BioMarin Announces Second Quarter 2021 Financial Results and Corporate Updates

SAN RAFAEL, Calif., July 28, 2021 /PRNewswire/ --

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) today announced financial results for the second quarter ended June 30, 2021.

"We enjoyed a strong first half of 2021, including financial results and the achievement of our key regulatory goals," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "With the positive CHMP opinion for VOXZOGO for children, 2 years of age and older, our commercial team is eagerly preparing for potential European approval and launch later this summer.  If approved, VOXZOGO would be the first pharmacological treatment option for children with achondroplasia. Following potential approval in Europe later this summer, we hope to have a positive outcome in the United States pending our November 20 PDUFA target action date. Upon potential global approvals of VOXZOGO for achondroplasia, we have a tremendous opportunity to address the interest from families seeking treatment for their children."

Mr. Bienaimé continued, "We were pleased to receive validation of our MAA in Europe for valoctocogene roxaparvovec for the treatment of severe hemophilia A, paving the way for a CHMP opinion in the first half of 2022. Our data presentations at the International Society on Thrombosis and Haemostasis in July provided further evidence of  hemostatic efficacy following treatment with valoctocogene roxaparvovec, including significant reductions in bleeding events and Factor VIII infusions. We look forward to two year data results from our complete Phase 3 study with 134 participants in early 2022, and anticipate submitting a BLA in the second quarter of 2022, should data be supportive.  More than ever, we believe that valoctocogene roxaparvovec gene therapy will be an important treatment option for people with severe hemophilia A. Given the superior bleed control seen at one year with valoctocogene roxaparvovec, and the burden of frequent, regular infusions with standard of care prophylaxis, we believe one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control, supported by our clinical results to date."

Financial Highlights:

• Total Revenues for the second quarter of 2021 increased to $501.7 million, compared to $429.5 million for the same period in 2020. The increase in Total Revenues was primarily attributed to the following:
• Higher Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and Middle East; and
• Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy; partially offset by
• Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S.
• GAAP Net Income increased to $12.9 million for the second quarter of 2021 compared to a GAAP Net Loss of  $29.2 million for the same period in 2020. The increase was primarily due to profits from higher revenues and lower research and development (R&D) expense driven by lower in-licensing expenses.
• Non-GAAP Net Income for the second quarter of 2021 increased to $97.8 million compared to Non-GAAP Income of $57.4 million for the same period in 2020. The increase in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to higher gross profit partially offset by higher SG&A expense.

Commercial Portfolio (Naglazyme, Vimizim, Brineura, Palynziq, Kuvan and Aldurazyme) 

• Naglazyme, Vimizim and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
• The number of patients on commercial Naglazyme and Vimizim therapy increased by over 10% each year-over-year.
• The number of commercial patients on commercial Brineura therapy increased by over 30% year-over-year.
• Order timing for both Naglazyme and Vimizim for the full year 2021 are expected to be more concentrated in the first half of the year.
• Palynziq top-line results for the full year 2021 are expected to increase approximately 40% as compared to full-year 2020 results based on the mid-point of full-year 2021 guidance.  
• Palynziq growth in the European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start treating additional patients.
• The number of U.S. patients on commercial Palynziq therapy increased approximately 30% year-over-year and is expected to continue to grow as U.S. PKU clinics are expected to re-open over the coming quarters.
• Loss of Kuvan U.S. market share to generics was consistent with expectations.

Late-stage Regulatory Portfolio (VOXZOGO and valoctocogene roxaparvovec)

• In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of VOXZOGO for the treatment of children, ages 2 years and older. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the third quarter of  2021.
• The U.S. Food and Drug Administration (FDA) is reviewing the VOXZOGO New Drug Application (NDA) with the Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021.
• The European Medicines Agency (EMA) validated BioMarin's MAA for valoctocogene roxaparvovec:  CHMP opinion anticipated in the second quarter of 2022.
• BioMarin is targeting a Biologics License Application (BLA) submission for valoctocogene roxaparvovec in the second quarter of 2022 assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, followed by an expected 6-month review procedure by the FDA.

Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, DiNA-001, Allen Institute Collaboration)

• BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
• BMN 255 for a subset of chronic renal disease: BMN 255 is a small molecule for the treatment of a subset of chronic renal disease. The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects.
• BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin's third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.  
• BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.
• DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
• On April 28, 2021, BioMarin and the Allen Institute announced a collaboration to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute's leadership in large-scale genomic science in the CNS therapeutic area with BioMarin's expertise in developing transformational gene therapies.

2021 Full-Year Financial Guidance (in millions, except %)

BioMarin will host a conference call and webcast to discuss second quarter and full-year 2021 financial results today, Wednesday, July 28, 2021 at 4:30 p.m. ET. This event can be accessed 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 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, 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 2021; cash flows from operating activities; the timing of orders for commercial products; expectations regarding the ongoing impact of the COVID-19 pandemic; 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) BioMarin's plans to re-submit a BLA for valoctocogene roxaparvovec to the FDA with two-year follow-up results from all the subjects from the Phase 3 GENEr8-1 study in the second quarter of 2022, (ii) BioMarin's anticipated IND submission for BMN 351 in the first half of 2022, and (iii) BioMarin and Allen Institute collaborating to create new gene therapies; the potential approval and commercialization of BioMarin's product candidates, including VOXZOGO for the treatment of achondroplasia and valoctocogene roxaparvovec for the treatment of severe hemophilia A; the timing of such approval decisions, including (i) the expectation of a CHMP opinion in the second quarter of 2022 for valoctocogene roxaparvovec, (ii) the expectation of a European Commission Decision in the third quarter of 2021 following the receipt of the positive CHMP opinion for VOXZOGO and (iii) the extended PDUFA target action date with respect to VOXZOGO of November 20, 2021; and the expected benefits and availability of BioMarin's product candidates, including that VOXZOGO would be the first potential pharmacological therapeutic option for children with achondroplasia; and potential growth opportunities and trends, including (i) that BioMarin would have a tremendous opportunity to address the interest from families seeking treatment for their children upon potential global approval of VOXZOGO, (ii) that BioMarin believes that one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control and (iii) growth expectations regarding Palynziq.

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; 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 March 31, 2021 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®, Brineura®, Kuvan®, Naglazyme®, Palynziq® and Vimizim® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. VOXZOGO™ is our trademark. 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.

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 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 (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.

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SOURCE BioMarin Pharmaceutical Inc.