BioMarin JPM 2025

Company and Industry Overview * Company: BioMarin Pharmaceutical Inc. * Industry: Biotechnology, specializing in treatments for genetically defined conditions. Key Financial Highlights * Total Revenues: $2.1 billion (3Q'24 YTD) * Non-GAAP Operating Margin: 27.7% * Non-GAAP Diluted Earnings Per Share: $2.60 * Revenue Growth: +19% YOY * Operating Margin Growth: +7.6 ppts YOY Pipeline and Pipeline Highlights * VOXZOGO: * Phase 3 data for 5 indications (achondroplasia, hypochondroplasia, idiopathic short stature, Noonan Syndrome, Turner Syndrome, SHOX deficiency) * Expansion into additional countries, including Europe, Japan, Australia, and Canada * Strong growth potential in the U.S. market * Palynziq: * Phase 3 data and U.S. & EU sBLA filings * Expansion of label to include 12-17 year-olds * Potential for $1.25B+ in 2027 operating cash flow * BMN 390: * First-in-human study start for PKU with novel PEG * BMN 351: * Clinical POC data for Duchenne Muscular Dystrophy * BMN 349: * Study start for alpha-1 antitrypsin deficiency * BMN 370: * First-in-human study start for von Willebrand disease Business Development and External Innovation * Focus on disciplined deal execution with a focus on Skeletal Conditions, Enzyme Therapies, and First in Genetic Conditions * Strong global commercial infrastructure and world-class research, clinical, and regulatory capabilities * Rich biotech arena with many small companies at R&D stage Financial Outlook * Revenue growth targeting mid-teen CAGR from 2023-2034 * Non-GAAP operating margin targeting 40% in 2027 * Non-GAAP operating cash flow targeting $1.25B+ in 2027 Other Important Points * BioMarin is the global leader in treating genetically defined conditions * Strong execution in 2024, with significant growth in revenues and operating margin * Focus on innovation and growth through a robust pipeline and strategic business development initiatives