BioMarin Pharmaceutical(BMRN) - 2022 Q4 - Earnings Call Transcript

Financial Data and Key Metrics Changes - BioMarin achieved total revenues of $2.1 billion for the full year 2022, marking a record financial achievement for the company [33] - The company reported a revenue growth of close to 20% in Q4 2022, with total revenues of $538 million [34] - GAAP net income and non-GAAP income growth were achieved despite recognizing approximately $23 million in charges to SG&A expenses in 2022, primarily in Q4 [27] Business Line Data and Key Metrics Changes - VOXZOGO generated full-year global revenue of $169 million in 2022, with guidance for 2023 set between $330 million and $380 million, indicating over 100% growth from the midpoint compared to 2022 [12] - PALYNZIQ therapy in the U.S. and Europe saw net product revenue growth of 7% in 2022, totaling $255 million [15] - ROCTAVIAN is projected to generate revenues between $100 million and $200 million in 2023, contingent on U.S. approval [38] Market Data and Key Metrics Changes - As of the end of Q4 2022, only 6% of the total addressable patient population had received VOXZOGO treatment, indicating significant growth potential [12] - In Germany, a major payer agreement has been established for ROCTAVIAN, although the commercial ramp has been hindered by lengthy negotiations for additional outcomes-based agreements [39] Company Strategy and Development Direction - The company aims for over 15% top-line growth in 2023, with a focus on sustainable profitability and leveraging its enzyme products alongside new market opportunities from VOXZOGO and ROCTAVIAN [33][43] - BioMarin is actively pursuing regulatory approvals to expand VOXZOGO's label to younger patients, which could significantly increase the addressable market [13][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the interest from prescribers and patients for ROCTAVIAN, despite the lengthy reimbursement process in Germany [32] - The company anticipates increased demand for all brands in 2023, including enzyme products, VOXZOGO, and ROCTAVIAN, with a commitment to growth and sustainable profitability [43] Other Important Information - The company is transitioning to a new methodology for calculating non-GAAP income to better align with its profitable business model and peer group [75] - BioMarin is investing in its largest early-stage research pipeline to fuel growth throughout the decade [74] Q&A Session Summary Question: What is the expected timeline for revenue flow from ROCTAVIAN in Germany? - Management confirmed that the 10 patients undergoing companion diagnostic testing are in Germany and emphasized the importance of these tests as a step towards treatment [80][81] Question: What is the status of the three-year data submission for ROCTAVIAN? - The company indicated that the assessment of whether the submission constitutes a major amendment is subjective and cannot provide specific guidance at this time [85] Question: How many U.S. treatment centers are expected to be ready for ROCTAVIAN at launch? - The company plans to target a small number of the largest and most capable hemophilia treatment centers in the U.S. for the launch of ROCTAVIAN [96] Question: What are the implications of the outcomes-based agreements in Germany? - Management explained that while these agreements will be superseded by federal reimbursement, they still provide value in facilitating patient access to therapy [89][116]