On November 1, 2023 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported financial results for the nine months and third quarter ended September 30, 2023 (Press release, BioMarin, NOV 1, 2023, View Source [SID1234636626]).
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"Results in the quarter drove double-digit revenue growth year-over-year and supports BioMarin’s full-year 2023 revenue and profitability objectives, set at the beginning of the year," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin.
"We were very pleased to have recently received FDA approval for VOXZOGO for children under 5 years of age with achondroplasia, making it available in the U.S. for children of all ages with open growth plates. We also recently received approval in Europe to expand VOXZOGO treatment to children aged 4 months and older. These important age expansions will ensure that the youngest children treated with VOXZOGO have the opportunity to experience greater clinical benefit due to a longer potential treatment window. In parallel with exceptional commercial and regulatory execution of VOXZOGO during the quarter, we concluded key reimbursement steps to facilitate ROCTAVIAN treatment in Europe and the U.S. All together, these developments drive our expectation that total BioMarin revenues will approach $3 billion in 2024," said Mr. Bienaimé.
"In Germany, we have tentative agreement with the German Health Insurance Fund on the final ROCTAVIAN price, which we expect to be formalized in the coming weeks. With the final German price expected to be published by year-end, and 60 people in Germany eligible for next steps based on CDx testing results for AAV5 antibodies, we are encouraged by progress made in the quarter. We are also pleased to share that price negotiations with the Italian Medicines Agency are going well and that we expect a final price by year-end," Mr. Bienaimé added, "In the U.S. since gaining FDA approval of ROCTAVIAN on June 29, 2023, the commercial team has been building the reimbursement network to facilitate patient access, setting the stage for meaningful uptake of ROCTAVIAN in 2024."
Third Quarter Financial Highlights:
•Total Revenues for the third quarter of 2023 were $581.3 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 sales volume due to new patients initiating therapy across all regions;
◦higher PALYNZIQ product revenues primarily due to new patients initiating therapy, particularly in U.S.; partially offset by
◦lower ALDURAZYME product revenues primarily driven by the timing of order fulfillment to Sanofi; and
◦lower KUVAN product revenues attributed to increasing generic competition as a result of the loss of exclusivity in the U.S.
•GAAP and Non-GAAP Net Income of $40.4 million and $89.5 million resulted in increases of $47.1 million and $37.5 million for the third quarter of 2023 compared to the same period in 2022, respectively. The increased net income was primarily due to higher gross profit driven by increased revenues, as well as lower income tax expense, partially offset by higher spend in research and development programs to support both early-stage research and clinical activities and higher sales and marketing expenses to support the commercial launch of ROCTAVIAN.
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Global Commercial Launches of ROCTAVIAN and VOXZOGO
•In the U.S., following the June 29, 2023 FDA approval of ROCTAVIAN, a one-time, gene therapy for the treatment of adults with severe hemophilia A, the company executed a number of critical steps to drive awareness among patients, physicians and patient advocates, as well as prepare Hemophilia Treatment Center (HTC) sites for patient uptake. At the end of the quarter, payers representing more than 205 million U.S. lives had published ROCTAVIAN coverage policies. Warranty policies had been secured by payers representing more than 95 million lives. As reimbursement and HTC readiness become more connected, U.S. patient testing and treatment will be more readily accessible.
•Today in Germany, the second commercial patient was treated with ROCTAVIAN. Over the last several weeks, the company and the German National Association of Statuary Health Insurance Funds (GKV) tentatively agreed on a final ROCTAVIAN price and expect to complete all formalities by year-end. In Germany, 60 people are eligible for next steps ahead of treatment with ROCTAVIAN based on CDx testing to determine seronegativity to AAV5. In Italy, final price negotiations with the Italian Medicines Agency are going well and are expected to be formalized by year-end 2023.
•As a result of global delays securing pricing and reimbursement, and other market preparations for ROCTAVIAN treatment, and proximity to the holiday season, full-year 2023 guidance has been lowered to less than $10 million.
•At the end of September 2023, approximately 2,320 children with achondroplasia were being treated with VOXZOGO across 38 active markets. In the third quarter, patient growth remained strong worldwide. Based on these trends, and the expectation that approximately 2,600 children will be receiving VOXZOGO treatment by year-end, today the company updated full-year 2023 VOXZOGO guidance to between $435 million and $455 million. Additionally, based on increased fill-finish manufacturing commitments, VOXZOGO supply is planned to increase from 2023 levels through the first and second quarters of 2024 and is expected to be fully unconstrained by mid-year 2024.
•On October 24, 2023, the European Commission adopted the decision to expand the indication for VOXZOGO to treat children with achondroplasia aged 4 months and older with open growth plates. VOXZOGO is approved for the treatment of children with achondroplasia of all ages with open growth plates in Japan and the U.S.
VOXZOGO and ROCTAVIAN Market Expansion Opportunities
•In the coming weeks, the company plans to begin 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. The 6-month observation arm of the study will be followed by the 52-week randomized, double-blind, placebo-controlled phase of the 80-participant clinical trial. If successful, this study is expected to support regulatory approval in this large indication.
•The company is also preparing to initiate two additional clinical programs in 2024 with VOXZOGO, one in idiopathic short stature and one in genetic short stature conditions.
•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 On-track; Seven Product Candidates Advancing
•BMN 255 for hyperoxaluria in chronic liver disease. 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. The company expects to have a determination of clinical proof of concept in 2024.
•BMN 331 gene therapy for Hereditary Angioedema (HAE) is in the Phase 1/2 HAERMONY study to evaluate this investigational AAV5-mediated gene therapy for people living with HAE. The company expects to dose additional patients with an optimized corticosteroid regimen with an anticipated clinical proof of concept determination by 2025.
•BMN 351 for Duchenne Muscular Dystrophy (DMD), is an antisense oligonucleotide therapy for individuals with exon 51-skip-amenable DMD. The company is currently enabling a global clinical development plan and expects to have a determination of clinical proof of concept in 2025.
•BMN 349 for alpha-1 antitrypsin deficiency, 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. The company plans to initiate a global clinical program with BMN 349 in 2024 and expects to have a determination of clinical proof of concept in 2025.
•BMN 293 for MYBPC3 hypertrophic cardiomyopathy (HCM). IND enabling studies are underway and have incorporated pre-IND feedback from the FDA. The company plans to initiate a global clinical program with BMN 293 in 2024 and expects to have a determination of clinical proof of concept in 2026.
•BMN 365 for AAV gene therapy for PKP2 arrhythmogenic cardiomyopathy. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2027.
•BMN 355 monoclonal antibody for long-QT syndrome. The company is currently conducting IND-enabling studies and expects to initiate global clinical programs in 2025 with an anticipated clinical proof of concept determination by 2026.