On July 28, 2021 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2021 (Press release, BioMarin, JUL 28, 2021, View Source [SID1234585271]).

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"We enjoyed a strong first half of 2021, including financial results and the achievement of our key regulatory goals," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "With the positive CHMP opinion for VOXZOGO for children, 2 years of age and older, our commercial team is eagerly preparing for potential European approval and launch later this summer. If approved, VOXZOGO would be the first pharmacological treatment option for children with achondroplasia. Following potential approval in Europe later this summer, we hope to have a positive outcome in the United States pending our November 20 PDUFA target action date. Upon potential global approvals of VOXZOGO for achondroplasia, we have a tremendous opportunity to address the interest from families seeking treatment for their children."

Mr. Bienaimé continued, "We were pleased to receive validation of our MAA in Europe for valoctocogene roxaparvovec for the treatment of severe hemophilia A, paving the way for a CHMP opinion in the first half of 2022. Our data presentations at the International Society on Thrombosis and Haemostasis in July provided further evidence of hemostatic efficacy following treatment with valoctocogene roxaparvovec, including significant reductions in bleeding events and Factor VIII infusions. We look forward to two year data results from our complete Phase 3 study with 134 participants in early 2022, and anticipate submitting a BLA in the second quarter of 2022, should data be supportive. More than ever, we believe that valoctocogene roxaparvovec gene therapy will be an important treatment option for people with severe hemophilia A. Given the superior bleed control seen at one year with valoctocogene roxaparvovec, and the burden of frequent, regular infusions with standard of care prophylaxis, we believe one infusion of valoctocogene roxaparvovec gene therapy has the potential to address the unmet need for bleed control, supported by our clinical results to date."

Financial Highlights:

Total Revenues for the second quarter of 2021 increased to $501.7 million, compared to $429.5 million for the same period in 2020. The increase in Total Revenues was primarily attributed to the following:
Higher Vimizim and Naglazyme product revenues primarily driven by timing of orders from Europe and Middle East; and
Higher Palynziq product revenues primarily driven by a combination of revenue from more U.S. patients achieving maintenance dosing and new patients initiating therapy; partially offset by
Lower Kuvan product revenues primarily due to generic competition due to the loss of exclusivity in the U.S.
GAAP Net Income increased to $12.9 million for the second quarter of 2021 compared to a GAAP Net Loss of $29.2 million for the same period in 2020. The increase was primarily due to profits from higher revenues and lower research and development (R&D) expense driven by lower in-licensing expenses.
Non-GAAP Net Income for the second quarter of 2021 increased to $97.8 million compared to Non-GAAP Income of $57.4 million for the same period in 2020. The increase in Non-GAAP Income for the quarter, compared to the same period in 2020, was primarily attributed to higher gross profit partially offset by higher SG&A expense.
Commercial Portfolio (Naglazyme, Vimizim, Brineura, Palynziq, Kuvan and Aldurazyme)

Naglazyme, Vimizim and Brineura maintained robust patient compliance in the quarter and continue to grow based on strong underlying demand.
The number of patients on commercial Naglazyme and Vimizim therapy increased by over 10% each year-over-year.
The number of commercial patients on commercial Brineura therapy increased by over 30% year-over-year.
Order timing for both Naglazyme and Vimizim for the full year 2021 are expected to be more concentrated in the first half of the year.
Palynziq top-line results for the full year 2021 are expected to increase approximately 40% as compared to full-year 2020 results based on the mid-point of full-year 2021 guidance.
Palynziq growth in the European, Middle East and African regions (EMEA) has been impacted by ongoing challenges due to COVID-19 with Palynziq revenue from EMEA expected to increase when PKU clinics have more freedom to operate and start treating additional patients.
The number of U.S. patients on commercial Palynziq therapy increased approximately 30% year-over-year and is expected to continue to grow as U.S. PKU clinics are expected to re-open over the coming quarters.
Loss of Kuvan U.S. market share to generics was consistent with expectations.
Late-stage Regulatory Portfolio (VOXZOGO and valoctocogene roxaparvovec)

In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of VOXZOGO for the treatment of children, ages 2 years and older. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in the third quarter of 2021.
The U.S. Food and Drug Administration (FDA) is reviewing the VOXZOGO New Drug Application (NDA) with the Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021.
The European Medicines Agency (EMA) validated BioMarin’s MAA for valoctocogene roxaparvovec: CHMP opinion anticipated in the second quarter of 2022.
BioMarin is targeting a Biologics License Application (BLA) submission for valoctocogene roxaparvovec in the second quarter of 2022 assuming favorable results from the two-year follow-up safety and efficacy data from the GENEr8-1 study, followed by an expected 6-month review procedure by the FDA.
Earlier-stage Development Portfolio (BMN 307, BMN 255, BMN 331, DiNA-001, Allen Institute Collaboration)

BMN 307: Dose escalation in PHEarless, the Phase 1/2 study of BMN 307 continues based on encouraging Phe lowering and safety profile observed in study participants who were treated with the lowest dose.
BMN 255 for a subset of chronic renal disease: BMN 255 is a small molecule for the treatment of a subset of chronic renal disease. The Investigational New Drug application (IND) for BMN 255 is active and the Company is dosing subjects.
BMN 331 gene therapy product candidate for Hereditary Angioedema (HAE): BMN 331 is BioMarin’s third gene therapy candidate. BioMarin plans to leverage its broad expertise in developing gene therapies for severe hemophilia A and PKU to improve efficiencies in the development process of BMN 331. The IND for BMN 331 was recently cleared by FDA and is active.
BMN 351 for Duchenne Muscular Dystrophy (DMD): IND-enabling studies continue with BMN 351, an antisense oligonucleotide therapy that has demonstrated dystrophin expression levels of 30-50% of wild-type levels in the quadriceps in a DMD mouse model treated at 18.7 mg/kg/week for 13 weeks (measured 2 weeks following last administration). If results from the ongoing pre-clinical studies are supportive, BioMarin anticipates filing an IND for BMN 351 in the first half of 2022.
DiNA-001 for MYBPC3 hypertrophic cardiomyopathy (HCM): Pre-clinical studies are underway with DiNA-001 following a collaboration announced in 2020 with DiNAQOR, a gene therapy platform company, to develop novel gene therapies to treat rare genetic cardiomyopathies. DiNAQOR received an undisclosed upfront payment and is eligible to receive development, regulatory and commercial milestones on product sales in addition to tiered royalties on worldwide sales.
On April 28, 2021, BioMarin and the Allen Institute announced a collaboration to create new gene therapies aimed at rare genetic diseases of the central nervous system (CNS). The goal is to combine the Allen Institute’s leadership in large-scale genomic science in the CNS therapeutic area with BioMarin’s expertise in developing transformational gene therapies.

BioMarin will host a conference call and webcast to discuss second quarter and full-year 2021 financial results today, Wednesday, July 28, 2021 at 4:30 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.biomarin.com.