Strong Q4 and Full-Year Revenue Growth
Q4 worldwide Pyrukynd revenue of $20,000,000, up 86% year-over-year and up 55% sequentially from $13,000,000; full year 2025 revenue of $54,000,000.
Afesmi U.S. Approval and Launch
FDA approval for Afesmi in thalassemia (Dec 23, 2025) with REMS implemented late January 2026 and initial dispensing underway.
Early Afesmi Commercial Traction
44 prescriptions written by REMS-certified U.S. physicians in first five weeks (through Jan 30); early prescribers show broad geographic distribution and predominance of community physicians, with initial patient mix of transfusion-dependent and engaged non–transfusion-dependent patients.
Solid Balance Sheet and Financial Flexibility
Cash, cash equivalents and marketable securities of approximately $1,200,000,000 at year-end, providing runway to support Afesmi launch, sickle cell regulatory path and pipeline advancement.
Robust Pipeline with Multiple 2026 Catalysts
Key near-term catalysts: pre-sNDA meeting for mitapivat in sickle cell (Q1 2026); tebipivat Phase II top-line in lower-risk MDS (H1 2026) and Phase II sickle cell top-line (H2 2026); AG-236 Phase I top-line (H1 2026); initiation of AG-181 Phase 1b in PKU.
Commercial and Access Preparations
Single specialty pharmacy dispensing model and MyAgios patient support program in place; pharmacy education component of REMS completed; gross-to-net assumptions for thalassemia aligned with PK deficiency (~10%–20%).
Revenue Guidance and Operating Discipline
Company expects 2026 U.S. PK deficiency revenue of $45,000,000–$50,000,000 and anticipates operating expenses roughly flat with 2025, emphasizing disciplined capital allocation and a stated path to profitability.