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BioMarin Pharmaceutical (BMRN) investor relations material
BioMarin Pharmaceutical Jefferies London Healthcare Conference 2025 summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Jefferies London Healthcare Conference 2025 summary18 Nov, 2025
Strategic transformation and operational execution
Strategy reorganized around growth, innovation, and value commitment, with $500 million in cost transformation identified and largely executed.
Improved cash flow and strengthened balance sheet, with reinvestment into growth drivers and pipeline assets.
Targeting a 40% non-GAAP operating margin in 2025, more than doubling from 2023, with current margins at 33-34% excluding in-process R&D charges.
Focus on disciplined investments and operating efficiency, balancing profitability with reinvestment.
Shareholder value creation prioritized, with flexibility to adjust long-term margin targets if compelling opportunities arise.
Pipeline and growth opportunities
Multiple near-term pipeline readouts expected, including BMN 351 for Duchenne muscular dystrophy by year-end and phase III data for Voxogo in hypochondroplasia in early 2025.
Phase III readout for the 4.0.1 program from the INNERZYM acquisition and pending FDA approval for Palynziq in adolescents, with an action date of February 28 next year.
Voxogo expected to continue growing globally, with new indications and launches in 55 of 80 countries supporting expansion.
Hypochondroplasia addressable market estimated at 14,000 patients, with commercial launch readiness and awareness-building underway.
Study for hypochondroplasia powered for a 1.5 cm/year AGV delta, with conservative assumptions and potential for a bolus launch upon approval.
Business development and competitive positioning
Business development focused on genetically defined conditions, a segment with limited competition from large pharma due to specialized expertise required.
Global footprint in 80 countries and in-house manufacturing provide a competitive advantage for acquiring and commercializing rare disease assets.
Assets in rare diseases seen as more valuable within the company due to established infrastructure and global reach.
Acquisitions are evaluated for earnings accretion and alignment with long-term financial guidance.
Ongoing litigation and regulatory strategies, including orphan drug exclusivity and IP actions, supplement market competition efforts.
How does global reach enhance BD asset value?
What IP/regulatory defenses protect Voxzogo?
Expect a bolus launch for hypochondroplasia?
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Next BioMarin Pharmaceutical earnings date
Q4 202518 Feb, 2026
Next BioMarin Pharmaceutical earnings date
Q4 202518 Feb, 2026
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Frequently asked questions
BioMarin Pharmaceutical Inc is a biotechnology company focused on developing and commercializing therapies for rare genetic diseases. Its research and product development efforts target conditions with limited treatment options, primarily in the fields of enzyme replacement and gene therapy. The company serves healthcare providers and patients worldwide through approved treatments and scientific initiatives. The company is headquartered in San Rafael, California, and its shares are listed on the NASDAQ.
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