On July 31, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the six months and second quarter ended June 30, 2023 (Press release, BioMarin, JUL 31, 2023, View Source,-Including-13-Year-Over-Year-Growth-Year-to-date [SID1234633536]).

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BioMarin Announces Record Breaking Revenues for the First Half of 2023
"Outstanding execution across our business led to record revenues in the first half of 2023. We reached more children with VOXZOGO around the world, as physicians and families sought treatment with the only approved medicine targeting the genetic cause of achondroplasia," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We were also very pleased to have received the highly anticipated U.S. approval of ROCTAVIAN, the only gene therapy treatment for severe hemophilia A. U.S. commercial launch activities are well underway following the June 29 approval, in parallel with launch progress across a number of European countries."

Mr. Bienaimé added, "for the remainder of 2023, we plan to build on the foundation of growth and profitability achieved in the first half of the year, expand VOXZOGO globally and treat the first ROCTAVIAN patients in the U.S. and Europe."

Financial Highlights:

Total Revenues for the second quarter of 2023 were $595.3 million, an increase of 12% compared to the same period in 2022. The increase in Total Revenues was primarily attributed to the following:
higher VOXZOGO sales volume due to continued global market expansion and rapid patient uptake across all regions;

higher PALYNZIQ product revenues primarily due to new patients initiating therapy, particularly in the U.S.; partially offset by

lower NAGLAZYME product revenues primarily driven by the timing of orders in countries that place large government orders, particularly in Latin America.

GAAP and Non-GAAP Net Income increased by $28.3 million and $28.4 million, respectively, for the second quarter of 2023 compared to the same period in 2022. The increased net income was primarily due to higher gross profit and interest income, partially offset by higher spend in research and development programs to support both early-stage research and clinical activities, as well as higher selling, general and administrative expenses due to higher foreign currency losses and to support the commercial launches of VOXZOGO and ROCTAVIAN.
Recent Product Approvals and Launches (ROCTAVIAN and VOXZOGO)

On June 29, 2023 the FDA approved ROCTAVIAN gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The FDA approval is based on data from the global Phase 3 GENEr8-1 study, the largest Phase 3 trial of any gene therapy in hemophilia. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A in the U.S. ROCTAVIAN was first conditionally approved by the European Commission in August 2022.

Following FDA approval, the Company activated its U.S.-based salesforce and communicated that ROCTAVIAN is expected to be available for commercial use in August. BioMarin estimates that there are approximately 2,500 people living with severe hemophilia A in the United States who are eligible for treatment and receiving care at approximately 140 hemophilia treatment centers.

In Europe, BioMarin continues to make progress on the pricing and reimbursement process for ROCTAVIAN in Germany, France and Italy to facilitate access. BioMarin is working directly with the German National Association of Statuary Health Insurance Funds (GKV) to finalize access to ROCTAVIAN. 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 Outcomes Based Agreements. In France and Italy, BioMarin is working directly with the single public insurance funds in each country to secure reimbursement and access to ROCTAVIAN, expected later in 2023.

As of the end of June 2023, more than 2,000 children with achondroplasia were being treated with VOXZOGO across 36 active markets. In the second quarter, patient growth remained strong worldwide. Based on these trends, today BioMarin updated full-year 2023 VOXZOGO guidance to between $400 million and $440 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.
VOXZOGO and ROCTAVIAN Market Expansion Opportunities

Today, BioMarin announced its plan to begin enrollment in 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.

Leveraging years of safety data from the VOXZOGO development program in achondroplasia, emerging data from an investigator-led Phase 2 study and following receipt of feedback from FDA, BioMarin plans to begin the 6-month observation arm of the study later this year, followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, BioMarin believes this study will be able to support regulatory approval in this large indication.

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 and the importance of starting treatment as early as feasible. Age expansions would provide access to treatment with VOXZOGO to more than 1,000 additional children in the U.S. and Europe.

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 (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293)

BioMarin plans to showcase its Research and Development capabilities and earlier-stage product candidate updates at its R&D Day on September 12, 2023. Details on accessing the live event will be available on BioMarin’s website in early September.

BMN 255 for hyperoxaluria in chronic liver disease: The Company has concluded the multi-ascending dose study with BMN 255 in healthy human volunteers. Based on early data demonstrating a rapid and potent increase in plasma glycolate following treatment with BMN 255, BioMarin plans to open enrollment in an expanded study in patients with chronic liver disease and hyperoxaluria in the second half of 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 2023, the second sentinel participant was safely dosed at 6e13vg/kg and this individual has had a similar initial response. BioMarin will continue to monitor the trajectory of expression in these two individuals before deciding on next steps in this program.

BMN 351 for Duchenne Muscular Dystrophy (DMD): Investigational New Drug application (IND)-enabling activities 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.

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 concluding and BioMarin plans to submit the IND in the second half of 2023.

BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM): 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. IND enabling studies are underway and have incorporated pre-IND feedback from the FDA. 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 July 31, 2023

Total Revenues

$2,375

to

$2,500

Unchanged

Enzyme Product Revenues(1)

$1,700

to

$1,850

Unchanged

ROCTAVIAN Revenues

$50

to

$150

Unchanged

VOXZOGO Revenues

$380

to

$430

$400

to

$440

Unchanged

Gross Profit %

77.5 %

to

79 %

Unchanged

R&D % of Revenue

30 %

to

32 %

Unchanged

SG&A % of Revenue

36 %

to

38 %

35.5 %

to

37.5 %

GAAP Net Income

$155

to

$205

$165

to

$215

GAAP Diluted EPS

$0.78

to

$1.03

$0.83

to

$1.08

Non-GAAP Income

$360

to

$410

$370

to

$420

Non-GAAP Diluted EPS

$1.80

to

$2.05

$1.85

to

$2.10

(1)

Enzyme-based Products include ALDURAZYME, BRINEURA, NAGLAZYME, PALYNZIQ and VIMIZIM.

BioMarin will host a conference call and webcast to discuss second quarter 2023 financial results today, Monday, July 31, 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.

U.S./Canada Dial-in Number: 888-886-7786

Replay Dial-in Number: 877-674-7070

International Dial-in Number: 416-764-8658

Replay International Dial-in Number: 416-764-8692

No Conference ID: 93773398

Conference ID: 773398 #