BioMarin Pharmaceutical (BMRN) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Strategic vision and recent developments

• Expanded portfolio to nine commercial products across skeletal and metabolic conditions, with recent acquisition of Amicus adding Galafold and PomUp, projected to reach $1.4B and $1.2B peak revenues in the mid-2030s.

• Achieved 20% year-over-year growth, with four products expected to surpass $1B in annual sales, including VOXZOGO, which is guided to $1B-$1.05B in 2026.

• Focused on driving top-line growth, margin expansion, and cash flow to reinvest in pipeline innovation.

• Announced $220M in synergies from the Amicus deal by 2028 and substantial EPS accretion in 2027.

• Pipeline strengthened with new assets, including ALE-001 for hypophosphatasia and BMN 333 as a next-generation CNP.

Product and pipeline updates

• VOXZOGO remains a key growth driver, with strong retention (90%) despite new competition and expanded international presence (75% of revenues ex-U.S.).

• Settlement with Ascendis Pharma secures royalties on YUVIWEL sales and reduces competitive overhang.

• Hypochondroplasia data exceeded expectations, showing significant improvements in annualized growth velocity and arm span, with FDA submission completed.

• Commercial launch preparations for hypochondroplasia are underway, targeting a global addressable population of 14,000.

• BMN 333 aims for a 50% improvement in growth over VOXZOGO, with phase II/III studies ongoing and data expected in 2027.

Commercial and financial outlook

• Raised long-term peak sales guidance for Galafold ($1.4B) and PomUp ($1.2B), driven by geographic expansion and enhanced diagnosis initiatives.

• Revenue growth is the primary driver of accretion from the Amicus deal, complemented by $220M in cost synergies, mainly from G&A.

• Real-world evidence and patient engagement strategies are central to driving adoption and switching for new therapies.

• Pipeline evolution will balance organic and inorganic growth, leveraging a strong balance sheet for future acquisitions.

• Continued focus on expanding both skeletal and metabolic franchises, with ongoing efforts to improve diagnosis and access globally.