On April 27, 2022 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the first quarter ended March 31, 2022 (Press release, BioMarin, APR 27, 2022, View Source [SID1234613039]).

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"We begin 2022 from a position of financial strength including significant contributions from our newest product, Voxzogo, the only approved therapy for children with achondroplasia. We continue to be encouraged by the high level of interest in Voxzogo from families and physicians worldwide seeking treatment that addresses the underlying cause of achondroplasia," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "As we stated last quarter, in 2022 we expect to return to double-digit revenue growth and profitability. We begin the journey with record first quarter revenues, and foresee continued momentum based on the essential nature of our innovative medicines for our patients around the world."

BioMarin Announces Record Revenues in First Quarter 2022

Mr. Bienaimé continued, "The regulatory team has been working collaboratively with the European Medicines Agency as we near the completion of the application review procedure for potential approval of valoctocogene roxaparvovec gene therapy for the treatment of severe hemophilia A. We remain encouraged by the potential benefit of valoctocogene roxaparvovec for people with severe hemophilia A based on the clinically meaningful study results to date. These demonstrate an 85% reduction in annualized bleeding rates compared to baseline using standard of care. With potential approvals of valoctocogene roxaparvovec in Europe and United States, the continued strong launch of Voxzogo and our anticipated transition to sustainable profitability, we believe 2022 will be a transformational year for all BioMarin stakeholders."

Financial Highlights:

Total Revenues for the first quarter of 2022 were $519.4 million, an increase of 7% 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:

Higher Vimizim and Naglazyme product revenues primarily driven by new patients initiating therapy and timing of orders in the Middle East and Europe.

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 Brineura product revenues primarily attributed to new patients initiating therapy in Europe.
These factors were offset by the following:

Lower Aldurazyme product revenues due to timing of bulk lot product fulfillment to Sanofi. BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Sanofi.

Lower Kuvan product revenues primarily due to generic competition as a result of the loss of exclusivity in the U.S. that occurred in October 2020, consistent with expectations.
GAAP Net Income increased to $120.8 million for the first quarter of 2022 compared to GAAP Net Income of $17.4 million for the same period in 2021. The increase was primarily related to the $89.0 million gain, net of tax, on the sale to a third party of the Rare Pediatric Disease Priority Review Voucher (PRV) we received from the FDA in connection with the U.S. approval of Voxzogo and an increase in gross profit.
Non-GAAP Income for the first quarter of 2022 was $105.3 million, essentially flat compared to the same period in 2021. The increase in gross profit was offset by higher sales and marketing expenses to support the commercial launch of Voxzogo and pre-commercialization activities for valoctocogene roxaparvovec and higher research and development expenses driven by the ramp up of activities for early research programs.
Late-stage Regulatory Portfolio (Voxzogo and valoctocogene roxaparvovec)

The global launch of Voxzogo is actively underway, with market access and reimbursement progressing as anticipated. As of March 31, 2022, an estimated 284 children were being treated with commercial Voxzogo globally, 201 from countries outside the United States and 83 within the United States. As of March 31, 2022, there were 15 active markets contributing to Voxzogo sales, including the addition of Saudi Arabia, Slovenia, the Czech Republic, United Arab Emirates and Italy, since the February 2022 update.
Marketing authorization reviews of Voxzogo are in process in Japan and Australia, with potential approvals in those countries in 2022.
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. 52-week results trended in favor of Voxzogo compared to placebo on height Z-score, annualized growth velocity, and with no worsening in proportionality in the overall study population. BioMarin intends to initiate discussions with regulatory health authorities to discuss next steps regarding efforts to expand access to Voxzogo treatment for this younger age group. Results from this study are expected to be shared at a scientific meeting mid-year 2022.
The EMA continues the review of BioMarin’s Marketing Authorization Application (MAA) for valoctocogene roxaparvovec and we anticipate a CHMP opinion mid-year 2022. BioMarin has provided the EMA with two-year follow-up safety and efficacy data from the GENEr8-1 study.
Based on the favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, BioMarin is targeting a BLA resubmission for valoctocogene roxaparvovec in June 2022 followed by an expected 6-month review procedure by the FDA. A pre-submission interaction is scheduled with the FDA later this quarter to discuss BioMarin’s BLA resubmission efforts.
During the first quarter of 2022, the Company announced that a subject treated with valoctocogene roxaparvovec in the Phase 2 study over 5 years ago reported a salivary gland mass in late 2021. The event was reported as unrelated to valoctocogene roxaparvovec by the investigator. The subject was successfully treated, and the Company conducted a genomic analysis from a tissue sample containing the mass. Today, BioMarin announced that the findings from the completed analysis showed a comparable pattern of integration between healthy and tumor containing tissues, with no evidence emerging that vector integration contributed to the salivary gland mass. These data will be presented both in a workshop of the annual American Society of Gene & Cell Therapy meeting and the World Federation of Hemophilia 2022 World Congress in May, supplied to the EMA as part of the ongoing review of the MAA and included in the BLA resubmission.
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 has completed the single ascending dose arm of the First-in-Human study and is analyzing the results. 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 begun dosing patients in the Phase 1/2 HAERMONY study to evaluate BMN 331, an investigational AAV5-mediated gene therapy for people living with hereditary angioedema (HAE). The FDA granted Orphan Disease Designation status to BMN 331 in 2021.
BMN 351 for Duchenne Muscular Dystrophy (DMD): 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 first half of 2022, and anticipates treating clinical trial participants with Duchenne muscular dystrophy in the fourth quarter of 2022.
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 the IND 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 the IND in 2023.
2022 Full-Year Financial Guidance (in millions, except %)

(1) 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 first quarter and year to date 2022 financial results today, Wednesday, April 27, 2022 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.