BioMarin Pharmaceutical (BMRN) Jefferies London Healthcare Conference 2024 summary

Strategic focus and leadership

• Refreshed strategy centers on innovation, growth, and value, with a focus on genetically defined diseases.

• Emphasis on skeletal conditions, notably VOXZOGO, with five new indications in development.

• Business restructured into focused units and key leadership roles filled to drive execution.

• $500 million cost transformation identified to support margin expansion.

• Targeting 40%+ operating margin by 2026 and $4 billion revenue by 2027, with mid-teen CAGR through 2034.

Enzyme therapy and geographic growth

• Enzyme therapy franchise generates ~$2 billion revenue, growing at 8% CAGR over three years.

• Growth expected to accelerate, leveraging presence in 80 countries and expansion in less developed markets.

• PALYNZIQ highlighted as a key growth driver, with label expansion in progress.

• Business aims to improve patient identification and adherence globally.

VOXZOGO performance and market strategy

• U.S. market for VOXZOGO expanded after FDA approval for use from birth, driving growth.

• Business unit approach increases focus and accountability for skeletal conditions.

• Geographic diversity remains critical, with reimbursement strategies in 80 countries.

• Goal to shift from prevalent to incident market before competition enters, leveraging early treatment indications.

• 95% real-world compliance and strong payer retention expected to limit patient switching.