On August 4, 2020 BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) reported financial results for the second quarter ended June 30, 2020 (Press release, BioMarin, AUG 4, 2020, View Source [SID1234562801]).
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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.