Our third quarter earnings call agenda is shown on the next slide. In the third quarter, we reported $12.9 million in net revenue, underscoring the strong value proposition of PYRUKYND. With continued momentum across our commercial portfolio and pipeline, our team has demonstrated strong execution and agility, keeping us firmly focused on our mission to deliver transformative medicines for patients. Please move to the next slide and I'll turn the call over to Cecilia to provide commentary on our third quarter performance and full year outlook.
Third quarter net revenue growth reflects continued commercial execution in PKD ahead of potential U.S. R&D expenses were $86.8 million, an increase of $14.3 million compared to the third quarter of 2024. This increase was primarily driven by increased clinical trial costs associated with our PK activation franchise. SG&A expenses were $41.3 million in the third quarter, an increase of $2.7 million compared to the prior year, driven by disciplined investment.
Our capital allocation strategy, backed by a strong balance sheet, enables strategic investment in future growth and delivery of our ongoing pipeline programs. First, we have built a capital-efficient global commercial model, prioritizing our investment in potential U.S. We have executed partnerships with NewBridge Pharmaceuticals in the GCC and Avanzanite Bioscience in Europe, both of which are structured as revenue-sharing arrangements that favor Agios. Over the long term, we will record our share of sales as net revenues.
| Metric | Period | Current guidance |
|---|---|---|
| PYRUKYND thalassemia PDUFA date | 2025 | extended to December 7, 2025 following FDA REMS request |
| Full-year 2025 net revenue | FY2025 | robust growth vs 2024, on a relatively small revenue base; Q4 to benefit from an additional ordering week |
| RISE UP phase III sickle cell top-line results | 2025 | reiterated by year end |
| Tebapivat phase II-B lower-risk MDS top-line data | early 2026 | reiterated early 2026 following enrollment completion |
| Metric | YoY | Note |
|---|---|---|
| PYRUKYND net revenue | +44% ($12.9M vs $9.0M) | continued commercial execution in PKD ahead of potential U.S. thalassemia approval; also +3% sequentially vs $12.5M in Q2 2025 |
| R&D expense | +$14.3M ($86.8M) | primarily increased clinical trial costs associated with the PK activation franchise |
| SG&A expense | +$2.7M ($41.3M) | disciplined investment ahead of the potential PYRUKYND thalassemia commercial launch |
| Cost of sales | — | $1.7M for the quarter |
| Cash, cash equivalents and marketable securities | — | ended Q3 at approximately $1.3B, supporting U.S. launches and pipeline |
| Patients on therapy | — | 149 patients on therapy, up 5% sequentially; 262 cumulative prescription enrollment forms with 14 added in Q3 (+6%) |
| Topic | Previous mention | Current period | Trend |
|---|---|---|---|
| Thalassemia launch readiness under REMS | Original PDUFA date without a REMS requirement | PDUFA extended to Dec 7; management uses added time for community engagement and asserts REMS is not viewed as a barrier to prescribing | — |
| Global commercialization model | U.S.-centric launch focus | capital-efficient ex-U.S. partnerships (NewBridge in GCC, Avanzanite in Europe) via revenue-sharing arrangements favoring Agios, preserving capital for U.S. launches | — |
| Sickle cell disease opportunity | RISE UP phase III enrolling/ongoing | top-line results expected by year end; positioned as potential first-in-class oral therapy targeting hemolysis and vaso-occlusion | — |
| Pipeline diversification / business development | Organic pipeline focus | actively and opportunistically pursuing internal and external assets, not tied to any specific catalyst or the sickle cell readout | — |
| Tebapivat in lower-risk MDS | Phase II-B enrolling | enrollment complete; positioned as potential first oral therapy for anemia due to ineffective erythropoiesis, data early 2026 | — |