- **AYVAKIT Growth Accelerates**: 61% YoY revenue growth, guidance raised to $700-720M on <10% free goods mix.
- **Pipeline Momentum**: BLU-808 Phase 2a trials underway; elenestinib pivotal study enrolling for 2030 $2B revenue target.
- **Financial Strength**: $900M cash, disciplined R&D spend ($75M Q1) to fuel pipeline expansion.
- **Long-Term Vision**: AYVAKIT to $2B by 2030; SM franchise peak at $4B with new patient starts and international expansion.

**【CEO Summary Section】**
- AYVAKIT revenue grew 61% YoY in Q1 2025, driven by strong demand in systemic mastocytosis (SM) market.
- Raised 2025 revenue guidance to $700-720 million due to favorable free vs. paid goods mix (<10%) and new patient starts.
- Advanced pipeline programs: elenestinib (pivotal HARBOR study) and BLU-808 (wild-type KIT inhibitor) with Phase 2a trials in allergic rhinoconjunctivitis and chronic urticaria.
- Maintained strong cash position ($900 million) to support disciplined capital allocation.

**【CFO Financial Commentary】**
- Q1 2025 total revenue: $149.4 million (US: $129.4M, ex-US: $20M).
- Operating expenses: $134.2 million (R&D: $75.1M, SG&A: $59.1M).
- Free cash flow: $115.4 million (excluding stock-based compensation).
- 2025 R&D investment focused on elenestinib (SM) and BLU-808 (allergic diseases).

**【Commercial Performance Highlights】**
- US discontinuation rate <3%, multi-year therapy duration trends improving.
- Global patient starts growth: ISM (intermediate SM) and advanced SM both up YoY.
- Ex-US market: Germany ISM reimbursement driving 10-15% of total revenue growth.

**【Pipeline Updates】**
- BLU-808 Phase 2a trials initiated in allergic rhinoconjunctivitis and chronic urticaria.
- Elenestinib (KIT D816V inhibitor) pivotal HARBOR study enrolling patients for disease-modifying outcomes.
- AYVAKIT long-term safety data: 3-year treatment with <3% discontinuation rate at 25mg dose.

Blueprint Medicines Corporation (BPMC) Q1 2025 Earnings Call Transcript

- **Record Growth**: AYVAKIT 2024 revenue ($479M) up 135%, 2025 guidance ($680-710M) reflects 45% growth, targeting $2B by 2030.
- **Expanded Market Potential**: SM peak value raised to $4B with 60,000+ U.S. patients and <10% diagnosed penetration.
- **Pipeline Momentum**: BLU-808 POC studies launching Q2 2025; elenestinib Phase III evaluates disease modification.
- **Financial Strength**: 2024 cash burn halved to $200M; $864M cash + $80M GSK deal support sustainable growth.

**【CEO Summary Section】**
- AYVAKIT 2024 revenue reached $479 million, up 135% year-over-year. 2025 guidance set at $680-$710 million (45% midpoint growth), targeting $2 billion by 2030.
- SM franchise peak value updated to $4 billion, driven by 60,000+ U.S. SM patients (double prior estimate) and <10% penetration of diagnosed patients.
- Elenestinib Phase III (HARBOR) underway, targeting disease modification beyond symptom control.
- BLU-808 proof-of-concept studies launching in 2025 for chronic urticaria, allergic rhinitis, and MCAS.

**【CFO Financial Commentary】**
- 2024 net product revenue: $479 million (135% YoY growth). Operating expenses reduced by >50% to $200 million, cash burn halved.
- 2025 operating cash burn expected to decline further. 2025 R&D/S&M investments to support AYVAKIT growth and pipeline expansion.
- Cash position: $864 million + $80 million from GSK deal, enabling sustainable growth.

**【Commercial Execution】**
- AYVAKIT prescribers grew 10x among allergists, with 95% patient satisfaction. 2025 field team expansion to engage 40% more providers (dermatologists, GIs).
- Q1 2025 challenges: insurance reverification delays, FX headwinds. 2025 ex-U.S. revenue expected to remain 10-15% of total.

**【Pipeline Updates】**
- BLU-808 healthy volunteer data: wide therapeutic index, rapid PK response. First POC data expected late 2025.
- Elenestinib Phase III focuses on bone health and anaphylaxis reduction. AYVAKIT 3-year safety data confirm chronic tolerability.
- MCAS strategy: advanced diagnostic testing to identify KIT D816V mutation patients for AYVAKIT/BLU-808 dual targeting.

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