BioMarin Announces Third Quarter 2022 Year-over-Year Total Revenue Growth of 24% (31% Excluding KUVAN®) VOXZOGO® $48 Million Contribution in the Quarter Results in BioMarin Raising Full-year 2022 VOXZOGO Net Product Revenue Guidance to Between $140...

SAN RAFAEL, Calif. , Oct. 26 , 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) (BioMarin or the Company) today announced financial results for the third quarter ended September 30, 2022.

"As anticipated, BioMarin is on-track to deliver double-digit revenue growth and profitability for the full-year 2022, underscored by our record year-to-date operating results. VOXZOGO demand is driving our financial performance and we expect additional launches in Japan and other global markets to further accelerate sales of this innovative product," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Our third quarter and year-to-date performance not only showcased the continuing success of our VOXZOGO commercial launch, but also the European regulatory approval of ROCTAVIAN, the world's first gene therapy approved for the treatment of severe hemophilia A. The launch in the EU is underway and, in the United States , the BLA for ROCTAVIAN was accepted by the FDA with an assigned PDUFA target action date of March 31, 2023 . With two key product approvals and commercial launches over the past 12-months, the foundation of our 5-year strategic plan is in place."

BioMarin Announces Third Quarter 2022 Year-over-year Total Revenue Growth of 24% (31% Excluding KUVAN)

Financial Highlights :

Total Revenues for the third quarter of 2022 were $505.3 million , an increase of 24% compared to the same period in 2021 despite continued erosion of the U.S. KUVAN market, and incremental foreign exchange headwinds. The increase in Total Revenues was primarily attributed to the following: Higher VOXZOGO commercial sales due to new patients initiating therapy globally following regulatory approvals by the European Medicines Agency (EMA) and the FDA in the third and fourth quarters of 2021, respectively and Higher NAGLAZYME and VIMIZIM product revenues primarily driven by the timing of orders in countries that place large government orders, particularly in Europe and Latin America and new patients initiating therapy in Europe and the Middle East ; partially offset by Lower KUVAN product revenues primarily due to generic competition as a result of the loss of exclusivity in the U.S., consistent with expectations. GAAP Net Loss decreased to $6.7 million for the third quarter of 2022 compared to GAAP Net Loss of $36.5 million for the same period in 2021. The decrease was primarily related to higher gross profit driven by increased sales volume, partially offset by higher selling, general and administrative (SG&A) expenses and a higher tax provision. The increase in SG&A expenses was largely due to higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN, higher foreign currency exchange losses and severance costs associated with the Company's organizational redesign announced in October 2022 . The increase to the tax provision was primarily attributed to higher year-to-date income driven by increased gross profits and the net gain on the sale of the Priority Review Voucher during the first quarter of 2022. Non-GAAP Income increased to $82.7 million for the third quarter of 2022 compared to Non-GAAP Income of $33.5 million for the same period in 2021 driven by higher gross profit due to increased sales volume partially offset by higher SG&A expenses largely driven by higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN and higher foreign currency losses. 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))

BioMarin will host a conference call and webcast to discuss third quarter and year to date 2022 financial results today, Wednesday, October 26, 2022 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.

About BioMarin 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 seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit www.biomarin.com.

Forward-Looking Statements

NEURA®, 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, Non-GAAP Income is defined by the Company as GAAP Net Income/Loss excluding net interest expense, provision for 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 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.