BioMarin Announces Record Fourth Quarter and Full Year 2022 Total Revenues Driven by Strong Global Demand for VOXZOGO® and Steady Growth of Enzyme Business VOXZOGO $169 Million Contribution Drives Record Full Year 2022 Total Revenues of $2.1 Billion In 2023, More than 15% Growth in Total Revenues and...

SAN RAFAEL, Calif. , Feb. 27, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) (BioMarin or the Company) today announced financial results for the fourth quarter and full year ended December 31, 2022.

"As expected, in 2022 BioMarin delivered double-digit revenue growth and profitability for the full-year driven by the strong global launch of VOXZOGO, consistent growth of our enzyme business and continued focus on operational excellence," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Our record-setting 2023 outlook underscores BioMarin's proven and fully-scaled development and commercial capabilities and attention to sustainable growth. With global market expansion of VOXZOGO well underway, we turn our focus to the European commercial launch of ROCTAVIAN, the world's first gene therapy approved for the treatment of severe hemophilia A. Our team in Germany is working with the leading hemophilia centers of excellence to drive awareness and uptake of ROCTAVIAN, now that it is commercially available. In the United States , we are actively preparing for the launch of ROCTAVIAN upon potential approval this year. We are encouraged by the level of interest from U.S. adult hemophilia A patients seeking information about ROCTAVIAN and are pleased that roughly 300 patients have engaged directly with BioMarin to learn more. Acknowledging that many of these patients may not be eligible for treatment with ROCTAVIAN, we are glad to see this level of engagement with the bleeding disorders community."

BioMarin Announces Record Furth Quarter and Full Year 2022 Total Revenues

Financial Highlights:

Total Revenues for the fourth quarter of 2022 were $537.5 million , an increase of 19% compared to the same period in 2021 despite continued erosion of the U.S. KUVAN market. The increase in Total Revenues was primarily attributed to the following: Higher VOXZOGO commercial sales due to continued global market expansion and rapid patient uptake following regulatory approvals in late 2021 and early 2022, Higher NAGLAZYME product revenues primarily driven by new patients initiating therapy and the timing of orders in countries that place large government orders, particularly in Europe and the Middle East , and Higher ALDURAZYME product revenues primarily due to the timing of order fulfillment to Sanofi. BioMarin ALDURAZYME revenues are driven by the timing of when the product is released and control is transferred to Sanofi, Lower KUVAN product revenues primarily due to generic competition as a result of the loss of market exclusivity in the U.S., consistent with expectations. GAAP Net Loss decreased to $0.2 million for the fourth quarter of 2022 compared to GAAP Net Loss of $57.9 million for the same period in 2021. The decreased net loss was primarily related to higher gross profit, driven by increased sales volume. This was partially offset by higher selling, general and administrative (SG&A) and research and development (R&D) expenses. The increase in SG&A expenses was largely due to severance costs associated with the Company's organizational redesign announced in October 2022 and higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN in the EU. The increase in R&D expenses was primarily attributed to higher spend for programs in our earlier-stage development portfolio. Non-GAAP Income increased to $67.4 million for the fourth quarter of 2022 compared to Non-GAAP Income of $7.1 million for the same period in 2021 driven by higher gross profit due to increased sales volume partially offset by higher SG&A and R&D expenses for the same reasons noted above. New Product Approvals and Launches (ROCTAVIAN and VOXZOGO)

Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN)

Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))

Change in Non-GAAP Measures Beginning in 2023

Beginning with the first quarter of 2023, the Company defines Non-GAAP Income as GAAP Net Income excluding amortization of intangible assets, stock-based compensation expense, and certain other specified items. Reflecting this change in the Company's full year 2022 financial results as detailed above would have lowered the Company's full year 2022 Non-GAAP Income by $73.8 million and its full year 2022 Non-GAAP diluted earnings per share (EPS) by $0.38 . The Company is also introducing a new Non-GAAP financial measure, Non-GAAP Diluted EPS, which is defined as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. Refer to page 10 of this press release for a complete discussion of the Company's current Non-GAAP financial information and reconciliations to comparable information reported under U.S. GAAP.

2023 Full-Year Financial Guidance (in millions, except % and EPS amounts)

The full-year 2023 ROCTAVIAN revenue guidance range, provided above, represents global revenue estimates and assumes a U.S. approval in 2023, regardless of approval timing.

BioMarin will host a conference call and webcast to discuss fourth quarter and full year 2022 financial results today, Monday, February 27, 2023 at 4:30 p.m. ET . This event can be accessed through this link or on the investor section of the BioMarin website at www.biomarin.com.

Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The Company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The Company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit www.biomarin.com .

Forward-Looking Statements

BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ROCTAVIAN TM is a trademark of BioMarin Pharmaceutical Inc. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other brand names and service marks, trademarks and other trade names appearing in this release are the property of their respective owners.

Non-GAAP Information

The results presented in this press release include both GAAP information and Non-GAAP information. As used in this release for 2022 and 2021 periods presented, Non-GAAP Income is defined by the Company as GAAP Net Income (Loss) excluding net interest income (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 financial measure.

Non-GAAP Income and its components are not meant to be considered in isolation or 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 financial 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 financial measures; likewise, the Company may in the future cease to exclude items that it has historically excluded for purposes of its Non-GAAP financial measures. Because of the non-standardized definitions, the Non-GAAP financial 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 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.

The following table presents the reconciliation of 2023 guidance of GAAP Net Income and Diluted EPS to updated Non-GAAP Income and Non-GAAP Diluted EPS:

SOURCE BioMarin Pharmaceutical Inc.