On October 26, 2022 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported that financial results for the third quarter ended September 30, 2022 (Press release, BioMarin, OCT 26, 2022, View Source [SID1234622394]).
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"As anticipated, BioMarin is on-track to deliver double-digit revenue growth and profitability for the full-year 2022, underscored by our record year-to-date operating results. VOXZOGO demand is driving our financial performance and we expect additional launches in Japan and other global markets to further accelerate sales of this innovative product," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "Our third quarter and year-to-date performance not only showcased the continuing success of our VOXZOGO commercial launch, but also the European regulatory approval of ROCTAVIAN, the world’s first gene therapy approved for the treatment of severe hemophilia A. The launch in the EU is underway and, in the United States, the BLA for ROCTAVIAN was accepted by the FDA with an assigned PDUFA target action date of March 31, 2023. With two key product approvals and commercial launches over the past 12-months, the foundation of our 5-year strategic plan is in place."
Financial Highlights:
Total Revenues for the third quarter of 2022 were $505.3 million, an increase of 24% compared to the same period in 2021 despite continued erosion of the U.S. KUVAN market, and incremental foreign exchange headwinds. The increase in Total Revenues was primarily attributed to the following:
Higher VOXZOGO commercial sales due to new patients initiating therapy globally following regulatory approvals by the European Medicines Agency (EMA) and the FDA in the third and fourth quarters of 2021, respectively and
Higher NAGLAZYME and VIMIZIM product revenues primarily driven by the timing of orders in countries that place large government orders, particularly in Europe and Latin America and new patients initiating therapy in Europe and the Middle East; partially offset by
Lower KUVAN product revenues primarily due to generic competition as a result of the loss of exclusivity in the U.S., consistent with expectations.
GAAP Net Loss decreased to $6.7 million for the third quarter of 2022 compared to GAAP Net Loss of $36.5 million for the same period in 2021. The decrease was primarily related to higher gross profit driven by increased sales volume, partially offset by higher selling, general and administrative (SG&A) expenses and a higher tax provision. The increase in SG&A expenses was largely due to higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN, higher foreign currency exchange losses and severance costs associated with the Company’s organizational redesign announced in October 2022. The increase to the tax provision was primarily attributed to higher year-to-date income driven by increased gross profits and the net gain on the sale of the Priority Review Voucher during the first quarter of 2022.
Non-GAAP Income increased to $82.7 million for the third quarter of 2022 compared to Non-GAAP Income of $33.5 million for the same period in 2021 driven by higher gross profit due to increased sales volume partially offset by higher SG&A expenses largely driven by higher costs to support the commercial launch of VOXZOGO and ROCTAVIAN and higher foreign currency losses.
New Product Approvals and Launches (ROCTAVIAN and VOXZOGO)
Following EMA approval in the quarter, the commercial launch of ROCTAVIAN is now underway. It is estimated that approximately 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin’s footprint covered by the EMA approval, there are an estimated 3,200 patients who are indicated for ROCTAVIAN based on the current label.
To determine eligibility for ROCTAVIAN, treating physicians in countries covered by the EMA approval can use a companion diagnostic (CDx) test to ensure that patients do not have pre-existing antibodies to AAV5. The CDx test is CE-marked and designed to ensure the highest safety standards for use in determining patient eligibility for treatment with ROCTAVIAN.
On October 12, 2022, BioMarin’s resubmission of the BLA for ROCTAVIAN was accepted by the FDA with a PDUFA target action date of March 31, 2023. The FDA recently communicated plans to hold an advisory committee meeting but has yet to provide a date. If approved, ROCTAVIAN would be the first gene therapy in the U.S. for the treatment of severe hemophilia A.
At present, in the U.S. the Premarket Approval (PMA) application is under review at the Center for Devices and Radiological Health to support contemporaneous approval of a CDx along with the ROCTAVIAN BLA.
The global expansion of VOXZOGO is actively underway, with market access and reimbursement progressing as anticipated. As of September 30, 2022, there were 29 active markets contributing to VOXZOGO sales with an estimated 713 children being treated, as compared to an estimated 446 children as of June 30, 2022.
In the quarter, VOXZOGO became commercially available in Japan resulting in meaningful contributions from the early launch. Japan accounts for approximately half of the 1,500 patient opportunity in the Asia-Pacific region.
Mid-stage Product Life Cycle Expansion Opportunities (VOXZOGO and ROCTAVIAN)
During the quarter, the Company held discussions with global regulatory health authorities regarding the favorable results from the Phase 2 randomized, double-blind, placebo-controlled VOXZOGO study in infants and young children up to five years of age with achondroplasia. Based on these interactions, BioMarin intends to submit supplemental marketing applications by the end of 2022 in the U.S. and EU to expand access to VOXZOGO treatment for this younger age group.
Product expansion opportunities with ROCTAVIAN are supported by a number of clinical studies currently underway. The Phase 3b study to evaluate ROCTAVIAN with prophylactic corticosteroids has completed enrollment and is expected to read-out in early 2023. Two additional studies, one investigating ROCTAVIAN treatment in those with active or prior inhibitors, as well as one study investigating 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))
BMN 255 for primary hyperoxaluria, a prognostic factor for chronic renal disease: The Company is proceeding with the multi-ascending dose phase of the First-in-Human study with BMN 255. BioMarin believes the availability of a potent, orally bioavailable, small molecule like BMN 255 may be able to significantly reduce disease and treatment burden in certain people with chronic renal disease.
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, including dose escalation to the 6e13vg/kg dose, which our non-clinical studies project to provide therapeutic levels of C1-inhibitor.
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, upstream, 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. The IND is expected to be activated in early 2023 to enable initiation of the clinical phase of development.
BMN 349 for alpha-1 antitrypsin deficiency: Preclinical studies have demonstrated that BMN 349 is an orally bioavailable, small molecule that is titratable with rapid onset and high potency and efficacy. Preclinical results have strong implications for potential improvement of current management, particularly for severe liver disease requiring rapid action. IND enabling studies are well-underway and BioMarin’s goal is to file 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 gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. Mutations in MYBPC3 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 file an IND for BMN 293 in 2023.
BioMarin will host a conference call and webcast to discuss third quarter and year to date 2022 financial results today, Wednesday, October 26, 2022 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.