BioMarin Announces 20% Y/Y Total Revenue Growth in the Second Quarter and Increase in Full-year 2024 Guidance Second Quarter 2024 Total Revenues of $712 million (+20% Y/Y and +25% at Constant Currency Y/Y); First Half 2024 Total Revenues of $1.36 billion...

Second Quarter 2024 Total Revenues of $712 million (+20% Y/Y and +25% at Constant Currency Y/Y); First Half 2024 Total Revenues of $1.36 billion (+14% Y/Y and +19% at Constant Currency Y/Y)

Second Quarter 2024 GAAP Diluted EPS of $0.55 (+90% Y/Y); First Half 2024 GAAP Diluted EPS of $1.01 (+80% Y/Y)

Second Quarter 2024 Non-GAAP Diluted Earnings per Share (EPS) of $0.96 (+78% Y/Y); First Half 2024 Non-GAAP Diluted EPS of $1.67 (+46% Y/Y)

Strong VOXZOGO ® Demand in the Quarter Resulted in 73% Y/Y Increase in the Number of Children Receiving Treatment

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif. , Aug. 5, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) today announced financial results for the quarter and six months ended June 30, 2024.

"Strong execution across our business resulted in record double-digit revenue growth in the second quarter and first half of 2024. These top-line results, along with a focus on operational efficiency, resulted in a 78% year-over-year improvement in quarterly non-GAAP earnings per share," said Alexander Hardy , President and Chief Executive Officer of BioMarin. "Strong global demand for VOXZOGO led to nearly 900 new patient starts in the first half of 2024, the highest in VOXZOGO's history. Record VOXZOGO contributions in the quarter, driven by patient growth in all geographies, combined with double-digit growth from our enzyme therapies drove today's increased 2024 full-year guidance."

Mr. Hardy added, "During the quarter, global demand for VOXZOGO continued to increase as more families sought treatment with the only approved and genetically-targeted medicine for achondroplasia. Approximately 3,500 children were receiving VOXZOGO by the end of the second quarter, with more than half of new treatment starts in the United States for children under the age of 5. We are seeing strong interest from families in the United States seeking VOXZOGO treatment for their children and we expect this market to continue to drive significant expansion over the coming quarters." Mr. Hardy continued, "With VOXZOGO's safety and efficacy well-established, based on nearly 6,000 patient years of demonstrated and durable evidence, we are confident in our rapidly expanding leadership. Enrollment in our pivotal study with VOXZOGO for the treatment of hypochondroplasia is proceeding well, and enrollment in our separate studies for idiopathic short stature, Noonan Syndrome, Turner Syndrome and SHOX deficiency are advancing this year as planned, following alignment with health authorities."

Financial Highlights:

2Q Update on 2024 Strategic Priorities

In the second quarter, BioMarin continued to deliver on its four strategic priorities, first outlined in January, and focused on value creation through accelerating growth, optimizing efficiencies and driving operational excellence.

Accelerate and maximize the VOXZOGO opportunity

Establish ROCTAVIAN ® opportunity

Focus R&D on the most promising assets

Accelerate EPS growth and expand margins

Investor Day to be held Wednesday, September 4th . Event details will be available later in August.

2024 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated)

BioMarin will host a conference call and webcast to discuss second quarter 2024 financial results today, Monday, August 5, 2024, 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

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: future financial performance, including the expectations of Total Revenues, Non-GAAP Operating Margin percentage, and Non-GAAP Diluted EPS for the full-year 2024 and the underlying drivers of those results; BioMarin's new corporate strategy, including the timing of the completion and announcement; BioMarin's ability to accelerate the VOXZOGO opportunity; the anticipated benefits from its ongoing strategic review and the associated organizational redesign efforts; BioMarin's updated strategy for ROCTAVIAN and its anticipated benefits, including BioMarin's expectations regarding reduction of annual direct ROCTAVIAN expenses beginning in 2025 and ROCTAVIAN being profitable by the end of 2025; the timing of orders for commercial products; BioMarin's ability to meet product demand; 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, idiopathic short stature, Noonan Syndrome, Turner Syndrome, SHOX deficiency and other genetic short stature pathway conditions, (ii) the expected expansion of VOXZOGO in the U.S. and the anticipated start and growth of commercial sales of VOXZOGO in additional countries, (iii) the commercialization of ROCTAVIAN for the treatment of severe hemophilia A in the U.S., Germany and Italy , (iv) BioMarin's expectation to receive data regarding the first dose cohort for BMN 351 by year-end, (v) BioMarin's plans to enroll its first-in-human study with BMN 349 later this year, and (vi) BioMarin's plans to initiate a clinical program with BMN 333 in early 2025; the expected benefits and availability of BioMarin's product candidates; and potential growth opportunities and trends, including BioMarin's expectation that VOXZOGO product growth will continue to expand rapidly.

BioMarin ® , BRINEURA ® , KUVAN ® , NAGLAZYME ® , PALYNZIQ ® , ROCTAVIAN ® , VIMIZIM ® and VOXZOGO ® are registered trademarks of BioMarin Pharmaceutical Inc., or its affiliates. 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 excluding amortization of intangible assets, stock-based compensation 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 Operating Margin percentage is defined by the company as GAAP Income from Operations, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items, divided by GAAP Total Revenues. Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. The company's presentation of percentage changes in total revenues at constant currency rates, which is computed using current period local currency sales at the prior period's foreign exchange rates, is also a Non-GAAP financial measure. This measure provides information about growth (or declines) in the company's total revenue as if foreign currency exchange rates had not changed between the prior period and the current period.

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:

SOURCE BioMarin Pharmaceutical Inc.