BioMarin Pharmaceutical Third Quarter 2026 Conference Call - AMICUS THERAPEUTICS INC

Explicit Guidance:

• FY2026 Total Revenue: Updated range revised to $3.8 billion – $3.9 billion (down from prior guidance due to $70M order timing deferrals from a single international customer, shifting expected shipments from Q4 2026 to late Q4/Q1 2027).
• FY2026 Non-GAAP Diluted EPS: Updated range revised to $2.80 – $3.10 (impacted by $1.55 dilution from Alesta IPR&D/transition costs and $0.32 dilution from BMN450 license IPR&D; midpoint effectively unchanged from Q2 guidance absent these transactions).
• Q4 2026 Impact: Expect normalization of Q3's 24-cent tax benefit (net zero for full year) and potential revenue recognition for the $70M deferred orders.
• FY2028 Cost Synergies: Expect full realization of approximately $220 million in non-GAAP cost synergies from the Amicus acquisition.
• 2027 Outlook: Expect VoxOgo revenue growth in 2027, driven by hypochondroplasia launch and international expansion, though specific 2027 guidance will be provided later.

Business-Segment Outlook:

• Metabolic Conditions:
  • Pompily and Opfolda: Revenue build from new patient starts (driven by competitor supply constraints and switching) expected to build into 2027 and beyond; currently market share leader in four European markets.
  • GallaFold: Patient count grew 13% YoY; continuing global expansion and new market penetration.
  • Palynziq: Adolescent revenue contribution expected to build into 2027 and beyond as patients titrate to maintenance doses.
  • Amicus Integration: Cost synergies realized ahead of schedule; deal expected to be accretive within nine months (calendar year 2026), with substantial accretion beginning in 2027.
• Skeletal Conditions:
  • VoxOgo:
    • Hypochondroplasia: PDUFA date set for May 30, 2027; anticipated launch in US and Europe as a potential seventh first-in-disease medicine. Global TAP estimated at 14,000 children.
    • Achondroplasia: 75% of revenue generated outside US; continued global expansion into three new international markets approved; patients under two remain majority of new starts in US.
    • VoxOgo 2027: Growth anticipated driven by hypochondroplasia launch and offsetting of competitive share losses.
  • BMN333: Phase 2 data expected in 2027 focusing on Pharmacodynamics (AGV) and Exposure-Response; aims to establish superiority over VoxOgo.
• Pipeline Expansion:
  • BMN450 (Fabry): Phase 1-2 data expected to advance program; potential to become first subcutaneous ERT; addresses mutation-agnostic population; ongoing study assumes Biomarin sole responsibility for commercialization outside Korea.
  • BMN440 (HPP): Phase 1-2a data expected in 2027 for PK, PD, and safety to establish proof of mechanism; potential first oral treatment for adult-onset HPP.

Market and Competitive Outlook:

• Supply Constraints: Competitor Pompe therapy supply constraints in Europe expected to continue for an indeterminate period; Biomarin has increased supply to capture switching patients, though the duration and full revenue impact remain uncertain.
• Competitive Landscape (FGFR2): Management views safety concerns regarding FGFR2 inhibition in the developing brain as "hypothetical"; confidence in VoxOgo's safety profile backed by 5,000+ patients and 10,000+ patient-years of real-world data.
• Competitive Landscape (HPP): No approved therapies for adult-onset HPP in US or Europe; BMN440 positioned to address systemic symptoms (muscle weakness, pain, fatigue) beyond bone mineralization.
• Competitive Landscape (Achondroplasia): Growth hormone combination data suggests potential waning effects and risk of premature growth plate closure; VoxOgo and BMN333 positioned as preferred single-agent solutions.

Macro / Industry Outlook:

• Geopolitics: Prolonged conflict in the Middle East causing increased customer liquidity/cash flow disruption; impact expected to persist through Q4 2026, contributing to the revenue guidance reduction.
• International Markets: Two-thirds of business is international (single-payer national health care systems); large order timing fluctuations are normal but magnitude near year-end creates guidance uncertainty.
• Regulatory: FDA guidance on small molecule CNS crossing is structured; VoxOgo full approval data package for hypochondroplasia submitted for review with Feb 28 PDUFA date.

Strategic Initiatives:

• M&A / Business Development:
  • Alesta Acquisition (Closed Aug 31, 2026): Delivered BMN440 (HPP) and BMN430; reduced FY2026 non-GAAP EPS by ~$1.55 due to $320M IPR&D and operating costs.
  • BMN450 License: Added Fabry asset; reduced FY2026 non-GAAP EPS by ~$32 cents due to $83M upfront IPR&D expense (Q4 recognition).
  • Future Strategy: Continue acquiring clinical-stage assets in genetic conditions, skeletal, and metabolic disease; $4-$5 billion liquidity expected mid-2027 to fuel future transactions.
• Commercial Execution:
  • Hypochondroplasia Launch Prep (2027): Focus on evidence sharing (Phase 3 data), diagnosis expansion (consensus recommendations published, 1,200+ educators reached), and payer engagement.
  • VoxOgo Switching: Transitioning from reporting US switching metrics to revenue updates in Q4 to reflect changing market mix with new indications.

Risks and Headwinds to Outlook:

• Order Timing: $70M in international orders deferred from Q4 2026 to Q1 2027 or later due to customer liquidity issues; exact timing uncertain.
• Regulatory Uncertainty: PDUFA outcome and FDA views on FGFR2 safety debates for VoxOgo/hypochondroplasia.
• Clinical Data Risks:
  • BMN333: Potential failure to demonstrate superiority or plateau in growth velocity relative to exposure; risk of needing to pivot to non-inferiority or less frequent dosing hypotheses.
  • BMN440: Proof of mechanism data in 2027 may not support further development.
• Competitive Supply: Unknown duration of competitor Pompe supply shortages could limit the magnitude of permanent switch gains.
• Integration Risks: Alesta transaction costs and IPR&D write-offs impacting 2026 earnings.

Management Tone on the Future:

• Confidence: "Energized," "confident," and "enthusiastic" regarding pipeline expansion and commercial diversification.
• Hedges: Management emphasizes that revenue guidance changes are driven by "order timing" not "underlying patient demand"; frequently notes that international single-payer ordering is volatile.
• Strategic Shift: Shift from "quarterly guidance" to focusing on "full-year metabolic conditions guidance" as a better indicator of underlying performance due to order timing volatility.

Miscellaneous:

• Investor Relations Note: Commentary focuses on non-GAAP financial measures; Q3 included $290M pre-tax expense from Alesta ($1.48/share) and $0.23 non-GAAP EPS.
• Data Readouts: 5-year Propel extension data for Pompe disease presented at World Muscle Society (Oct 2026) shows sustained motor/pulmonary function.