BioMarin Pharmaceutical(US:BMRN)2024-02-23 02:08Financial Data and Key Metrics Changes - Total revenue grew 20% in Q4 2023 compared to Q4 2022, and 15% for the full year 2023 compared to the prior year, with constant currency growth of 25% in Q4 and 20% for the full year [11][12] - Non-GAAP earnings per share increased 48% in Q4 versus Q4 2022, and 36% year-over-year for the full year [12][48] - GAAP net income for Q4 was $20.4 million, contributing to $168 million for the full year, with GAAP diluted earnings per share of $0.87 [47][48] Business Line Data and Key Metrics Changes - VOXZOGO revenues reached $146 million in Q4, representing 118% quarter-over-quarter growth, with a year-over-year growth of 178% [13][44] - Enzyme products generated over $1.7 billion in net product revenues for the full year 2023, an increase of 4% year-over-year [43] - Double-digit revenue growth was observed in both VIMIZIM and PALYNZIQ in Q4, partially offset by lower KUVAN revenues [44] Market Data and Key Metrics Changes - 70% of new US prescriptions for VOXZOGO in Q4 were for children under five, following FDA's age expansion approval [14] - The company is actively engaging global health authorities regarding development programs in idiopathic short stature and multiple genetic short stature pathway conditions [19] Company Strategy and Development Direction - The company aims to maximize the VOXZOGO opportunity and expand into multiple other statural conditions [20] - A strategic portfolio review is underway to prioritize R&D assets with the highest potential patient impact and value creation for shareholders [25][30] - The company plans to increase profitability faster than originally planned, with guidance reflecting double-digit revenue growth for 2024 [26][55] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong demand for VOXZOGO and the potential for ROCTAVIAN, with expectations for patient treatment in 2024 [21][24] - The company is focused on disciplined spending and prioritization of impactful R&D assets, with updates to be communicated at an Investor Day later in 2024 [25][37] - Management noted the importance of aligning patient motivation, payer support, and treatment site readiness for successful ROCTAVIAN treatment [23] Other Important Information - The company is transitioning to a cash flow positive status and is committed to developing a capital allocation strategy that maximizes shareholder value [123] - Non-GAAP operating margin for 2024 is guided to be between 23% and 24%, representing a 4 percentage point expansion from 2023 [55] Q&A Session Summary Question: EPS guidance for 2024 and assumptions - Management indicated that the non-GAAP EPS guidance midpoint implies 30% growth over 2023, with some tax benefits and unique SG&A charges impacting the analysis [60][62] Question: R&D portfolio prioritization criteria - The strategic assessment will rank and prioritize assets based on time, cost, market opportunity, and competitive landscape [65][66] Question: Thoughts on divesting gene therapy platform - Management is still assessing ROCTAVIAN's opportunity and will evaluate gene therapy exposure in the pipeline [71][72] Question: VOXZOGO revenue dynamics and channel inventory - Management clarified that Q4 revenue included additional commercial supply, but Q1 growth may not replicate the same dynamic [74] Question: Update on VOXZOGO's approval status - No specific updates on regulatory status, but management emphasized the sustained effect of VOXZOGO as a key feature [84] Question: Commercial investments impact on VOXZOGO - Management noted good returns on commercial investments, with a focus on expanding the prescriber base [90] Question: Market opportunity for VOXZOGO - Management indicated that approximately 20,000 patients are eligible for VOXZOGO across 41 active markets [118] Question: Future capital allocation strategy - Management is committed to internal innovation while also reviewing the pipeline for potential cuts and reinvestments [123]