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 Million and $170 Million
• U.S. Biologics Application for ROCTAVIAN™ Accepted by U.S. Food and Drug Administration (FDA); Prescription Drug User Fee Act (PDUFA) Target Action Date is March 31, 2023; Commercial Launch of ROCTAVIAN Underway in the European Union (EU) Following August 24, 2022 Approval

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."

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)

• Following EMA approval in the quarter, the commercial launch of ROCTAVIAN is now underway. It is estimated that approximately 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by the EMA approval, there are an estimated 3,200 patients who are indicated for ROCTAVIAN based on the current label.
• To determine eligibility for ROCTAVIAN, treating physicians in countries covered by the EMA approval can use a companion diagnostic (CDx) test to ensure that patients do not have pre-existing antibodies to AAV5. The CDx test is CE-marked and designed to ensure the highest safety standards for use in determining patient eligibility for treatment with ROCTAVIAN.
• On October 12, 2022, BioMarin's resubmission of the BLA for ROCTAVIAN was accepted by the FDA with a PDUFA target action date of March 31, 2023. The FDA recently communicated plans to hold an advisory committee meeting but has yet to provide a date. If approved, ROCTAVIAN would be the first gene therapy in the U.S. for the treatment of severe hemophilia A.
• At present, in the U.S. the Premarket Approval (PMA) application is under review at the Center for Devices and Radiological Health to support contemporaneous approval of a CDx along with the ROCTAVIAN BLA.
• The global expansion of VOXZOGO is actively underway, with market access and reimbursement progressing as anticipated. As of September 30, 2022, there were 29 active markets contributing to VOXZOGO sales with an estimated 713 children being treated, as compared to an estimated 446 children as of June 30, 2022.
• In the quarter, VOXZOGO became commercially available in Japan resulting in meaningful contributions from the early launch. Japan accounts for approximately half of the 1,500 patient opportunity in the Asia-Pacific region.

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

• During the quarter, the Company held discussions with global regulatory health authorities regarding the favorable results from the Phase 2 randomized, double-blind, placebo-controlled VOXZOGO study in infants and young children up to five years of age with achondroplasia. Based on these interactions, BioMarin intends to submit supplemental marketing applications by the end of 2022 in the U.S. and EU to expand access to VOXZOGO treatment for this younger age group.
• Product expansion opportunities with ROCTAVIAN are supported by a number of clinical studies currently underway. The Phase 3b study to evaluate ROCTAVIAN with prophylactic corticosteroids has completed enrollment and is expected to read-out in early 2023. Two additional studies, one investigating ROCTAVIAN treatment in those with active or prior inhibitors, as well as one study investigating ROCTAVIAN in people with pre-existing antibodies against AAV5.

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

• BMN 255 for primary hyperoxaluria, a prognostic factor for chronic renal disease: The Company is proceeding with the multi-ascending dose phase of the First-in-Human study with BMN 255. BioMarin believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in certain people with chronic renal disease.
• BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): Dosing continues in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE, including dose escalation to the 6e13vg/kg dose, which our non-clinical studies project to provide therapeutic levels of C1-inhibitor.
• BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling studies continue with BMN 351, an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. BMN 351 was developed using familiar chemistry and superior biology, by targeting a novel, upstream, splice enhancer site demonstrating improved binding affinity and tolerability in preclinical models. Preclinical data suggest that restored expression of near-full-length dystrophin protein at levels of up to 40% will convert phenotypes from rapid loss to durable preservation of strength and ambulation. The IND is expected to be activated in early 2023 to enable initiation of the clinical phase of development.
• BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that is titratable with rapid onset and high potency and efficacy. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are well-underway and BioMarin's goal is to file an IND for BMN 349 in the second half of 2023.
• BMN 293 (formerly DiNA-001) for MYBPC3 hypertrophic cardiomyopathy (HCM): Preclinical studies are underway with BMN 293 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. Mutations in MYBPC3 are the most common cause of inherited HCM. Early investigations suggest that gene therapy-mediated gene transfer can lead to widespread expression of the gene product, cardiac myosin-binding protein C (MyBP-C), in cardiac tissue, which can normalize cardiac hypertrophy, improve relaxation kinetics and potentially alleviate functional deficits in individuals suffering from cardiomyopathy. BioMarin's goal is to file an IND for BMN 293 in 2023.

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

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, Research and Development Expense, Selling, General and Administrative Expense, Cost of Sales, GAAP Net Loss, Non-GAAP Income, and other specified income statement guidance for the full-year 2022; cash flows from operating activities; the timing of orders for commercial products; the timing of BioMarin's clinical development and commercial prospects, including announcements of data from clinical studies and trials; the clinical development and commercialization of BioMarin's product candidates and commercial products, including (i) the outcome of BioMarin's BLA resubmission for ROCTAVIAN to the FDA (as well as the outcome of the PMA application submitted for contemporaneous approval of the related CDx test), (ii) the results from clinical studies regarding product expansion opportunities for ROCTAVIAN, (iii) BioMarin's plans to submit supplemental marketing applications in the U.S. and EU to expand access to VOXZOGO for infants and young children up to five years of age with achondroplasia by the end of 2022, (v) BioMarin's anticipated IND activation for BMN 351 in early 2023, (vi) BioMarin's anticipated IND submission for BMN 349 in the second half of 2023, and (vii) BioMarin's goal to file an IND for BMN 293 in 2023; the potential approval and commercialization of BioMarin's product candidates, including ROCTAVIAN for the treatment of severe hemophilia A, and the timing of such approval decisions and product launches, including (i) the anticipated start of commercial sales of VOXZOGO in additional countries, and (ii) the duration of the FDA's review procedure of our BLA resubmission for ROCTAVIAN, including the possibility that the FDA determines more review time is necessary based on the number of data read-outs that will emerge during the procedure; and the expected benefits and availability of BioMarin's product candidates; the anticipated benefits of BioMarin's organizational redesign plan announced in October 2022; and potential growth opportunities and trends, including that BioMarin expects (i) double-digit growth in revenues and profitability in 2022, and (ii) increasing access to VOXZOGO as the product launch continues in future quarters, including BioMarin's expectation that the addition of Japan and other global markets will increase the pace of uptake in demand for VOXZOGO.

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: BioMarin's success in the commercialization of its commercial products, including BioMarin's projected impact of the COVID-19 pandemic on its global revenue sources, including due to demand interruptions such as missed patient infusions and delayed treatment starts for new patients; results and timing of current and planned preclinical studies and clinical trials, as well as the potential impact of the COVID-19 pandemic on (i) BioMarin's ability to continue such preclinical studies and clinical trials and (ii) the timing of such preclinical studies and clinical trials, and the release of data from those trials; BioMarin's ability to successfully manufacture its commercial products and product candidates; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities concerning each of the described products and product candidates, including the potential impact of the COVID-19 pandemic on the regulatory authorities' abilities to issue such decisions and the timing of such decisions; the market for each of these products; actual sales of BioMarin's commercial products and the impact that the COVID-19 pandemic may have on such sales; the introduction of generic versions of BioMarin's commercial products, in particular generic versions of KUVAN; successful implementation of BioMarin's organizational redesign plan announced in October 2022; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 as such factors may be updated by any subsequent reports. 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®, BRI

NEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, VIMIZIM® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ROCTAVIANTM 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.

BioMarin regularly uses both GAAP and Non-GAAP results and expectations internally to assess its financial operating performance and evaluate key business decisions related to its principal business activities: the discovery, development, manufacture, marketing and sale of innovative biologic therapies. Because Non-GAAP Income and its components are important internal measurements for BioMarin, the Company believes that providing this information in conjunction with BioMarin's GAAP information enhances investors' and analysts' ability to meaningfully compare the Company's results from period to period and to its forward-looking guidance, and to identify operating trends in the Company's principal business. BioMarin also uses Non-GAAP Income internally to understand, manage and evaluate its business and to make operating decisions, and compensation of executives is based in part on this measure.

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.

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