for the year ended December 31, 2013.

GAAP net loss was $69.8 million ($0.47 per share, basic and diluted) for the fourth quarter of 2014, compared to GAAP net loss of $62.0 million ($0.43 per share basic and $0.44 per share diluted) for the fourth quarter of 2013. GAAP net loss for the year ended December 31, 2014 was $134.0 million ($0.92 per share, basic and diluted), compared to GAAP net loss of $176.4 million ($1.28 per share, basic and diluted) for the year ended December 31, 2013. As of December 31, 2014, BioMarin had cash, cash equivalents and investments totaling $1,043.1 million, as compared to $1,114.7 million on September 30, 2014.

"BioMarin delivered another year of strong results from our five commercialized products. Our accomplishments in 2014 have set the stage for a very productive 2015 both in terms of commercial success and clinical and regulatory milestones," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "The acquisition of Prosensa in January 2015 adds exciting potential products to our rare disease portfolio. Importantly for shareholders, we believe the near-term approval of drisapersen in either the U.S. or Europe will drive us to profitability on a non-GAAP basis in 2017. Of even greater significance, we can now leverage our capabilities getting orphan-drugs approved for the benefit of boys with Duchenne Muscular Dystrophy, who currently have no approved treatment options."

Net Product Revenue

2015 Guidance

Acquisition of Prosensa

With the acquisition of Prosensa completed this quarter, BioMarin added to its portfolio multiple orphan-drug candidates, including Prosensa's exon-skipping product candidate drisapersen for the treatment of Duchenne Muscular Dystrophy (DMD). Drisapersen has Orphan, Fast Track and Breakthrough Therapy designation and is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Drisapersen, a potential first-to-market and best-in-class product for treating a large population of patients with a rare, fatal genetic disease represents up to 10,000 DMD patients. Follow-on products leverage the same technology platform and target an additional 35,000 DMD patients in BioMarin's commercial territories. All of the pipeline products added to BioMarin's portfolio through the acquisition leverage a proprietary RNA-modulating technology platform for the treatment of various genotypes of DMD and other genetic disorders. BioMarin expects to complete the NDA submission for drisapersen in April 2015.

Select Development Programs

Drisapersen for the treatment of Duchenne Muscular Dystrophy patients with the exon 51 mutation is currently under rolling review with the FDA. The NDA submission to the FDA is expected to be completed in April 2015 and the Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) is expected to be submitted this summer.

Pegvaliase (PEG PAL) for the treatment of phenylketonuria (PKU) is currently in Phase 3 studies. Results are expected in the first quarter of 2016.

Talazoparib (BMN 673) for the treatment of deleterious germline BRCA mutation metastatic breast cancer is currently in Phase 3 studies. Enrollment completion is expected in the second half of 2015.

Reveglucosidase alfa (BMN 701) for the treatment of late onset Pompe Disease is currently being tested in a Phase 2 switch study. Proof of concept results in 20 first patients is expected in the fourth quarter of 2015.

BMN 111 for the treatment of achondroplasia has enrolled all patients in the Phase 2 study and data from the first three cohorts of the study are expected late in the second quarter of 2015.

BMN 044, BMN 045 and BMN 053 are all currently in pilot Phase 2 studies for the treatment of DMD patients with dystrophin gene deletions of exon 44, 45 and 53, respectively.

Cerliponase alfa (BMN 190) for the treatment of late infantile CLN2 disease, a form of Batten disease, is currently in Phase 1/2 studies. In January 2015, BioMarin shared interim data from nine patients who have been followed for at least six months and up to 15 months. Preliminary data suggest that treatment with cerliponase alfa appears to result in stabilization of the disease compared to the natural history based on a standardized measure of motor and language function. Complete Phase 1/2 results are expected in the fourth quarter of 2015.

IND or equivalent filing for BMN 270 for the treatment of hemophilia A is expected to be completed in the first quarter of 2015.

IND or equivalent filing for BMN 250 for the treatment of MPS IIIB (Sanfilippo B syndrome) is expected to be completed mid-2015.

Non-GAAP Financial Information and Reconciliation

The results for the three months and year ended December 31, 2014 and December 31, 2013 and the guidance for 2015 are all determined in accordance with GAAP except for non-GAAP net loss which is determined on a non-GAAP basis. As used in this release, non-GAAP net loss 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 net loss).

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

BioMarin believes that the non-GAAP information in this press release 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, Vimizim, 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 fourth quarter and full year 2014 financial results today, Wednesday, February 25, at 4:30 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 five approved products and multiple clinical and pre-clinical product candidates. Approved products include Vimizim® (elosulfase alfa) for MPS IVA, a product wholly developed and commercialized by BioMarin; Naglazyme® (galsulfase) for MPS VI, a product wholly developed and commercialized by BioMarin; Aldurazyme® (laronidase) for MPS I, a product which BioMarin developed through a 50/50 joint venture with Genzyme Corporation; Kuvan® (sapropterin dihydrochloride) Powder for Oral Solution and 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 pegvaliase (PEGylated recombinant phenylalanine ammonia lyase, formerly referred to as BMN 165 or PEG PAL), which is currently in Phase 3 clinical development for the treatment of PKU, talazoparib (formerly referred to as BMN 673), a poly ADP-ribose polymerase (PARP) inhibitor, which is currently in Phase 3 clinical development for the treatment of germline BRCA breast cancer, reveglucosidase alfa (formerly referred to as BMN 701), a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), which is currently in Phase 3 clinical development for the treatment of Pompe disease, BMN 111, a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, cerliponase alfa (formerly referred to as BMN 190), a recombinant human tripeptidyl peptidase-1 (rhTPP1) for the treatment of CLN2 disorder, a form of Batten disease, which is currently in Phase 1, BMN 270, an AAV-factor VIII vector, for the treatment of hemophilia A and BMN 250, a novel fusion of alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2), for the treatment of MPS IIIB. 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 Vimizim, Naglazyme, Kuvan, Firdapse, and Aldurazyme; the financial performance of the BioMarin as a whole; the timing of BioMarin's clinical trials of PEG PAL, BMN 673, BMN 701, BMN 111, BMN 190, BMN 270, BMN 250 and other product candidates; the continued clinical development and commercialization of Vimizim, Naglazyme, Kuvan, Firdapse, Aldurazyme and its product candidates; 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 success in the commercialization of Vimizim, 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, BMN 111 and BMN 190; 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 each of the described products and product candidates; the market for each of these products; actual sales of Vimizim, Naglazyme, Kuvan, Firdapse and Aldurazyme; Merck Serono's activities related to Kuvan; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's 2013 Annual Report on Form 10-K, and the factors contained in BioMarin's reports on Form 10-Q. 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^®, Naglazyme^®, Kuvan^®, Firdapse® and Vimizim® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates.

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