- Impact of the Coronavirus (COVID-19) Pandemic as Expected in the Second Quarter; 2020 Full-Year Revenue Guidance Reaffirmed Assuming Demand Patterns Return to Normal in Late 2020

- U.S. Marketing Application for Valoctocogene Roxaparvovec for Severe Hemophilia A under Priority Review by Food and Drug Administration (FDA) with Prescription Drug User Fee Act (PDUFA) Target Action Date of August 21, 2020

- Marketing Authorization Application (MAA) for Valoctocogene Roxaparvovec for Severe Hemophilia A Validated by the European Medicines Agency (EMA); Committee for Medicinal Products for Human Use (CHMP) Opinion Expected by Late 2020/Early 2021

- MAA Submitted to EMA for Vosoritide to Treat Children with Achondroplasia July 23; On track to Submit a New Drug Application (NDA) to the FDA Late in the Third Quarter of 2020

- For the Full-Year 2020 BioMarin Continues to Expect to be Profitable on a GAAP Basis for the First Time

SAN RAFAEL, Calif., Aug. 4, 2020 /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 second quarter ended June 30, 2020.

Net Product Revenues for the second quarter of 2020 increased to $419.0 million, compared to $379.1 million in the second quarter of 2019. The increase in Net Product Revenues was attributed to the following:

• Aldurazyme Net Product Revenues increased by $26.5 million due to higher sales volume to Genzyme;
• Palynziq Net Product Revenues increased by $21.9 million driven by a combination of revenue from U.S. patients achieving maintenance dosing and new patients initiating therapy;
• Brineura Net Product Revenues increased by $11.0 million due in large part growth in the number of patients in all regions; and
• Kuvan Net Product Revenues increased by $9.3 million driven primarily by a U.S. price increase and Kuvan product mix; partially offset by
• Naglazyme and Vimizim Net Product Revenues combined decreased by $23.2 million primarily due to timing of orders as well as the impact of missed infusions resulting from the COVID-19 pandemic.

The decrease in GAAP Net Loss for the second quarter of 2020, compared to GAAP Net Loss for the same period in 2019 was primarily due to the following:

• an increase in gross profit (Total Revenues less Cost of Sales) of $21.2 million primarily driven by higher product sales; and
• an increase in the benefit from income taxes; partially offset by
• the effect of the one-time gain recognized in the second quarter of 2019 due to a third party's achievement of a commercial milestone related to previously sold intangible asset; and
• higher selling, general and administrative (SG&A) expense related to pre-commercialization activities for valoctocogene roxaparvovec.

Non-GAAP Income for the second quarter of 2020 increased to $57.4 million, compared to Non-GAAP Income of $17.1 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to decreased R&D expense and higher gross profit, partially offset by higher SG&A expense.

As of June 30, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.7 billion, as compared to $1.2 billion on December 31, 2019.

Commenting on second quarter 2020 results, Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin, said, "In the second quarter, BioMarin employees worked collaboratively to ensure access to our critically-important medicines to the people we serve, despite the global impact of COVID-19. In these challenging times, our strong financial results underscore both the essential nature of our products to patients and our ongoing efforts to maintain supply around the world."

Mr. Bienaimé continued, "In the second quarter at the World Federation of Hemophilia Virtual Congress, we were pleased to share the four-year data update from our ongoing Phase 1/2 study, which demonstrated sustained clinical benefit following a single administration of valoctocogene roxaparvovec. All participants in the study received a single administration of valoctocogene roxaparvovec in 2016 and remained off exogenous factor prophylaxis through year four. These data strengthen our confidence in valoctocogene roxaparvovec and the opportunity to address the unmet therapeutic needs of people with severe hemophilia A. With our marketing applications under review in both the United States and Europe, we await the potential approval of valoctocogene roxaparvovec. We believe each of the submissions represent the first time a gene therapy product for any type of hemophilia indication is under review by health authorities. With the outcome of the Priority Review of our BLA anticipated August 21, 2020, our commercial team is preparing to launch what we believe is the most innovative product yet for people with bleeding disorders."

"Another key milestone in the third quarter of this year, representing the culmination of years of clinical study and development, was the July 23 submission of a MAA to the EMA for vosoritide to treat children with achondroplasia. The company remains on track to submit a NDA to the FDA later in the third quarter. Our multi-pronged dossier of data encompasses long-term clinical results in 5 to 18 year-olds from our Phase 2 study, natural history data, the ongoing study of newborns through 5 years, and highly statistically significant placebo-controlled Phase 3 results. The positive results from our vosoritide clinical programs bolster our confidence in the potential for this drug to be the first pharmacological treatment for the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that families are keen to seek early treatment for their children."

Mr. Bienaimé concluded, "Despite impact from COVID-19 on our business in the short-term, we remain focused on working towards significant achievements that we believe will drive long-term value. Key milestones for the second half of 2020 include reaching GAAP profitability for a full year for the first time in our history, the potential approval of valoctocogene roxaparvovec, and the pursuit of vosoritide approval. With these exciting possibilities on the horizon, 2020 has the potential to be the most momentous year in our 20-year history."

2020 Full-Year Financial Guidance

GAAP Net Income guidance for 2020 has been updated to include the potential impact of intangible asset transfers between BioMarin entities.  These intangible asset transfers are expected to occur in the second half of 2020, and are estimated to result in a one-time, non-cash income tax benefit of approximately $700 million to $900 million.  The range acknowledges that the intangible asset transfers have not yet been completed and therefore the final value cannot yet be determined with certainty. The final valuation will be completed when the transactions occur.  As a result, full year GAAP net income guidance has been updated to be in the range between $720 million and $980 million.  The intangible asset transfers are not expected to impact Non-GAAP income.  

Key Program Highlights

• Valoctocogene roxaparvovec gene therapy for severe hemophilia A: On June 17, 2020, the Company provided a four-year update with the 6e13 vg/kg dose subjects and a three-year update with the 4e13 vg/kg dose subjects from the ongoing Phase 2 study in a late-breaking oral presentation at World Federation of Hemophilia Virtual Summit. The results from both dose cohorts demonstrated that all subjects had remained off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec. In addition, cumulative mean annualized bleed rates (ABR) remained less than one in both cohorts and below pre-treatment baseline levels. In the six study participants who were previously on Factor VIII prophylaxis in the 6e13 vg/kg cohort, the data showed substantial and sustained reductions in bleeding that required Factor VIII infusions. During the four years following treatment with valoctocogene roxaparvovec, the cumulative mean ABR was 0.8, which represents a 95% reduction from baseline. In the fourth year, the mean ABR was 1.3 and the median was zero. There was a 96% reduction in mean Factor VIII usage to 5.4 infusions per year cumulatively over four years from the baseline of 135.6 infusions per year on Standard of Care prophylaxis.
• Similarly, in the six study participants in the 4e13 vg/kg cohort, the data showed substantial and sustained reductions in bleeding requiring Factor VIII infusions following treatment with valoctocogene roxaparvovec. The cumulative mean ABR was reduced by 93% to 0.9 with continued absence of target joint bleeds in 5 of 6 subjects during the three years observed, which represents a 93% reduction from baseline. During the third year of follow-up, the mean ABR was 0.5 and the median ABR was zero, and 67% or four out of six, of the study participants were bleed-free. Five out of six participants had no spontaneous bleeds. There was a 96% reduction in mean Factor VIII usage to 5.7 infusions per year cumulatively over three years from the baseline of 142.8 infusions per year. The FDA review of the BLA, under Priority Review, for valoctocogene roxaparvovec is on-track with a PDUFA target action date of August 21, 2020. In Europe, the MAA filing remains under accelerated assessment at this time. However, as communicated on the first quarter results call, the review procedure was extended by at least 3 months due to COVID-19 delays. Further, as is the case with most filings that initially receive accelerated assessment, there is a high possibility that the MAA will revert to a standard review procedure from accelerated assessment. Based on these assumptions, the CHMP opinion is expected in late 2020/early 2021.
• Vosoritide for children with achondroplasia: On July 23, 2020, the Company announced that it had submitted the MAA for vosoritide for the treatment of children with achondroplasia to the EMA. Vosoritide is an investigational, once daily injection of an analog of C-type Natriuretic Peptide (CNP). BioMarin plans to submit a vosoritide marketing application to the FDA later in the third quarter of 2020.

The applications include positive final results from its randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide. The placebo-adjusted increased change from baseline in growth velocity after one year of treatment with vosoritide, the primary endpoint, was 1.6 cm/yr (p