We also advanced AG-236 into an operationally seamless Phase II/III program in polycythemia vera, adding another potential growth driver within hematology. Turning to our second quarter highlights on the next slide, we delivered a quarter marked by strong commercial performance, meaningful pipeline progress, and continued portfolio discipline. First, we delivered sustained commercial momentum with $44.7 million in total net revenue, including $40.9 million in the U.S. Second, we further diversified our pipeline through the in-licensing of cevidoplenib, a next-generation, highly selective oral Syk inhibitor for ITP, progressing toward phase III and strengthening our rare hematology pipeline.

Finally, we ended the quarter with approximately $1 billion in cash equivalents, and marketable securities, providing financial flexibility to support both commercial growth and pipeline progression. Overall, we entered the second half of 2026 with strong commercial delivery, a more diversified pipeline, an important near-term regulatory catalyst, and the capital position to execute on our strategy. Turning to our second quarter financial results, total mitapivat net revenue was $44.7 million, including $40.9 million in the U.S. Selling, general, and administrative expense was $61.5 million, compared to $45.9 million in the prior year's period, reflecting an increase in commercial-related activities as we executed a launch of AQVESME in February.

Turning to our outlook for 2026, we continue to expect approximately $45 million-$50 million from PK deficiency revenues in the U.S. Our priorities for the remainder of the year remain clear: driving the AQVESME launch, preparing for a potential sickle cell disease approval, advancing our pipeline, and maintaining financial discipline. In the U.S., performance reflected continued growth in thalassemia demand and solid commercial execution. Net revenue in the quarter reflected approximately $5 million of one-time benefits related to stocking in thalassemia, along with modest gross-to-net favorability.

What went well
  • Total mitapivat net revenue of $44.7M ($40.9M U.S., $3.8M ex-U.S.) reflecting sustained commercial momentum
  • AQVESME thalassemia launch added 200 REMS-certified prescriptions in Q2, reaching 442 cumulative as of June 30
  • FDA accepted the mitapivat sickle cell disease sNDA with priority review and a November 1 PDUFA goal date
  • Ended the quarter with approximately $1B in cash, equivalents and marketable securities
  • Diversified the pipeline via in-licensing of cevidoplenib (oral Syk inhibitor) for ITP and advanced AG-236 into a Phase II/III program in polycythemia vera
  • Thalassemia payer coverage strengthened to roughly 75% of lives under payer policies
What went wrong
  • Net loss of $100.7M for the quarter (though narrower than the $112M prior-year loss)
  • R&D expense rose to $100.8M from $91.9M, driven by a $25M upfront payment tied to the cevidoplenib/Oscotec deal
  • SG&A jumped to $61.5M from $45.9M on increased commercial activity for the AQVESME launch
  • Revenue benefited from ~$5M of one-time thalassemia stocking, implying underlying demand is lower than headline
  • New patient start growth is naturally moderating as the launch moves beyond the earliest highly motivated patients

Guidance Changes

MetricPeriodCurrent guidance
U.S. PK deficiency revenueFY2026~$45M-$50M (reaffirmed)
Full-year operating expensesFY2026approximately flat vs 2025 (excl. $25M cevidoplenib upfront)
Gross-to-netongoing10%-20% (reaffirmed, with quarter-over-quarter variability)

Performance Breakdown

MetricYoYNote
Total mitapivat net revenue Q2 2026 total net revenue of $44.7M; no prior-year comparator disclosed on the call
R&D expense up ~$8.9M ($100.8M vs $91.9M) Increase in process R&D of ~$15M driven by the $25M upfront payment tied to the Oscotec/cevidoplenib agreement
SG&A expense up ~$15.6M ($61.5M vs $45.9M) Higher commercial-related activities following the February AQVESME launch
Net loss improved ~$11.3M ($100.7M vs $112M) Revenue growth partially offsetting higher operating expenses

Earnings Call Themes & Trends

TopicPrevious mentionCurrent periodTrend
Thalassemia launch metricReporting cumulative REMS-certified prescriptions as demand indicatorWill discontinue prescription reporting after Q3 and transition to revenue as primary performance measure
Patient mixEarly adoption skewed to highly motivated transfusion-dependent (TDT) patientsGrowing proportion of non-transfusion-dependent (NTDT) patients, the larger commercial opportunity
Time to treatment initiationFaster than the anticipated 10-12-week range due to motivated early patientsTrending toward and expected to stabilize within the 10-12-week range as NTDT adoption broadens
Sickle cell diseasesNDA under FDA reviewAccepted with priority review, Nov 1 PDUFA; first patient dosed in REIGNITE confirmatory trial; commercial launch prep underway
Pipeline breadthAnchored by mitapivatExpanded into ITP (cevidoplenib), polycythemia vera (AG-236), and PKU (AG-181)

Q&A Summary

Is time on/to treatment moving toward the 10-12-week range, and are you seeing repeat REMS prescriptions yet?
Early quarters benefited from faster-than-expected time to treatment from motivated patients; as adoption moves into NTDT settings it is trending toward the 10-12-week range. Continuation is expected in line with the ENERGIZE study, and repeat prescriptions are already being seen with the REMS process running smoothly.
What gives confidence on persistence, and what factors decide whether sickle cell is added to AQVESME or PYRUKYND (and whether a REMS or thalassemia price cut is needed)?
Open-label extension data (over 90% continuation from ENERGIZE, rising to ~60% response with longer exposure) are the best current proxy for persistence. The sickle cell brand name is undecided; because no hepatocellular injury was seen in SCD patients, a REMS may not be warranted, and teams are ready to launch with or without one.
How should we think about the run rate of new patient starts in H2, and where are you within the 10%-20% gross-to-net range?
Prescription and revenue growth will no longer correlate directly as the launch matures and shifts toward NTDT patients with less frequent visits. Gross-to-net is expected to remain within the 10%-20% range with quarter-over-quarter variability.
Is the 200 patient additions per quarter a sustainable rate, or does it still reflect the Q1 backlog bolus?
Engagement is a gradient and the launch is still on the front end of it; as adoption moves deeper into the NTDT population with fewer clinical interactions, prescription and revenue growth will decouple, which is why the company will move to revenue as the primary metric after Q3.
What clinical bar do physicians use at the six-month evaluation point, and how strict is it?
It varies patient-by-patient. For TDT patients physicians weigh transfusion reduction (not strictly the trial's 50% threshold) and how patients feel; for NTDT patients they look at hemoglobin improvement (not a hard 1 g/dL cutoff), hemolytic parameters, and especially reductions in fatigue.
What exactly is in the 442 cumulative prescriptions, and do you know refill rates?
The figure represents unique patient prescriptions equivalent to completed START forms written by REMS-certified physicians, excluding refills. Refills are continuing according to plan as patients reach their second and third months, but specific refill dynamics are not being disclosed.
Were any Q2 revenues from Europe, and will sickle cell contribute revenue in Q4?
Ex-U.S. revenue reflects continued GCC demand plus anticipated European thalassemia demand following May approval, but the vast majority of revenue is still expected from the U.S. and neither ex-U.S. region nor a November sickle cell approval will be a material 2026 contributor.
What is the TDT vs NTDT breakout and when will NTDT be the majority; is 10-12 weeks the long-run run rate?
NTDT is a growing proportion after an early TDT skew; of roughly 4,000 initially targeted patients about 50% are NTDT, and the 10-12-week time to treatment initiation is expected to stabilize and remain constant over time.
What is the LOE for mitapivat, and when might you guide to revenue once scripts stop being reported?
Composition-of-matter LOE is 2035 plus potential extensions. The company is not giving specific script guidance; it will assess the appropriate time to provide franchise revenue guidance, likely around the potential November sickle cell approval.
Does sickle cell have a higher Medicaid mix affecting gross-to-net and pricing versus Novo competition?
Yes, SCD has a higher Medicaid proportion carrying a mandatory 23% rebate, which will push gross-to-net higher than PKU and thalassemia. Specific pricing will be set at approval based on the label and competitive environment.

More on Agios Pharmaceuticals, Inc.

Reported 2026-07-30 · figures from the Agios Pharmaceuticals, Inc. Q2 2026 earnings call.

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