- Full-year 2020 Total Revenues Increased 9% to $1.86 billion

- Company Dose-escalates in PHEarless, a Phase 1/2 Study with BMN 307 Gene Therapy for the Treatment of Phenylketonuria (PKU); the Third Potential Therapeutic Modality in its PKU Franchise

- During the Fourth Quarter, BioMarin Announced Benefit Maintained for Over Two Years in Children with Achondroplasia Treated with Vosoritide in Phase 3 Extension Study

- Marketing Applications for Vosoritide to Treat Children with Achondroplasia under Review in Europe and the U.S.; Committee for Medicinal Products for Human Use (CHMP) Opinion Expected in June 2021

- In January 2021, BioMarin Announced Positive Phase 3 Results with Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A; Study Met All Primary and Secondary Efficacy Endpoints in One-Year Data Set; Mean Annualized Bleed Rate was 0.9 in Subset Dosed More than Two Years Ago

- Submission of Phase 3 Results from 134 Subjects with 1 to 2 Years of Data with Valoctocogene Roxaparvovec for Severe Hemophilia A to the European Medicines Agency (EMA) Targeted in 2Q21

SAN RAFAEL, Calif., Feb. 25, 2021 /PRNewswire/ --

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) today announced financial results for the fourth quarter and full year ended December 31, 2020.

Net Product Revenues for the fourth quarter of 2020 were essentially flat as compared to the fourth quarter of 2019. The change in Net Product Revenues was primarily attributed to the following:

• Kuvan Net Product Revenues decreased by $33.6 million, primarily due to the U.S. loss of market exclusivity in October 2020 resulting from generic competition; and
• Aldurazyme Net Product Revenues decreased by $22.7 million due to timing of product fulfillment to Genzyme. Aldurazyme is marketed by Genzyme and BioMarin Aldurazyme revenues are driven by the timing of when the product is released and control is transferred to Genzyme. Revenues for the fourth quarter of 2020 were comparatively lower than 2019 due to such timing. Based on data provided to us by Genzyme, patients receiving commercial Aldurazyme increased by 10% during 2020; partially offset by
• Naglazyme and Vimizim Net Product Revenues increased by an aggregate of $35.1 million primarily due to timing of sales in the Middle East and Latin America;
• Palynziq Net Product Revenues increased by $17.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy; and
• Brineura Net Product Revenues increased by $9.8 million driven by growth in the number of patients in all regions.

The increase in GAAP Net Income for the fourth quarter of 2020, compared to the same period in 2019 was primarily due to the following:

• decreased research and development (R&D) expense of $16.1 million primarily due to lower clinical activity spend for valoctocogene roxaparvovec gene therapy programs and decreased tralesinidase alfa costs as the program was licensed to a third-party in 2019; and
• an increase in the benefit from income taxes of $37.5 million primarily due to the change in the jurisdictional mix of earnings and the related tax impact from the completion of an intra-entity transfer of certain intellectual property rights to an Irish subsidiary where the Company's ex-US regional headquarters are located during the third quarter of 2020; partially offset by
• an increase in Cost of Sales of $30.2 million primarily due to inventory reserves and higher sales volumes of products with lower margins.

Non-GAAP Income for the fourth quarter of 2020 decreased to $39.5 million, compared to Non-GAAP Income of $46.4 million for the same period in 2019. The decrease in Non-GAAP Income for the quarter, compared to the same period in 2019, was primarily attributed to lower gross profits and higher SG&A expenses, partially offset by lower R&D expenses.

As of December 31, 2020, BioMarin had cash, cash equivalents and investments totaling $1.35 billion, which includes net proceeds of $535.8 million from the Company's May 2020 convertible debt offering, as compared to $1.17 billion as of December 31, 2019. On October 15, 2020, the Company's 1.50% senior subordinate convertible notes matured and were settled in cash for approximately $375.0 million.

Commenting on full-year 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "Despite the impact in 2020 from the COVID-19 pandemic and a delay in the potential approval of valoctocogene roxaparvovec for severe hemophilia A, demand for our current product portfolio continued to drive steady revenue growth and expansion of our pipeline.  Excluding contributions from Kuvan, for which a generic became available during 2020, total revenues grew 13% in 2020, and generated $85 million of positive operating cash flows for the full year, underscoring the essential nature of our medicines." 

Mr. Bienaimé continued, "The most recent Phase 3 data updates from our latest-stage development programs in achondroplasia and severe hemophilia A demonstrated significant efficacy.  In the largest gene therapy trial ever conducted for the treatment of severe hemophilia A, we were pleased that valoctocogene roxaparvovec was the first in hemophilia A to demonstrate statistically significant evidence of annualized bleed rate superiority over standard of care recombinant FVIII. Based on these results, we are very encouraged that one infusion of valoctocogene roxaparvovec gene therapy may potentially address the high treatment burden for people with severe hemophilia A.  We are targeting submission of the one-year Phase 3 results to the European Medicines Agency in the second quarter of 2021 and planning to dialog with the FDA to align on steps to obtain approval in the United States."

"Also in 2021, we look forward to the potential approval of vosoritide, which would be the first pharmacological treatment to address the underlying cause of achondroplasia, the most common form of dwarfism.  We announced in the fourth quarter of 2020 that vosoritide demonstrated sustained growth effects for over two years in children with achondroplasia participating in our Phase 3 extension study.  In addition to the large, Phase 3 program currently in the extension phase, we have built a multi-pronged dossier of additional studies to support our understanding of the unmet medical need for children with achondroplasia and the effects of vosoritide in this condition. In addition to the highly statistically significant placebo-controlled Phase 3 results, the program includes the long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, and the ongoing study of newborns through 5 years. Many families are keen to seek early treatment for their children so we are hopeful that, if approved, vosoritide will become available later in 2021 upon potential approvals."

2021 Full-Year Financial Guidance (in millions, except %)

Key Program Highlights

• Valoctocogene roxaparvovec gene therapy for severe hemophilia A: On January 10, 2021, the Company announced positive top-line, one-year data results from its ongoing global Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A. Data from the study in the pre-specified primary analysis for Annualized Bleeding Rate (ABR) showed that a single dose of valoctocogene roxaparvovec significantly reduced ABR by 84% compared with prior treatment with prophylactic FVIII infusions. These results were from a pre-specified group of participants in a non-interventional prospective baseline observational study (rollover population; N=112) with a median follow-up of 60.1 weeks after dosing with valoctocogene roxaparvovec. 80% of the rollover participants were bleed-free starting at week five after treatment.

Additionally, at the end of the first year post-infusion with valoctocogene roxaparvovec, participants in the modified intent-to-treat (mITT) population (N=132) had a mean endogenous Factor VIII expression level of 42.9 (SD 45.5, median 23.9) IU/dL, as measured by the chromogenic substrate (CS) assay, supporting the marked clinical benefits observed with abrogation of bleeding episodes and Factor VIII infusion treatment rate. Factor VIII expression declined at a slower rate compared to the Phase 1/2 study, and remained in a range to provide hemostatic efficacy. In a subset of the mITT population that had been dosed at least two years prior to the data cut date (N=17), Factor VIII expression declined from a mean of 42.2 (SD 50.9, median 23.9) IU/dL at the end of year one to a mean of 24.4 (SD 29.2, median 14.7) IU/dL at the end of year two with continued hemostatic efficacy demonstrated by a mean ABR of 0.9 (median 0.0) bleeding episodes per year.

Valoctocogene roxaparvovec also significantly reduced the mean annualized Factor VIII usage in the rollover population by 99% from 135.9 (median 128.6) to 2.0 (median 0.0) infusions per year (p-value