• Full-year 2023 Total Revenues and Earnings Per Share Financial Guidance Narrowed; VOXZOGO^® and ROCTAVIAN^® Net Product Revenue Guidance Adjusted for Full-year 2023

• VOXZOGO Now Approved for Children without Age Restrictions in the United States and for Children Aged 4 Months and Older in Europe

• ROCTAVIAN Reimbursement Network On-track to Support Global Patient Access and Treatment in 2024 and Beyond; Final German and Italian Prices Expected in 2023; Over 100 Treatment Eligible Companion Diagnostic Tests Completed Globally

• For Full-year 2024, BioMarin Expects Revenues to Approach $3 Billion

SAN RAFAEL, Calif., Nov. 1, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the nine months and third quarter ended September 30, 2023.

"Results in the quarter drove double-digit revenue growth year-over-year and support BioMarin's full-year 2023 revenue and profitability objectives, set at the beginning of the year," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.

"We were very pleased to have recently received FDA approval for VOXZOGO for children under 5 years of age with achondroplasia, making it available in the U.S. for children of all ages with open growth plates. We also recently received approval in Europe to expand VOXZOGO treatment to children aged 4 months and older. These important age expansions will ensure that the youngest children treated with VOXZOGO have the opportunity to experience greater clinical benefit due to a longer potential treatment window. In parallel with exceptional commercial and regulatory execution of VOXZOGO during the quarter, we concluded key reimbursement steps to facilitate ROCTAVIAN treatment in Europe and the U.S. All together, these developments drive our expectation that total BioMarin revenues will approach $3 billion in 2024," said Mr. Bienaimé.

"In Germany, we have tentative agreement with the German Health Insurance Fund on the final ROCTAVIAN price, which we expect to be formalized in the coming weeks. With the final German price expected to be published by year-end, and 60 people in Germany eligible for next steps based on CDx testing results for AAV5 antibodies, we are encouraged by progress made in the quarter. We are also pleased to share that price negotiations with the Italian Medicines Agency are going well and that we expect a final price by year-end," Mr. Bienaimé added, "In the U.S. since gaining FDA approval of ROCTAVIAN on June 29, 2023, the commercial team has been building the reimbursement network to facilitate patient access, setting the stage for meaningful uptake of ROCTAVIAN in 2024."

Third Quarter Financial Highlights:

• Total Revenues for the third quarter of 2023 were $581.3 million, an increase of 15% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following:
  • higher VOXZOGO sales volume due to new patients initiating therapy across all regions;
  • higher PALYNZIQ product revenues primarily due to new patients initiating therapy, particularly in U.S.; partially offset by
  • lower ALDURAZYME product revenues primarily driven by the timing of order fulfillment to Sanofi, and
  • lower KUVAN product revenues attributed to increasing generic competition as a result of the loss of exclusivity in the U.S.
• GAAP and Non-GAAP Net Income of $40.4 million and $89.5 million resulted in increases of $47.1 million and $37.5 million for the third quarter of 2023 compared to the same period in 2022, respectively. The increased net income was primarily due to higher gross profit driven by increased revenues, as well as lower income tax expense, partially offset by higher spend in research and development programs to support both early-stage research and clinical activities and higher sales and marketing expenses to support the commercial launch of ROCTAVIAN.

Global Commercial Launches of ROCTAVIAN and VOXZOGO

• In the U.S., following the June 29, 2023 FDA approval of ROCTAVIAN, a one-time, gene therapy for the treatment of adults with severe hemophilia A, the company executed a number of critical steps to drive awareness among patients, physicians and patient advocates, as well as prepare Hemophilia Treatment Center (HTC) sites for patient uptake. At the end of the quarter, payers representing more than 205 million U.S. lives had published ROCTAVIAN coverage policies. Warranty policies had been secured by payers representing more than 95 million lives. As reimbursement and HTC readiness become more connected, U.S. patient testing and treatment will be more readily accessible.
• Today in Germany, the second commercial patient was treated with ROCTAVIAN. Over the last several weeks, the company and the German National Association of Statuary Health Insurance Funds (GKV) tentatively agreed on a final ROCTAVIAN price and expect to complete all formalities by year-end. In Germany, 60 people are eligible for next steps ahead of treatment with ROCTAVIAN based on CDx testing to determine seronegativity to AAV5. In Italy, final price negotiations with the Italian Medicines Agency are going well and are expected to be formalized by year-end 2023.
• As a result of global delays securing pricing and reimbursement, and other market preparations for ROCTAVIAN treatment, and proximity to the holiday season, full-year 2023 guidance has been lowered to less than $10 million.
• At the end of September 2023, approximately 2,320 children with achondroplasia were being treated with VOXZOGO across 38 active markets. In the third quarter, patient growth remained strong worldwide. Based on these trends, and the expectation that approximately 2,600 children will be receiving VOXZOGO treatment by year-end, today the company updated full-year 2023 VOXZOGO guidance to between $435 million and $455 million. Additionally, based on increased fill-finish manufacturing commitments, VOXZOGO supply is planned to increase from 2023 levels through the first and second quarters of 2024 and is expected to be fully unconstrained by mid-year 2024.
• On October 24, 2023, the European Commission adopted the decision to expand the indication for VOXZOGO to treat children with achondroplasia aged 4 months and older with open growth plates. VOXZOGO is approved for the treatment of children with achondroplasia of all ages with open growth plates in Japan and the U.S.

VOXZOGO and ROCTAVIAN Market Expansion Opportunities

• In the coming weeks, the company plans to begin the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia, a condition characterized by impaired bone growth. Hypochondroplasia is a genetic statural condition caused by a mutation (gene change) in the fibroblast growth factor receptor-3 (FGFR3) gene. The 6-month observation arm of the study will be followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, this study is expected to support regulatory approval in this large indication.
• The company is also preparing to initiate two additional clinical programs in 2024 with VOXZOGO, one in idiopathic short stature and one in genetic short stature conditions.
• Additional product expansion opportunities with ROCTAVIAN continue, including a clinical study investigating ROCTAVIAN treatment in those with active or prior inhibitors and continued exploration of methods of administering ROCTAVIAN in people with pre-existing antibodies against AAV5.

Earlier-stage Development Portfolio On-track; Seven Product Candidates Advancing

• BMN 255 for hyperoxaluria in chronic liver disease. The company believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in a patient population with significant unmet need. The company expects to have a determination of clinical proof of concept in 2024.
• BMN 331 gene therapy for Hereditary Angioedema (HAE) is in the Phase 1/2 HAERMONY study to evaluate this investigational AAV5-mediated gene therapy for people living with HAE. The company expects to dose additional patients with an optimized corticosteroid regimen with an anticipated clinical proof of concept determination by 2025.
• BMN 351 for Duchenne Muscular Dystrophy (DMD), is an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. The company is currently enabling a global clinical development plan and expects to have a determination of clinical proof of concept in 2025.
• BMN 349 for alpha-1 antitrypsin deficiency, an orally bioavailable, small molecule that preferentially sequesters mutant protein, preventing polymerization in liver cells that drive the progressive liver disease form of the illness. The company plans to initiate a global clinical program with BMN 349 in 2024 and expects to have a determination of clinical proof of concept in 2025.
• BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM). IND enabling studies are underway and have incorporated pre-IND feedback from the FDA. The company plans to initiate a global clinical program with BMN 293 in 2024 and expects to have a determination of clinical proof of concept in 2026.
• BMN 365 for AAV gene therapy for PKP2 arrhythmogenic cardiomyopathy. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2027.
• BMN 355 monoclonal antibody for long-QT syndrome. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2026.

BioMarin will host a conference call and webcast to discuss third quarter 2023 financial results and full-year financial guidance today, Wednesday, November 1, 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.

About BioMarin

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

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, Enzyme Product Revenues, Gross Profit, Research and Development Expense (R&D), Selling, General and Administrative Expense (SG&A), GAAP Net Income (Loss), Non-GAAP Income, GAAP Diluted EPS and Non-GAAP Diluted EPS for the full-year 2023; 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 potential to leverage VOXZOGO in conditions beyond achondroplasia, such as hypochondroplasia, and to expand the indication of VOXZOGO for children with achondroplasia aged 4 months and older in Europe, (ii) the results from clinical studies regarding product expansion opportunities for ROCTAVIAN, (iii) BioMarin's plans to observe the highest dose of BMN 255 in a patient population and, if the response is positive, engage to move to a pivotal Phase 2/3 study in 2024, (iv) with respect to BMN 331, BioMarin's plan to dose additional patients with an alternative corticosteroid regimen in late 2023, with an anticipated proof of concept by 2025, (v) BioMarin's plans to initiate a global clinical program with BMN 349 in 2024, (vi) BioMarin's plans to initiate a global clinical program with BMN 293 in 2024, and (vii) BioMarin's plans to initiate global clinical programs with BMN 365 and BMN 355 in 2025; the potential approval and commercialization of BioMarin's product candidates, including commercialization of ROCTAVIAN for the treatment of severe hemophilia A in the U.S. following FDA approval in June 2023, and the timing of such approval decisions and product launches, including (i) the anticipated start and growth of commercial sales of VOXZOGO in additional countries, and (ii) BioMarin's expectation that EU health authorities take action on its supplemental marketing application for VOXZOGO in the coming weeks and the number of additional children that will be eligible for VOXZOGO in the U.S. and, if such age expansions are accepted, in Europe; the expected benefits and availability of BioMarin's product candidates; and potential growth opportunities and trends, including that BioMarin expects accelerated growth of VOXZOGO revenues as the product launch continues in future quarters and that BioMarin expects growth of ROCTAVIAN revenues as the product's access is expanded in Europe and following commercial launch in the U.S.

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, impacts of macroeconomic and other external factors on BioMarin's operations; results and timing of current and planned 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; the market for each of these products; actual sales of BioMarin's commercial products; 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 June 30, 2023 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^®, VIMIZIM^® and VOXZOGO^® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. ROCTAVIAN^® is a trademark of BioMarin Pharmaceutical Inc., with registration in Europe and pending in the U.S. 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. Non-GAAP Income is defined by the company as GAAP Net Income (Loss) excluding amortization expense, stock-based compensation expense, contingent consideration expense, and, in certain periods, certain other specified items, as detailed below when applicable. The company also includes a Non-GAAP adjustment for the estimated tax impact of the reconciling items. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding

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, Non-GAAP Diluted EPS and Non-GAAP Diluted Shares 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 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 tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information: