BioMarin Announces Second Quarter 2015 Financial Results and Company Update

- In First Six Months of 2015 Vimizim Sales Top $104 million and Total BioMarin Revenue Grows 32% Y/Y

- First Patients Enrolled in 4th Cohort of Phase 2 Study of Vosoritide for the Treatment of Achondroplasia

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

SAN RAFAEL, Calif., Aug. 3, 2015 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) today announced financial results for the second quarter ended June 30, 2015.  Non-GAAP net loss was $5.4 million for the second quarter of 2015, compared to non-GAAP net income of $10.1 million for the second quarter of 2014. GAAP net loss was $82.0 million, or ($0.51) per basic and diluted share for the second quarter of 2015, compared to GAAP net loss of $33.5 million, or ($0.23) per basic and diluted share for the second quarter of 2014. The increased non-GAAP net loss and GAAP net loss for the second quarter of 2015 compared to the second quarter of 2014 was primarily due to increased operating expenses, partially offset by increased revenues due to the strong commercial launch of Vimizim.

Total BioMarin Revenue was $250.5 million for the second quarter of 2015, an increase of 31% compared to the second quarter of 2014. This increase was driven by solid growth across all BioMarin products including Kuvan, Naglazyme and Vimizim.  Sales of Vimizim, now in its fifth full quarter of sales since being approved in early 2014, were recorded in 30 countries in the second quarter and totaled $53.9 million. Naglazyme Net Product Revenue in the second quarter benefitted from a large order in Latin America, consistent with historically uneven sales of the product in that region. This trend is not expected to continue for the remainder of 2015. Kuvan Net Product Revenue in the second quarter increased 28% to $60.1 million driven primarily by patient count increases and high rates of compliance. On a constant currency basis, Total BioMarin Revenue in the second quarter would have been approximately $260.5 million.

As of June 30, 2015, BioMarin had cash, cash equivalents and investments totaling $1,191.2 million, as compared to $1,043.1 million on December 31, 2014.

"The second quarter of 2015 was one of our most productive quarters to date as measured by commercial results and the advancement of all 10 potential products in our clinical development portfolio," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "On the regulatory front, in the quarter we were pleased to have both our New Drug Application and Marketing Authorization Application of drisapersen accepted for review by U.S. and European health authorities.  BioMarin is energized by the potential prospect of near-term approval of drisapersen for the treatment of children with Duchenne muscular dystrophy, a devastating muscle wasting disease for which there is no approved therapy in the U.S." Mr. Bienaimé continued, "Another highly anticipated event in the second quarter was the completion and results of the Phase 2 study with vosoritide for the treatment of achondroplasia. We were pleased to share that vosoritide increased the mean annualized growth velocity by 50% in children in the highest dose cohort who received 15 micrograms per kilogram daily. We believe growth velocity is a leading indicator of improvement in many of the severe complications associated with the disorder, such as foramen magnum compression, sleep apnea, bowed legs, spinal stenosis, recurrent ear infections and obesity.  Based on efficacy and safety observed in the first 3 cohorts of the Phase 2 study we are planning for registration enabling studies at 15 micrograms per kilogram daily and have also begun enrollment in a 4^th higher dose cohort with 30 micrograms per kilogram daily."

 Net Product Revenue (in millions)

2015 Guidance Unchanged

Revenue Guidance ($ in millions)

Select Program Advancements in the Second Quarter

In the second quarter, BioMarin announced that the U.S. Food and Drug Administration (FDA) had accepted for review the submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, and the Prescription Drug User Fee Act (PDUFA) goal date for a decision is December 27, 2015. The FDA has granted drisapersen Priority Review status, which is designated to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. In addition, the Company announced that the European Medicines Agency (EMA) had accepted for review the submission of a Marketing Authorization Application (MAA) for drisapersen for the same indication in the European Union.   

Also announced in the quarter were data from the 26 children participating in the Phase 2 study with vosoritide for the treatment of achondroplasia. Results from the study demonstrated a favorable safety profile and efficacy at the 15 micrograms/kilogram/daily dose. The 10 children in Cohort 3 treated with 15 micrograms per kilogram per day had a mean increase of 50% in their annualized growth velocity compared to their annualized prior 6 month natural history baseline growth velocity. Changes from baseline in proportionality as measured by upper to lower body ratio were not observed. No Serious Adverse Events (SAEs) were observed for the duration of the study. The complete data from the study will be presented at a medical meeting later in the year. Based on the Phase 2 results, BioMarin intends to move into pivotal registration study discussions with health authorities with a dose of 15 micrograms per kilogram daily. 

Today, the Company announced that it had dosed the first patients in the 4^th cohort of its ongoing Phase 2 study with vosoritide using 30 micrograms per kilogram daily to explore the possibility of "catch-up" growth in patients.

Conference Call Details

BioMarin will host a conference call and webcast to discuss second quarter 2015 financial results today, Monday, August 3, at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.

U.S. / Canada Dial-in Number: 877.303.6313
International Dial-in Number: 631.813.4734
Conference ID: 72822644

Replay Dial-in Number: 855.859.2056
Replay International Dial-in Number: 404.537.3406
Conference ID: 72822644

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 drisapersen, an exon skipping oligonucleotide, for which a marketing application has been submitted to FDA for the treatment of patients with Duchenne muscular dystrophy (DMD) with mutations in the dystrophin gene that are amenable to treatment with exon 51 skipping, 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, vosoritide (formerly referred to as BMN 111), a modified C-natriuretic peptide, which is currently in Phase 2 clinical development for the treatment of achondroplasia, BMN 044, BMN 045 and BMN 053, exon skipping oligonucleotides, which are currently in Phase 2 clinical development for the treatment of Duchenne muscular dystrophy (exons 44, 45 and 53), 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 1/2, 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 pegvaliase, talazoparib, reveglucosidase alfa, vosoritide, cerliponase alfa, 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 pegvaliase, talazoparib, reveglucosidase alfa, vosoritide and cerliponase alfa; 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 2014 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.

Non-GAAP Net Loss

The results for the three and six months ended June 30, 2015 and June 30, 2014 and the guidance for 2015 include both GAAP net loss and non-GAAP net loss. As used in this release, non-GAAP net loss is based on GAAP earnings before interest, taxes, depreciation and amortization and further adjusted to exclude certain non-cash stock compensation expense, non-cash contingent consideration expense and certain other nonrecurring material items (non-GAAP net loss). 

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.

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

The following reconciliation of the Company's GAAP Condensed Consolidated Statements of Operations to non-GAAP Net Income (Loss) provides the details of the effects of the non-GAAP adjustments on the components of the Company's operating results for each of the periods presented. Management believes that providing the effects of the non-GAAP adjustments on the components of operating results is useful to investors, and when reviewed in conjunction with the Company's GAAP results, provides additional information regarding key drivers of the Company's core operations. The Company uses operating results on both a GAAP an non-GAAP basis internally for operating, budgeting and financial planning purposes.

The following summarizes the reconciliation of the components of the non-GAAP adjustments to Cost of sales, Research and development and Selling, general and administrative expenses from the GAAP to the non-GAAP presentation (in millions of U.S. dollars, unaudited):