On April 26, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the first quarter ended March 31, 2023 (Press release, BioMarin, APR 26, 2023, View Source,-Including-15-Year-over-year-Growth-of-Total-Revenues [SID1234630514]).

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"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 Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.

Mr. Bienaimé continued, "During the quarter, with respect to ROCTAVIAN, we were pleased with progress made in Germany with physicians and those with severe hemophilia A interested in treatment. Specifically, we were encouraged by feedback from German hematologists interested in ROCTAVIAN, as well as the number of people pursuing antibody testing to determine their eligibility for treatment. To further support the hemophilia A community in Germany, today we announce the decision to work directly with the primary, public health insurer to facilitate access to ROCTAVIAN, rather than pursue additional Outcomes Based Agreements with sub-insurers, following months of complex negotiations. In the United States, we are actively preparing for the launch of ROCTAVIAN and are working with multiple key treatment centers to prepare for potential approval in June. Total revenue growth is tracking to plan, as supported by VOXZOGO accessibility to more families seeking treatment, our solid enzyme franchise, potential U.S. approval of ROCTAVIAN on the horizon and good progress on the commercial launch of ROCTAVIAN in Europe. 2023 is off to a great start and we are optimistic about the outlook ahead."

Financial Highlights:

Total Revenues for the first quarter of 2023 were $596.4 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 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 for the first quarter of 2023 compared to $120.8 million for the same period in 2022. The decreased net income was primarily due to the absence of the $89.0 million gain, net of tax, recognized in the first quarter of 2022 upon the sale to a third party the PRV the Company received in connection with FDA approval of VOXZOGO partially offset by an increase in gross profit.
Non-GAAP Income increased to $115.8 million for the first quarter of 2023 compared to Non-GAAP Income of $98.8 million for the same period in 2022 driven by higher gross profit due to increased sales volume partially offset by increased selling, general and administrative (SG&A) expenses to support the anticipated commercial launch of ROCTAVIAN, employee-related costs and other strategic initiatives.
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 in Japan and Brazil, followed by other markets, respectively. Based on these trends, today BioMarin updated full-year 2023 VOXZOGO guidance to between $380 million and $430 million. VOXZOGO is currently approved for the treatment of children 2 years old and older in Europe, for children 5 years old and older in the U.S., and approved for all ages from birth in Japan.
In Europe, BioMarin is making progress in Germany, France and Italy to facilitate access to ROCTAVIAN. In Germany, 18 people with severe hemophilia A have completed antibody testing to determine their seroprevalence to AAV5. On reimbursement in Germany, BioMarin is now working directly with the National 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 approximately 90% of the German population. At present, people in Germany with severe hemophilia A, who are eligible for treatment with ROCTAVIAN, can access treatment through either Named Patient authorizations or previously secured OBAs. In France and Italy, 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 of June 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 and $150 million. This updated ROCTAVIAN estimated revenue range assumes contributions from Europe, the U.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. and Europe, 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 the U.S. and Europe.
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 in New York City on September 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 April 26, 2023

Total Revenues

$2,375

to

$2,500

Unchanged

Enzyme Product Revenues(1)

$1,700

to

$1,850

Unchanged

ROCTAVIAN Revenues

$100

to

$200

$50

to

$150

VOXZOGO Revenues

$330

to

$380

$380

to

$430

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

$155

to

$205

Unchanged

GAAP Diluted EPS

$0.78

to

$1.03

Unchanged

Non-GAAP Income

$360

to

$410

Unchanged

Non-GAAP Diluted EPS

$1.80

to

$2.05

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 U.S. approval in June 2023.

BioMarin will host a conference call and webcast to discuss first quarter 2023 financial results today, Wednesday, April 26, 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.