Agios

Overview

Agios is a commercial-stage biopharmaceutical company developing medicines for rare hematologic and metabolic diseases. The company combines pyruvate kinase (PK) activation and metabolic biology with classical hematology to address conditions including PK deficiency, thalassemia, sickle cell disease (SCD), low‑risk myelodysplastic syndromes (LR MDS), phenylketonuria (PKU), and polycythemia vera (PV).

Commercial products

• Mitapivat (PK activator)
  • PYRUKYND® (mitapivat) — marketed in the United States for hemolytic anemia due to PK deficiency; approved in the European Union and Saudi Arabia for PK deficiency; approved in the United States and EU for other PK deficiency‑related indications.
  • AQVESME™ (mitapivat) — FDA‑approved December 2025 in the United States for anemia in adults with non‑transfusion dependent and transfusion‑dependent alpha‑ or beta‑thalassemia; US launch in January 2026 with a REMS in place.

Development pipeline (selected programs)

• Tebapivat — novel PK activator for LR MDS and SCD (phase 2b data anticipated in 2026; phase 2 for SCD; phase 2a for LR MDS completed).
• AG-181 — PAH stabilizer for PKU (IND 2023; phase 1 completed; phase 1b proof‑of‑mechanism planned for 2026).
• AG-236 — tmPRSS6‑targeting siRNA for PV (licensed from Alnylam; IND cleared June 2025; phase 1 healthy‑volunteer trial started July 2025; topline data expected in 2026).

Partnerships and licensing

• Alnylam (AG-236 TMPRSS6 siRNA): up‑front $17.5 million (2023); potential development and regulatory milestones up to $130 million plus sales milestones and royalties; $10.0 million regulatory milestone paid in 2025.
• Vorasidenib transactions:
  • Sale of vorasidenib oncology assets to Servier (closed March 31, 2021): approximately $1.8 billion upfront and potential $200 million milestone; 15% royalty on U.S. net sales up to $1.0 billion per year; retained 3% earn‑out on excess payments; upfront sale income recognized in 2024 of $889.1 million net of fees.
  • Sale of vorasidenib royalty rights to Royalty Pharma (May 2024; closing August 2024): $905.0 million upfront; income recognized in 2024 of $889.1 million net of fees; a $200.0 million vorasidenib milestone payment was recognized in 2024.

Global commercialization

• Built U.S. commercial infrastructure for PYRUKYND® and expanded for AQVESME™ in thalassemia.
• NewBridge Agreement (July 2024): PYRUKYND® rights in the GCC region via third‑party commercialization.
• Avanzanite Agreement (June 2025): PYRUKYND® rights in EU, EEA, Switzerland, and the UK via third‑party commercialization.
• Global managed access programs and patient access (for‑charge or free‑of‑charge) in applicable jurisdictions.

Financial highlights (selected)

• Net product revenues — PYRUKYND®:
  • 2025: $54.0 million
  • 2024: $36.5 million
  • 2023: $26.8 million
• Vorasidenib‑related:
  • Recognized $200.0 million vorasidenib milestone payment in 2024 within gain on sale of contingent payments.
  • Vorasidenib royalty right sale: $905.0 million upfront; $889.1 million recognized in 2024 net of fees.
• Net income (loss):
  • 2025: net loss $412.8 million
  • 2024: net income $673.7 million (driven by vorasidenib‑related transactions)
  • 2023: net loss $352.1 million
• Accumulated deficit as of December 31, 2025: $561.7 million
• 2025 notable non‑operating item: $10.0 million regulatory milestone payment to Alnylam recorded as research and development expense (investing cash flow classification).

Operating structure and workforce

• One operating segment.
• Employees (as of December 31, 2025): 539 full‑time and 1 part‑time.
• Diversity and hiring (as of December 31, 2025):
  • 61% female workforce
  • 35% ethnically diverse workforce
  • 34% of new hires in 2025 were ethnically diverse
• Headquarters: 88 Sidney Street, Cambridge, Massachusetts 02139
• Website: www.agios.com

Market and regulatory context

• Mitapivat approvals and regulatory status:
  • PYRUKYND® approved by the FDA in the U.S. for PK deficiency; approvals in EU and Saudi Arabia for PK deficiency and other PK‑related indications in applicable jurisdictions.
  • AQVESME™ FDA approval December 2025 for anemia in adults with non‑transfusion dependent and transfusion‑dependent alpha‑ or beta‑thalassemia; U.S. launch in late January 2026; REMS in place for AQVESME™ due to hepatocellular injury risk.
  • CHMP positive opinion (October 2025) for PYRUKYND® in adults with anemia associated with transfusion‑dependent and non‑transfusion‑dependent alpha‑ or beta‑thalassemia; final European Commission decision expected early 2026.
• Regulatory pathway for SCD: pre‑sNDA meeting with the FDA planned for Q1 2026 for mitapivat, with a potential U.S. regulatory filing to follow that engagement.

Intellectual property and development context

• Core platform centers on PK activators (wild‑type and mutant PK enzymes) to improve red blood cell health and ATP generation in hemolytic anemias.
• Additional programs include tebapivat, AG‑181, and AG‑236 (TMPRSS6 program licensed from Alnylam).
• A broad patent portfolio covers mitapivat, tebapivat, AG‑181 PAH stabilizers, and AG‑236; European patent decisions include oppositions and appeals.

Commercial and strategic priorities

• Commercialize PYRUKYND® and AQVESME™ in the United States and selected international markets while pursuing additional indications (SCD, thalassemia, pediatric PK deficiency considerations, LR MDS, PKU, PV).
• Expand geographic reach and pipeline potential through partnerships (NewBridge, Avanzanite) and existing license arrangements (Alnylam).
• Maintain emphasis on product safety, REMS programs where required, and the clinical development of experimental therapies with a view toward long‑term profitability and value creation.