BioMarin Announces Record Breaking First Quarter 2023 Results, Including 15% Year-over-year Growth of Total Revenues
BioMarin Pharmaceutical reported a 15% increase in total revenues for Q1 2023, reaching $596.4 million, driven by strong sales of VOXZOGO. This product saw revenue growth of 346%, indicating robust market uptake in Japan and Brazil. The company's enzyme product revenues also contributed positively, with ALDURAZYME showing a notable increase.
However, GAAP net income decreased to $50.9 million from $120.8 million in Q1 2022 due to the absence of a previous one-time gain. The company adjusted its revenue guidance for ROCTAVIAN, lowering expectations to between $50 million and $150 million, while reaffirming overall revenue guidance for 2023 between $2.375 billion and $2.5 billion.
BioMarin's operational focus includes preparations for ROCTAVIAN's launch in the U.S., with a PDUFA target action date set for June 30, 2023.
- Total revenues increased by 15%, reaching $596.4 million.
- VOXZOGO net product revenues surged by 346% to $87.8 million.
- ALDURAZYME revenue increased due to order fulfillment timing, enhancing overall revenue.
- GAAP net income dropped to $50.9 million, down from $120.8 million due to absence of prior gains.
- ROCTAVIAN revenue guidance was adjusted downward to $50 million - $150 million.
- Growth of VOXZOGO® Accelerated with Rapid Uptake in
Japan and Brazil Resulting in Increased Full-year 2023 Guidance - Full-year 2023 Top-line and Bottom-line Financial Guidance Reaffirmed; ROCTAVIAN™ Net Product Revenue Guidance Adjusted for Full-year 2023
- In
Germany , Growing Pipeline of People with Severe Hemophilia A Getting Tested for ROCTAVIAN Treatment Eligibility; ROCTAVIANU.S. Launch Preparations Underway Ahead ofJune 30 Prescription Drug User Fee Act (PDUFA) Target Action Date
Financial Highlights (in millions of | |||||
Three Months Ended | |||||
2023 | 2022 | % Change | |||
Total Revenues | $ 596.4 | $ 519.4 | 15 % | ||
Total Enzyme Product Revenues (1) | $ 448.1 | $ 426.5 | 5 % | ||
VIMIZIM® Net Product Revenues | $ 189.2 | $ 183.0 | 3 % | ||
NAGLAZYME® Net Product Revenues | $ 123.0 | $ 128.0 | (4) % | ||
VOXZOGO Net Product Revenues | $ 87.8 | $ 19.7 | 346 % | ||
PALYNZIQ® Net Product Revenues | $ 62.4 | $ 54.9 | 14 % | ||
KUVAN® Net Product Revenues | $ 50.5 | $ 59.3 | (15) % | ||
BRINEURA® Net Product Revenues | $ 39.1 | $ 36.2 | 8 % | ||
ALDURAZYME® Net Product Revenues | $ 34.4 | $ 24.4 | 41 % | ||
GAAP Net Income (2) | $ 50.9 | $ 120.8 | |||
Non-GAAP Income (3) | $ 115.8 | $ 98.8 | |||
GAAP Diluted Earnings per Share (EPS) | $ 0.27 | $ 0.63 | |||
Non-GAAP Diluted EPS (4) | $ 0.60 | $ 0.52 | |||
|
| ||
Total cash, cash equivalents & investments | $ 1,492.7 | $ 1,625.4 |
(1) | Enzyme-based products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ, and VIMIZIM. |
(2) | GAAP Net income in the first quarter of 2022, included a |
(3) | Non-GAAP Income is defined by the Company as reported GAAP Net Income/Loss, excluding amortization expense, stock-based compensation expense, contingent consideration, and, in certain periods, certain other specified items. The Company also includes a Non-GAAP adjustment for the estimated income tax effect 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 |
(4) | Non-GAAP Diluted EPS, is defined by the Company as Non-GAAP Income divided by Non-GAAP diluted shares outstanding. |
"We begin 2023 by delivering double-digit revenue growth in the first quarter driven by continued strong, global uptake of VOXZOGO, solid growth of our enzyme business and ongoing focus on operational excellence. In addition to record-breaking revenue, we were also pleased with continued profitability in the quarter," said
Financial Highlights:
- Total Revenues for the first quarter of 2023 were
$596.4 million 15% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following: - Higher VOXZOGO commercial sales due to continued global market expansion and rapid patient uptake following regulatory approvals in late 2021 and early 2022, and
- Higher ALDURAZYME product revenues primarily due to the timing of order fulfillment to Sanofi. BioMarin ALDURAZYME revenues are driven by the timing of when the product is released and control is transferred to Sanofi.
- GAAP Net Income decreased to
$50.9 million $120.8 million $89.0 million - Non-GAAP Income increased to
$115.8 million $98.8 million
Recent Product Approvals and Launches (VOXZOGO and ROCTAVIAN)
- As of the end of
March 2023 , approximately 1,500 children with achondroplasia were being treated with VOXZOGO across 35 active markets. In the first quarter, VOXZOGO growth accelerated inJapan andBrazil , followed by other markets, respectively. Based on these trends, todayBioMarin updated full-year 2023 VOXZOGO guidance to between$380 million $430 million Europe , for children 5 years old and older in theU.S. , and approved for all ages from birth inJapan . - In
Europe ,BioMarin is making progress inGermany ,France andItaly to facilitate access to ROCTAVIAN. InGermany , 18 people with severe hemophilia A have completed antibody testing to determine their seroprevalence to AAV5. On reimbursement inGermany ,BioMarin is now working directly with theNational Association of Statuary Health Insurance Funds (GKV) to finalize access to ROCTAVIAN, due to the complexity to reach final terms on the remaining Outcomes Based Agreements (OBAs) with sub-insurers. GKV is the primary provider of public health insurance, supplying coverage to approximately90% of the German population. At present, people inGermany with severe hemophilia A, who are eligible for treatment with ROCTAVIAN, can access treatment through either Named Patient authorizations or previously secured OBAs. InFrance andItaly ,BioMarin is working directly with the single public insurance funds in each country to secure reimbursement and access to ROCTAVIAN, which is expected later in 2023. - In March, the FDA extended review of the Biologics License Application (BLA) for ROCTAVIAN gene therapy for adults with severe hemophilia A. The FDA determined that the submission in the first quarter of the three-year data analysis from the ongoing Phase 3
GENEr8 -1 study, as requested by the FDA, constituted a Major Amendment due to the substantial amount of additional data and set a new PDUFA target action date ofJune 30, 2023 . - Based on the updated PDUFA target action date in the
U.S. , as well as the transition to working directly with GKV,BioMarin today lowered full-year 2023 ROCTAVIAN guidance to between$50 million $150 million Europe , theU.S. , and other markets, including Named Patient authorizations, in 2023.
Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN)
- In the coming months in the
U.S. andEurope , the Company expects to learn the outcome of its request to expand VOXZOGO access to younger age groups, based on favorable results from a Phase 2 study in infants and young children. If age expansions are accepted, more than 1,000 additional children will be eligible for VOXZOGO treatment in theU.S. andEurope . BioMarin is engaged in discussions with health authorities concerning the opportunity to leverage VOXZOGO, a natural regulator of bone growth, in other conditions characterized by impaired bone growth.- 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 (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))
BioMarin plans to showcase progress across its earlier-stage development pipeline at R&D Day inNew York City onSeptember 12, 2023 . Invitations to the event will be circulated in June.- BMN 255 for hyperoxaluria in chronic liver disease: The Company has concluded the multi-ascending dose study with BMN 255 in healthy human volunteers. In
January 2023 ,BioMarin shared early data that demonstrated a rapid and potent increase in plasma glycolate following treatment with BMN 255. Oral daily dosing at all tested levels for 14 days was safe and showed sustained elevations of plasma glycolate, which is predicted to have a profound reduction in oxalate excretion in patients.BioMarin now plans to initiate and enroll an expanded study in patients with chronic liver disease and hyperoxaluria in 2023. 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. - 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. In January 2023 ,BioMarin shared that the first participant treated with the 6e13vg/kg dose demonstrated C1-Inhibitor levels that were approaching the therapeutically relevant range. In March, the second sentinel participant was safely dosed at 6e13vg/kg. - 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, 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.BioMarin is working towards initiating clinical studies with BMN 351 in 2023. - BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is 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. In preclinical studies BMN 349 is titratable to effect, with rapid onset and high potency. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are underway and
BioMarin's goal is to submit 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 platform company that develops organ specific delivery of novel gene therapies to treat rare genetic cardiac and renal diseases. Mutations in the MYBPC3 gene 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 submit an IND for BMN 293 in the second half of 2023.
2023 Full-Year Financial Guidance (in millions, except % and EPS amounts) (Updated)
Item | 2023 Guidance | Updated | ||||||||||
Total Revenues | to | Unchanged | ||||||||||
Enzyme Product Revenues(1) | to | Unchanged | ||||||||||
ROCTAVIAN Revenues | to | to | ||||||||||
VOXZOGO Revenues | to | to | ||||||||||
Unchanged | ||||||||||||
Gross Profit % | 77.5 % | to | 79 % | Unchanged | ||||||||
R&D % of Revenue | 30 % | to | 32 % | Unchanged | ||||||||
SG&A % of Revenue | 36 % | to | 38 % | Unchanged | ||||||||
GAAP Net Income | to | Unchanged | ||||||||||
GAAP Diluted EPS | to | Unchanged | ||||||||||
Non-GAAP Income | to | Unchanged | ||||||||||
Non-GAAP Diluted EPS | to | Unchanged | ||||||||||
(1) | Enzyme-based Products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM. |
The full-year 2023 ROCTAVIAN revenue guidance range, provided above, represents global revenue estimates and assumes a
International Dial-in Number: 213-992-4616 | |
No Conference ID | Conference ID: 9184# |
About
Founded in 1997,
Forward-Looking Statements
This press release and the associated conference call and webcast contain forward-looking statements about the business prospects of
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 PHARMACEUTICAL INC. | |||
ASSETS | (unaudited) | ||
Current assets: | |||
Cash and cash equivalents | $ 580,074 | $ 724,531 | |
Short-term investments | 572,017 | 567,006 | |
Accounts receivable, net | 597,913 | 461,316 | |
Inventory | 918,921 | 894,083 | |
Other current assets | 173,180 | 104,521 | |
Total current assets | 2,842,105 | 2,751,457 | |
Noncurrent assets: | |||
Long-term investments | 340,635 | 333,835 | |
Property, plant and equipment, net | 1,068,142 | 1,073,366 | |
Intangible assets, net | 325,989 | 338,569 | |
196,199 | 196,199 | ||
Deferred tax assets | 1,510,568 | 1,505,412 | |
Other assets | 150,057 | 176,236 | |
Total assets | $ 6,433,695 | $ 6,375,074 | |
LIABILITIES AND STOCKHOLDERS' EQUITY | |||
Current liabilities: | |||
Accounts payable and accrued liabilities | $ 598,231 | $ 572,959 | |
Short-term contingent consideration | — | 15,925 | |
Total current liabilities | 598,231 | 588,884 | |
Noncurrent liabilities: | |||
Long-term convertible debt, net | 1,084,006 | 1,083,019 | |
Other long-term liabilities | 92,415 | 100,015 | |
Total liabilities | 1,774,652 | 1,771,918 | |
Stockholders' equity: | |||
Common stock, | 188 | 186 | |
Additional paid-in capital | 5,417,873 | 5,404,895 | |
Company common stock held by the Nonqualified Deferred Compensation Plan | (9,949) | (8,859) | |
Accumulated other comprehensive loss | (10,722) | (3,867) | |
Accumulated deficit | (738,347) | (789,199) | |
Total stockholders' equity | 4,659,043 | 4,603,156 | |
Total liabilities and stockholders' equity | $ 6,433,695 | $ 6,375,074 | |
(1) |
|
BIOMARIN PHARMACEUTICAL INC. | |||
Three Months Ended | |||
2023 | 2022 | ||
(unaudited) | (unaudited) | ||
REVENUES: | |||
Net product revenues | $ 586,426 | $ 505,525 | |
Royalty and other revenues | 9,989 | 13,834 | |
Total revenues | 596,415 | 519,359 | |
OPERATING EXPENSES: | |||
Cost of sales | 126,549 | 116,965 | |
Research and development | 171,846 | 160,836 | |
Selling, general and administrative | 223,003 | 194,619 | |
Intangible asset amortization and contingent consideration | 15,670 | 17,612 | |
Gain on sale of nonfinancial assets, net | — | (108,000) | |
Total operating expenses | 537,068 | 382,032 | |
INCOME FROM OPERATIONS | 59,347 | 137,327 | |
Interest income | 11,943 | 1,820 | |
Interest expense | (3,703) | (3,806) | |
Other expense, net | (10,830) | (1,154) | |
INCOME BEFORE INCOME TAXES | 56,757 | 134,187 | |
Provision for income taxes | 5,905 | 13,389 | |
NET INCOME | $ 50,852 | $ 120,798 | |
EARNINGS PER SHARE, BASIC | $ 0.27 | $ 0.66 | |
EARNINGS PER SHARE, DILUTED | $ 0.27 | $ 0.63 | |
Weighted average common shares outstanding, basic | 186,667 | 183,990 | |
Weighted average common shares outstanding, diluted | 194,363 | 194,886 | |
BIOMARIN PHARMACEUTICAL INC. | |||
Three Months Ended | |||
2023 | 2022 | ||
(unaudited) | (unaudited) | ||
CASH FLOWS FROM OPERATING ACTIVITIES: | |||
Net income | $ 50,852 | $ 120,798 | |
Adjustments to reconcile net income to net cash used in operating activities: | |||
Depreciation and amortization | 26,421 | 27,343 | |
Non-cash interest expense | 1,029 | 1,033 | |
Amortization of premium (accretion of discount) on investments | (1,970) | 1,652 | |
Stock-based compensation | 53,695 | 47,833 | |
Gain on sale of nonfinancial assets, net | — | (108,000) | |
Loss on equity investment | 12,650 | — | |
Deferred income taxes | (6,360) | 4,800 | |
Unrealized foreign exchange loss (gain) | 6,615 | (6,887) | |
Non-cash changes in the fair value of contingent consideration | — | 1,989 | |
Other | (222) | 700 | |
Changes in operating assets and liabilities: | |||
Accounts receivable, net | (138,796) | (54,813) | |
Inventory | (14,098) | 1,125 | |
Other current assets | (36,001) | (8,011) | |
Other assets | (323) | 1,440 | |
Accounts payable and other short-term liabilities | (31,686) | (78,143) | |
Other long-term liabilities | 4,262 | 1,710 | |
Net cash used in operating activities | (73,932) | (45,431) | |
CASH FLOWS FROM INVESTING ACTIVITIES: | |||
Purchases of property, plant and equipment | (24,456) | (28,817) | |
Maturities and sales of investments | 215,118 | 155,818 | |
Purchases of investments | (220,364) | (147,361) | |
Proceeds from sale of nonfinancial assets | — | 110,000 | |
Purchase of intangible assets | (310) | (1,858) | |
Net cash provided by (used in) investing activities | (30,012) | 87,782 | |
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from exercises of awards under equity incentive plans | 21,169 | 8,235 | |
Taxes paid related to net share settlement of equity awards | (51,422) | (32,949) | |
Payments of contingent consideration | (9,475) | — | |
Principal repayments of financing leases | (1,014) | (566) | |
Net cash used in financing activities | (40,742) | (25,280) | |
Effect of exchange rate changes on cash | 229 | 1,093 | |
NET INCREASE (DECREASE) IN CASH AND CASH EQUIVALENTS | (144,457) | 18,164 | |
Cash and cash equivalents: | |||
Beginning of period | $ 724,531 | $ 587,276 | |
End of period | $ 580,074 | $ 605,440 | |
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 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
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
The following tables present the reconciliation of GAAP reported to Non-GAAP adjusted financial information:
Reconciliation of GAAP Reported Net Income to Non-GAAP Income | |||
Three Months Ended | |||
2023 | 2022 | ||
GAAP Reported Net Income | $ 50.9 | $ 120.8 | |
Adjustments | |||
Stock-based compensation expense - COS | 4.3 | 4.3 | |
Stock-based compensation expense - R&D | 19.8 | 17.2 | |
Stock-based compensation expense - SG&A | 29.5 | 26.3 | |
Amortization of intangible assets | 15.7 | 15.6 | |
Contingent consideration | — | 2.0 | |
Gain on sale of non-financial assets (1) | — | (108.0) | |
Severance and employee termination benefits (2) | 2.1 | — | |
Loss on investment in equity securities (3) | 12.6 | — | |
Income tax effect of adjustments | (19.2) | 20.6 | |
Non-GAAP Income | $ 115.8 | $ 98.8 | |
Reconciliation of Certain GAAP Reported Information to Non-GAAP Information | |||
Three Months Ended | |||
2023 | 2022 | ||
GAAP Diluted EPS | $ 0.27 | $ 0.63 | |
Adjustments | |||
Stock-based compensation expense | 0.27 | 0.25 | |
Amortization of intangible assets | 0.08 | 0.08 | |
Contingent consideration | — | 0.01 | |
Gain on sale of non-financial assets (1) | — | (0.55) | |
Severance and employee termination benefits (2) | 0.01 | — | |
Loss on investment in equity securities (3) | 0.06 | — | |
Income tax effect of adjustments | (0.09) | 0.11 | |
Non-GAAP Diluted EPS | $ 0.60 | $ 0.52 | |
(1) | Represents the net gain on the sale in the first quarter of 2022 to a third party of the PRV the Company received from the FDA in connection with the |
(2) | Represents severance and employee termination benefit charge in SG&A related to the Company's organizational redesign announced in |
(3) | Represents the impairment loss on investment in non-marketable equity securities recorded in Other expense, net in the first quarter of 2023. |
Three Months Ended | ||||
2023 | 2022 | |||
GAAP Weighted-Average Dilutive Shares Outstanding | 194 | 195 | ||
Adjustments | ||||
Common stock issuable under convertible notes due in 2027 (1) | 5 | — | ||
Non-GAAP Weighted-Average Dilutive Shares Outstanding | 199 | 195 | ||
(1) | Common stock issuable under convertible notes due in 2027, were excluded from the computation of GAAP Weighted-Average Dilutive Shares Outstanding as they were anti-dilutive. |
Guidance for the Year Ended | ||||||||||
Net Income | Diluted Shares | Diluted EPS | ||||||||
GAAP Net Income and Diluted EPS | $ 155 | to | $ 205 | 200 | to | |||||
Amortization of intangible assets | 60 | 0.30 | ||||||||
Stock-based compensation expense | 200 | 1.00 | ||||||||
Severance and employee termination benefits | 2.1 | 0.01 | ||||||||
Loss on investment in equity securities | 12.6 | 0.06 | ||||||||
Income tax effect of adjustments (3) | (70) | (0.35) | ||||||||
Non-GAAP Income and Diluted EPS | $ 360 | to | $ 410 | 200 | to | |||||
(1) | The adjustments/reconciling items included in this table are presented to facilitate the reconciliation of Non-GAAP Income and Non-GAAP Diluted EPS to their closest GAAP financial metrics, GAAP Net Income and GAAP Diluted EPS. The specific amounts included in each reconciling line item above represent approximations of the underlying adjustments from GAAP Net Income to Non-GAAP Income and from GAAP Diluted EPS to Non-GAAP Diluted EPS. Actual 2023 results for each reconciling line item may be different, in some cases materially, than the amounts listed above as a result of uncertainty regarding, and the potential variability of, those items. |
(2) | Certain amounts may not sum or recalculate due to rounding. |
(3) | Income tax adjustments represent the estimated income tax impact of each pre-tax non-GAAP adjustment based on the applicable statutory income tax rate. |
Contact: | ||
Investors: | Media: | |
(415) 455-7558 | (650) 374-2803 |
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FAQ
What were BioMarin's total revenues for Q1 2023?
How did VOXZOGO perform in Q1 2023?
What is the adjusted revenue guidance for ROCTAVIAN in 2023?
When is the PDUFA target action date for ROCTAVIAN?
How did BioMarin's GAAP net income change in Q1 2023 compared to 2022?
