Agios Pharmaceuticals (AGIO) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic outlook and growth priorities

• Positioned at a major growth inflection point, aiming for profitability by leveraging recent thalassemia approval and expanding the PK activator franchise, with a clear path to profitability and flat operating expenses in 2026 versus 2025.

• Four strategic priorities for 2026: launch Aqvesme for thalassemia, expand PK activation into new indications, unlock future value with pipeline assets, and maintain financial discipline.

• Focused on maximizing U.S. market opportunity, with ex-U.S. growth expected to follow as reimbursement and distribution mature.

• Ongoing evaluation of internal and external opportunities, maintaining a high bar for pipeline advancement and disciplined capital allocation.

• Approximately $1.3 billion in cash and equivalents as of September 2025 supports future investments.

Product pipeline and market potential

• PK activator franchise anchored by mitapivat (Pyrukynd, Aqvesme) with approvals in PK deficiency and thalassemia; pursuing sickle cell disease and low-risk MDS, with tebapivat advancing in sickle cell and MDS.

• Early-stage assets include AG-181 for phenylketonuria and AG-236 for polycythemia vera, both progressing to first-in-human or Phase 1 trials by 2026.

• Total addressable market for pipeline indications estimated at over $10 billion globally by 2030, with thalassemia and sickle cell as major drivers.

• U.S. prioritized for thalassemia launch, with Gulf and select European markets as secondary growth areas; commercial agreements executed for these regions.

• Dedicated field teams and patient support services in place to drive adoption.

Aqvesme thalassemia launch and commercial strategy

• Aqvesme approved in December 2023 for both alpha and beta thalassemia, regardless of transfusion status; first oral, disease-modifying therapy for this population.

• U.S. launch targets 4,000 addressable adult patients, with initial focus on transfusion-dependent segment and $1B global peak-year sales potential.

• Price set at $425,000 per patient per year, reflecting clinical value and market potential exceeding $1 billion globally.

• Early physician and patient feedback is highly positive, with strong demand and education efforts underway.

• REMS program requires monthly liver monitoring for six months, but aligns with standard clinical practice.