Agios Q4 2025 Results: AQVESME Launch Underway, SCD Regulatory Path in Focus

What happened

Agios Pharmaceuticals reported its fourth quarter and full-year 2025 financial results, with PYRUKYND® generating $20 million in net revenue for Q4 and $54 million for the year, indicating steady but modest growth in its core rare disease business. The company confirmed that AQVESME™ for thalassemia is now commercially available in the U.S. following FDA approval, a critical milestone that shifts attention to launch execution under a restrictive REMS program. Agios updated on its sickle cell disease (SCD) strategy, scheduling a pre-sNDA meeting with the FDA in Q1 2026 to discuss regulatory pathways after mixed Phase 3 results, keeping SCD as a key sentiment driver. Additionally, the Phase 2 tebapivat trial in SCD is fully enrolled, with topline results expected in H2 2026, adding longer-term pipeline optionality. With $1.2 billion in cash as of December 31, 2025, Agios maintains a strong liquidity position to fund its commercial expansion, but high operating expenses mean that slow AQVESME adoption could accelerate cash burn.

Implication

The financial results show PYRUKYND's incremental growth, but the real test is AQVESME's launch under REMS, where certification bottlenecks and liver monitoring could delay patient starts and revenue conversion. The SCD pre-sNDA meeting in Q1 2026 is a pivotal catalyst; constructive FDA engagement could de-risk the regulatory path, while negative feedback would cap upside and refocus valuation on thalassemia alone. Cash reserves of $1.2 billion provide a near-term buffer, but quarterly operating expenses near $128 million mean that poor AQVESME execution could quickly erode liquidity, increasing financing risk. The Phase 2 tebapivat trial offers future optionality, but its impact is distant and dependent on positive data, not altering the immediate investment case. Overall, the stock's re-rating hinges on proving operational excellence in thalassemia and securing a viable SCD filing strategy, with failure in either area likely to pressure shares.

Thesis delta

The news reinforces the existing investment thesis without a material shift, as AQVESME's commercial availability and SCD regulatory update align with prior expectations. However, the lack of detailed REMS metrics or early AQVESME adoption data leaves execution risks unchanged, emphasizing that the thesis still depends on overcoming operational hurdles and positive FDA feedback. Investors should maintain a cautious stance, awaiting concrete evidence from the upcoming catalysts to validate the base scenario of steady thalassemia ramp and SCD path clarity.