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Mittwoch, 24.04.2024 16:03 von PR Newswire | Aufrufe: 154

BioMarin Reports Record Financial Results for the First Quarter 2024

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PR Newswire

SAN RAFAEL, Calif., April 24, 2024

First Quarter 2024 Total Revenues of $649 Million (+9% Y/Y and +13% at Constant Currency Y/Y); GAAP Diluted Earnings per Share (EPS) of $0.46 (+70% Y/Y) and Non-GAAP Diluted Earnings per Share of $0.71 (+18% Y/Y)

VOXZOGO^®Net Product Revenues of $153 Million in Q1'24 (+74% Y/Y); Children Treated Increased Over 100% Y/Y

For Full-year 2024 Guidance, Total Revenues Reaffirmed, Non-GAAP Operating Margin and Non-GAAP EPS Raised

R&D Prioritization Results in Acceleration of Three Highest Value Programs

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

SAN RAFAEL, Calif., April 24, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the quarter ended March 31, 2024.

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"During the quarter, execution across our business led to double digit revenue growth, on a constant currency basis, and an 18% increase in non-GAAP diluted earnings per share. At the same time, we made rapid progress on advancing our strategic priorities for the year, including accelerating and maximizing the VOXZOGO opportunity, focusing R&D on the most productive assets, and increasing profitability," said Alexander Hardy, President and Chief Executive Officer of BioMarin. "We were pleased with the outcome of our strategic R&D asset review, resulting in the acceleration and prioritization of the most potentially impactful medicines for patients. By advancing and focusing on these promising programs, we have taken decisive steps to drive value creation for all our stakeholders. This approach to delivering innovative therapies is an important part of BioMarin's operational transformation, and we look forward to sharing more details at Investor Day on September 4th."

Mr. Hardy continued, "Quarterly results were driven by strong demand for VOXZOGO, the only approved treatment for children with achondroplasia, and solid contributions from our established enzyme products. During the quarter, we made significant progress executing on our top priority to maximize and accelerate the reach of VOXZOGO, in achondroplasia and beyond. Globally, the number of children treated with VOXZOGO increased by over 500 from the prior quarter, and totaled over 3,100 by quarter end. In our largest single market, the U.S., we are realizing the tremendous opportunity ahead and expect U.S. uptake trends to continue. Our registration-enabling plans with VOXZOGO in hypochondroplasia, and ongoing discussions with health authorities to align on development plans for idiopathic short stature and pathway conditions are on-track, with all three studies expected to begin enrollment this year."

Financial Highlights:

Total Revenues for the first quarter of 2024 were $648.8 million, an increase of 9% compared to the same period in 2023. The increase in Total Revenues was primarily attributed to higher VOXZOGO sales volume driven by new patients initiating therapy across all regions. The increase was also due to higher PALYNZIQ product revenues primarily driven by sales volume growth in the U.S. The increase was partially offset by lower NAGLAZYME product revenues due to timing of orders in countries that place large government orders, particularly in the Middle East, as well as lower KUVAN product revenues attributed to continued generic competition as a result of the loss of market exclusivity.
GAAP Net Income increased by $37.8 million to $88.7 million in the first quarter of 2024 compared to the same period in 2023. The increase was primarily due to higher gross profit driven by increased VOXZOGO sales volume and a decrease in Other income (expense), net, related to an impairment charge in the first quarter of 2023, which did not recur in the first quarter of 2024. The increases were partially offset by higher spend in research and development (R&D) programs to support both early-stage research and clinical activities, including new indications with VOXZOGO, and higher spend in Selling, General and Administrative (SG&A) due to the continued support of global VOXZOGO market expansion and incremental administrative expenses in the quarter.
Non-GAAP Income increased by $23.9 million to $139.7 million in the first quarter of 2024 compared to the same period in 2023. The increased Non-GAAP Income was primarily due to higher gross profit driven by increased VOXZOGO revenues, partially offset by higher R&D expenses (as noted above).

1Q Update on 2024 Strategic Priorities

In the first quarter, BioMarin executed 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

During the first quarter, the number of children being treated with VOXZOGO for achondroplasia accelerated. In the period, more than 500 additional children began treatment with VOXZOGO, as compared to an increase of approximately 300 new treatment starts in the fourth quarter of 2023. Over 3,100 children were benefiting from VOXZOGO treatment across 43 active markets, as of the end of the first quarter. This represents a 102% increase in the number of children being treated with VOXZOGO, year-over-year.
In the largest single market, the U.S., more families with children under the age of 5 years sought treatment with VOXZOGO. This trend is expected to continue as real-world evidence, supported by VOXZOGO's extensive safety and efficacy profile, drives awareness and confidence for those families interested in treatment. With VOXZOGO now approved for children from infancy in most regions, families have the opportunity to benefit from longer treatment and potentially greater benefit.
Enrollment in the pivotal program with VOXZOGO for the treatment of children with hypochondroplasia continued. The observational run-in study is expected to complete enrollment by year-end, and the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial, is expected to start mid-year and is expected to complete enrollment in the first half of 2025.
During the quarter, BioMarin had productive engagement with the Food and Drug Administration to align on development programs in idiopathic short stature (ISS) and multiple genetic short stature pathway conditions (PC), and expects both studies to begin enrollment in the second half of 2024.

Establish ROCTAVIAN opportunity

During the quarter, reimbursement and market access challenges continued to impact the ability of interested patients to receive ROCTAVIAN treatment. For the remainder of 2024, BioMarin's global commercial team will continue to focus on key elements critical to supporting ROCTAVIAN uptake in the U.S., Italy and Germany. In April, BioMarin treated the first patient in Italy with ROCTAVIAN following the January price approval by the Italian Medicines Agency.

Focus R&D on the most productive assets

During the quarter, BioMarin completed a strategic portfolio assessment of R&D programs to determine which have the most transformative potential for patients and value creation for shareholders. With the combined focus on patient impact and commercial opportunity, three programs will be accelerated.
The programs, BMN 333, long-acting CNP for multiple growth-disorders, BMN 349, a first-in-class, oral therapeutic for AATD-associated liver disease, and BMN 351, BioMarin's next-generation oligonucleotide for DMD, all met the highest bar for advancement.
As a result of its prioritized portfolio, four programs will be discontinued, including BMN 331, BMN 255, BMN 355 and BMN 365. None of the programs were discontinued due to safety signals.
The additional programs in BioMarin's pipeline will move forward within the revised evaluation framework that provides a high bar for consistent and ongoing assessment to determine if they fit in the company's focused portfolio.

Accelerate EPS growth and expand margins

Strong performance in the first quarter underscored BioMarin's execution of its financial strategy to drive year-over-year Non-GAAP Operating Margin expansion and Non-GAAP EPS growth twice as fast as revenues.
The company reaffirmed full-year 2024 Total Revenue guidance, and raised Non-GAAP Operating Margin and Non-GAAP Diluted EPS guidance. The improved profitability guidance is a result of the planned reduction in operating expenses for the discontinued early-stage R&D programs, in the range of $50 million and $60 million. The planned reductions in R&D expense were partially off-set by planned increases in operating expenses for the accelerated programs, resulting in planned net reductions to 2024 operating expenses of $35 million to $40 million, driving the updated guidance. BioMarin's updated full-year 2024 guidance does not reflect the impact of potential additional future business decisions that may result from its ongoing strategic business review.

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

	Three Months Ended March 31,
	2024	2023	% Change

Total Revenues	$648.8	$596.4	9 %

Net Product Revenues by Product:
VIMIZIM^®	$192.6	$189.2	2 %
VOXZOGO	$152.9	$87.8	74 %
NAGLAZYME^®	$105.6	$123.0	(14) %
PALYNZIQ^®	$75.7	$62.4	21 %
BRINEURA^®	$39.0	$39.1	— %
KUVAN^®	$35.9	$50.5	(29) %
ALDURAZYME^®	$35.3	$34.4	3 %
ROCTAVIAN^®	$0.8	$—	nm

GAAP Net Income	$88.7	$50.9	74 %
Non-GAAP Income ⁽¹⁾	$139.7	$115.8	21 %
GAAP Operating Margin %⁽²⁾	13.6 %	10.5 %
Non-GAAP Operating Margin %⁽²⁾	23.8 %	22.4 %
GAAP Diluted Earnings per Share (EPS)	$0.46	$0.27	70 %
Non-GAAP Diluted EPS ⁽³⁾	$0.71	$0.60	18 %

	March 31, 2024		December 31, 2023
Total cash, cash equivalents & investments	$ 1,667.1		$ 1,684.9


(1)	Non-GAAP Income is defined by the company as reported GAAP Net Income, excluding amortization of intangible assets, stock-based compensation expense and, in certain periods, certain other specified items. The company also includes a Non-GAAP adjustment for the estimated income tax impact of reconciling items. Refer to Non-GAAP Information beginning on page 9 of this press release for a complete discussion of the company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP.

(2)	GAAP Operating Margin percentage is defined by the company as GAAP Income from Operations divided by Total Revenues. 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 specified items divided by Total Revenues.

(3)	Non-GAAP Diluted EPS is defined by the company as Non-GAAP Income divided by Non-GAAP diluted weighted-average shares outstanding. Non-GAAP weighted-average diluted shares outstanding is defined by the company as GAAP weighted-average diluted shares outstanding, adjusted to include any common shares issuable under the company's equity plans and convertible debt in periods when they are dilutive under Non-GAAP.

nm	Not meaningful

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

BioMarin does not provide guidance for GAAP reported financial measures (other than revenue) or a reconciliation of forward-looking Non-GAAP financial measures to the most directly comparable GAAP reported financial measures because the company is unable to predict with reasonable certainty the financial impact of changes resulting from our strategic portfolio and business operating model reviews; potential future asset impairments; gains and losses on investments; and other unusual gains and losses without unreasonable effort. These items are uncertain, depend on various factors, and could have a material impact on GAAP reported results for the guidance period. As such, any reconciliations provided would imply a degree of precision that could be confusing or misleading to investors.

Item

Provided February 22, 2024

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