BioMarin Announces Third Quarter 2019 Financial Results Financial Highlights (in millions of U.S. dollars, except per share data, unaudited) Three Months Ended September 30, Nine Months Ended September 30, ...

SAN RAFAEL, Calif. , Oct. 23, 2019 /PRNewswire/ --

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) (BioMarin or the Company) today announced financial results for the third quarter ended September 30, 2019.

Total Net Product Revenues for the third of quarter 2019 increased to $450.9 million , compared to $386.3 million for the third quarter of 2018. The increase in Net Product Revenues was attributed to the following:

The increase in GAAP Net Income for the third quarter of 2019, compared to GAAP Net Loss the same period in 2018 was primarily due to the following:

GAAP Net Income (Loss) Guidance for the full-year 2019 was narrowed to the low-end of the previously reported range to a loss of between $45 million to $65 million , reflecting continued expense management and strong net product sales.

Non-GAAP Income for the third quarter of 2019 increased $17.4 million , or 29%, to $78.1 million , compared to $60.7 million for the same period in 2018. The increase in Non-GAAP Income for the quarter, compared to the same period in 2018, was attributed to higher gross profit and driven by R&D expense management, partially offset by higher SG&A expense. The increase in Non-GAAP Income for the third quarter of 2019 resulted in narrowed full-year Non-GAAP income guidance to the high-end of the previously reported the range to between $150 million and $170 million .

As of September 30, 2019, BioMarin had cash, cash equivalents and investments totaling approximately $1.2 billion , as compared to $1.3 billion on December 31, 2018.

Commenting on third quarter results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "BioMarin is entering a stage that I believe will bring significant growth as we get closer to submitting marketing applications for valoctocogene roxaparvovec for severe hemophilia A and a pivotal data read-out for vosoritide for children with achondroplasia. These potential new products, combined with our strong base business and continued financial discipline, position us for significant growth and expansion beginning in the very near future. Later this quarter, both the U.S. and European marketing applications for valoctocogene roxaparvovec for severe hemophilia A will be submitted. If the applications are approved, we could potentially be launching the first approved gene therapy product in hemophilia A in the second half of 2020. We are also encouraged by the recent recommendation by the European Medicines Agency (EMA) to grant our request for accelerated assessment of valoctocogene roxaparvovec for severe hemophilia A. We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy will be the next wave of innovation for treating people with severe hemophilia A."

Mr. Bienaimé continued, "After valoctocogene roxaparvovec, vosoritide for the treatment of children with achondroplasia is our next significant commercial opportunity. Phase 3 results with vosoritide are expected by year-end. Our most recently initiated study with vosoritide, a global Phase 2 in infants and young children (less than 60 months old), is rounding the corner to complete enrollment. All subjects from ages 2 through 5 years have been enrolled. The second cohort, which includes children ages 6 months through 2 years old will complete by year-end, and the youngest cohort, infants up to 6 months old, has recently started enrolling. Interest in this study with very young children has been extremely robust, demonstrating that families are interested in treatment for their children as early as possible. Our most recently approved product, Palynziq for the treatment of phenylketonuria, is on a very strong launch trajectory since approval last year in the U.S. We have been very pleased with the pace of the U.S. launch, as we ended the third quarter with 670 patients on reimbursed Palynziq, and an additional 153 naïve patients having completed enrollment and awaiting their first injection. Building on this success, and as part of our strategy to further build our presence in the PKU market, we submitted the clinical trial application (CTA) for BMN 307, our gene therapy product for PKU, in September. BMN 307 demonstrated lifetime normalization of Phe in a validated PKU mouse model, and as a result, we believe it has the potential to be an important new treatment and market expander as part of our PKU franchise."

Key Program Highlights

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of several commercial therapies and multiple clinical and preclinical product candidates.

For additional information, please visit www.biomarin.com .

Forward-Looking Statements

This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the expectations of Total Revenues, Net Product Revenues and expenses for BioMarin's commercial products, Cost of Sales, GAAP Net Loss, Non-GAAP Income and other specified income statement guidance for the full-year 2019; the financial performance of BioMarin as a whole; BioMarin's potential for significant growth and expansion: BioMarin anticipating significant milestones over the coming months; BioMarin's entitlement to receive of milestone payments and royalties from Allievex that licensed rights from BioMarin for tralesinidase alfa and Allievex's assumption of financial obligations related to the development and commercialization of tralesinidase alfa; the timing of BioMarin's clinical development and commercial prospects, including (i) BioMarin's planned submissions to regulatory authorities, including marketing authorization applications for valoctocogene roxaparvovec in both the U.S. and Europe , (ii) BioMarin's clinical studies and trials, (iii) completion of enrollment of those studies and trials, including enrollment in BioMarin's Phase 3 program with valoctocogene roxaparvovec and global Phase 2 study with vosoritide, and (iv) announcements of data from those studies and trials, including BioMarin's Phase 3 program with valoctocogene roxaparvovec and the global Phase 3 study of vosoritide; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) BioMarin's planned submission of marketing authorization applications for valoctocogene roxaparvovec, which if approved could potentially be the first approved gene therapy product for hemophilia A, (ii) product to support clinical evaluation of BMN 307 being produced using a commercial scale manufacturing process to facilitate rapid clinical development, (iii) the possible approval and commercialization of BioMarin's product candidates, including vosoritide for the treatment of achondroplasia, and (iv) the Company's securing of reimbursement for Palyzniq on a country-by-country basis in the EU and Palynziq's revenue from the EU in 2020.

BioMarin®, Brineura®, Firdapse®, Kuvan®, Naglazyme®, Palynziq® and Vimizim® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. Aldurazyme® is a registered trademark of BioMarin/Genzyme LLC.

The following table presents Net Product Revenues by Product:

The following table presents Net Product Revenues for the PKU Franchise by Product:

Non-GAAP Information

The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release, Non-GAAP Income is defined by the Company as GAAP Net Income/Loss excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items, as detailed below when applicable. In addition, BioMarin includes in this press release the effects of these adjustments on certain components of GAAP Net Income/Loss for each of the periods presented. In this regard, Non-GAAP Income and its components, including Non-GAAP Cost of Sales, Non-GAAP Research and Development expenses, Non-GAAP Selling, General and Administrative expense, Non-GAAP Intangible Asset Amortization and Contingent Consideration, Non-GAAP Gain on the Sale of Intangible Asset and Non-GAAP Benefit From Income Taxes are statement of operations line items prepared on the same basis as, and therefore components of, the overall Non-GAAP measures.

Non-GAAP Income and its components are not meant to be considered in isolation, as a substitute for, or superior to comparable GAAP measures and should be read in conjunction with the consolidated financial information prepared in accordance with GAAP. Investors should note that the Non-GAAP information is not prepared under any comprehensive set of accounting rules or principles and does not reflect all of the amounts associated with the Company's results of operations as determined in accordance with GAAP. Investors should also note that these Non-GAAP measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future there may be other items that the Company may exclude for purposes of its Non-GAAP measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP measures. Because of the non-standardized definitions, the Non-GAAP measure as used by BioMarin in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by other companies.

The following table presents the reconciliation of GAAP Net Income (Loss) to Non-GAAP Income:

The following reconciliation of the GAAP reported to the Non-GAAP information provides the details of the effects of the Non-GAAP adjustments on certain components of the Company's operating results for each of the periods presented.

SOURCE BioMarin Pharmaceutical Inc.