On August 3, 2022 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2022 (Press release, BioMarin, AUG 3, 2022, View Source [SID1234617364]).
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BioMarin Announces Record Revenues in Second Quarter 2022; Increases Full-year 2022 Top and Bottom-line Guidance
"Continued strong growth of VOXZOGO and solid contributions from our other franchises drove record revenues exceeding $1 billion in the first half of the year, leading us to increase our full-year 2022 top and bottom-line guidance," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We also achieved many other significant milestones in the second quarter, including the CHMP’s positive opinion for conditional marketing authorization of Roctavian, the first gene therapy to be recommended for approval in Europe for hemophilia A. Assuming a positive decision from the European Commission, BioMarin’s commercial organization is ready to launch Roctavian in Europe in the third quarter. Based on the updated multi-year hemostatic efficacy observed to date following treatment with a single intravenous administration of Roctavian, we are on-track to resubmit the BLA in the U.S. to the FDA this September."
Mr. Bienaimé added, "In addition to continued robust demand for Voxzogo throughout the United States and Europe, underscored by today’s significant increase in full-year 2022 guidance for Voxzogo net product revenues to between $130 million and $160 million, we were thrilled to receive commercial approval in both Japan and Australia in the quarter. As we said previously, in 2022 we expect to return to double-digit revenue growth and profitability, and we are tracking to plan as demonstrated by record revenues in both the first and second quarters of this year."
Financial Highlights:
Total Revenues for the second quarter of 2022 were $533.8 million, an increase of 6% compared to the same period in 2021 despite continued erosion of the U.S. Kuvan market. The increase in Total Revenues was primarily attributed to the following:
Voxzogo commercial sales due to new patients initiating therapy in Europe and the U.S. following regulatory approvals by the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) in the third and fourth quarters of 2021, respectively.
Higher Aldurazyme product revenues due to timing of product fulfillment to Sanofi. BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Sanofi; 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 Income and Non-GAAP Income increased by $14.8 million and $11.6 million, respectively, for the second quarter of 2022 compared to the same period in 2021. The increase was primarily related to higher revenues partially offset by higher sales and marketing expenses to support the commercial launch of Voxzogo and pre-commercialization activities for Roctavian.
New Product Launches and Anticipated Approvals (Voxzogo and Roctavian)
The global launch of Voxzogo is actively underway, with market access and reimbursement progressing as anticipated. As of June 30, 2022, there were an estimated 446 children being treated with commercial Voxzogo globally, as compared to an estimated 284 children as of March 31, 2022. Of the estimated 446 children being treated with Voxzogo as of the end of the second quarter, 282 were from countries outside the U.S. and 164 were from the U.S. As of June 30, 2022, there were 20 active markets contributing to Voxzogo sales.
During the quarter, marketing authorization for Voxzogo was approved in both Japan and Australia, with commercial sales anticipated in these markets to begin in the third quarter of 2022. Japan accounts for approximately half of the 1,500 patient opportunity in the Asia-Pacific region.
During the quarter, the Company provided a top-line update on the Phase 2 randomized, double-blind, placebo-controlled Voxzogo study in infants and young children up to five years of age with achondroplasia at the 2022 Endocrine Society Annual Meeting (ENDO). BioMarin intends to initiate discussions on the favorable results from the study with regulatory health authorities to discuss next steps regarding efforts to expand access to Voxzogo treatment for this younger age group.
In the quarter, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending conditional marketing authorization for Roctavian, for adults with severe hemophilia A. The Company expects a final approval decision, which is typically consistent with the CHMP recommendation, from the European Commission in the third quarter of 2022.
BioMarin is targeting a BLA resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a 6-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.
In July, at the International Society on Thrombosis and Haemostasis 2022 Congress, the Company reported that durable hemostatic efficacy was maintained over six years in the ongoing Phase 1/2 study with Roctavian in the 6e13vg/kg dose cohort, representing the longest duration of clinical observation with any gene therapy treatment for adults with severe hemophilia A.
Mid-stage Product Life Cycle Expansion Opportunities (Voxzogo and Roctavian)
Also at the ENDO meeting, the investigator-initiated study with Voxzogo in children with selected genetic causes of short stature, preliminary 6-month results from 12 subjects demonstrated a positive response in all subgroups with interindividual variability. The 52-week study is ongoing, and is expected to complete in 2023.
In the quarter, the Company’s interventional Phase 2 study with Voxzogo for the treatment of infants under the age of two who are at risk for foramen magnum compression completed enrollment. The study is investigating the safety of Voxzogo in infants at risk of requiring surgery to alleviate compression at the foramen magnum, the opening in the base of the skull through which the spinal cord passes. The study will also measure a secondary endpoint to evaluate the effect of Voxzogo on growth of the foramen magnum volume through MRI scans.
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, are actively recruiting at sites around the world.
Earlier-stage Development Portfolio (BMN 255, BMN 331, BMN 351, BMN 349, BMN 293 (DiNA-001))
BMN 255 for primary hyperoxaluria type 1, a subset of chronic renal disease: The Company was recently given permission by the FDA to move forward with the multiple ascending dose portion 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): The Company announced that it has dosed patients in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with HAE.
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. BioMarin expects to file an IND for BMN 351 in the winter.
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. 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 relaxation kinetics and potentially ameliorate the disease phenotype in individuals suffering from cardiomyopathy. BioMarin’s goal is to file an IND for BMN 293 in 2023.
All Financial Guidance items are calculated based on U.S. GAAP with the exception of Non-GAAP Income. 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 corresponding GAAP reported information.
BioMarin will host a conference call and webcast to discuss second quarter and year to date 2022 financial results today, Wednesday, August 3, 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.