Agios Pharmaceuticals Q2 2025: Unraveling Key Contradictions in Safety, Spending, and Pediatric Strategy

Safety profile of mitapivat, SG&A spend trajectory, regulatory interaction and timeline, sickle cell trial protocol changes, and pediatric expansion timing and strategy are the key contradictions discussed in Agios Pharmaceuticals' latest 2025Q2 earnings call.



Revenue Growth and Pipeline Advancement:
- Agios Pharmaceuticals reported $12.5 million in net revenue for Q2 2025, reflecting a 45% increase from the previous year and a 44% increase from Q1 2025.
- The growth was driven by the strong value proposition of PYRUKYND, with continued commercial execution in PKD and an extra week of ordering in the second quarter, contributing to increased units processed directly by specialty pharmacy.

R&D and Pipeline Milestones:
- R&D expenses for the quarter were $91.9 million, an increase of $14.5 million compared to the previous year, primarily due to a $10 million milestone payment to partner Alnylam.
- Key R&D achievements included dosing the first patient in the Phase II trial of tebapivat in sickle cell disease and receiving IND clearance for AG-236, a siRNA targeting TEMPRSS6 for treating polycythemia vera, marking progress in pipeline advancement.

Commercial Expansion and International Agreements:
- Agios entered into agreements with Avanzanite Bioscience for the commercialization of PYRUKYND in Europe and NewBridge Pharmaceuticals for commercialization in the GCC, structured as revenue-sharing arrangements.
- These agreements are part of Agios' strategy to prioritize investment in potential U.S. launches, with partnerships supporting capital-efficient commercial build-outs outside of the U.S.

Thalassemia Launch Preparation:
- With less than 40 days to the September 7 PDUFA goal date for thalassemia, Agios is preparing for a potential launch in the U.S., pending FDA approval.
- The company anticipates that the launch in thalassemia will involve a shift in promotional focus from PKD, potentially impacting PKD demand during the transition.