Turning to first quarter highlights on the next slide, we delivered $20.7 million in net revenues, representing 138% growth year-over-year. commercial launch of AQVESME in thalassemia with the REMS fully operational as of the end of January, and already we have shown strong initial demand. Importantly, we've advanced two priorities that are key to our growth inflection. Over the longer term, we continue to advance our early-stage clinical programs and selectively evaluate expansion into other rare hematology diseases rather, to support our sustained growth.

We also reported $48 million in SG&A spend, up approximately $7 million from the prior year due to an increase in activities to support the U.S. commercial launch of AQVESME in thalassemia as well as an increase in stock compensation expense. We ended the quarter with over $1 billion in cash equivalents, and marketable securities, positioning us well to remain disciplined as we invest to maximize portfolio value and build a pipeline for long-term growth. Turning to our approach to capital allocation, on the next slide, our priorities remain clear.

We will continue to diversify our pipeline, leveraging both internal capabilities and external innovation as we continue to execute our 2026 priorities with a disciplined approach to long-term growth. In the U.S., net revenues were $18.8 million, driven by strong early AQVESME launch demand. Outside the U.S., we reported $1.9 million in net revenue, driven mainly by thalassemia utilization in the GCC. 242 prescriptions were written in the first quarter by REMS-certified physicians, serving as an important early indicator of strong demand.

What went well
  • Delivered $20.7M in worldwide mitapivat net revenues, up 138% year-over-year
  • Strong U.S. launch of AQVESME in thalassemia: 242 prescriptions by March 31 vs 44 at end of January
  • REMS became fully operational end of January with smooth onboarding and no payer hurdles so far
  • Ended quarter with over $1 billion in cash, equivalents and marketable securities
  • Aligned with FDA on Accelerated Approval path for mitapivat in sickle cell disease; sNDA now planned for Q2
What went wrong
  • Ex-U.S. revenue only $1.9M, reflecting early GCC market-access dynamics ahead of government procurement
  • R&D expense rose ~$8M year-over-year to $81M on workforce and process-development costs
  • SG&A rose ~$7M year-over-year to $48M on commercial launch and stock compensation
  • Q1 prescription pace not viewed as a sustainable run rate as adoption moves to less-engaged NTDT patients
  • Tebapivat low-risk MDS showed 50% lower drug exposure vs healthy volunteers, prompting higher-dose testing

Guidance Changes

MetricPeriodCurrent guidance
2026 operating expensesFY2026approximately flat vs 2025
PKD net revenueFY2026~$45M-$50M
Prescription-to-initiation timelinecoming quarters~10-12 weeks on average
Mitapivat sNDA filing (sickle cell disease)Q2 2026planned Q2 filing under Accelerated Approval

Performance Breakdown

MetricYoYNote
Worldwide mitapivat net revenue +138% U.S. commercial launch of AQVESME in thalassemia
U.S. net revenue — $18.8M driven by strong early AQVESME launch demand
Ex-U.S. net revenue — $1.9M, mainly GCC thalassemia utilization amid expected quarterly fluctuations
R&D expense +~$8M to $81M Workforce-related pipeline costs and increased mitapivat process development
SG&A expense +~$7M to $48M AQVESME U.S. launch support and higher stock compensation

Earnings Call Themes & Trends

TopicPrevious mentionCurrent periodTrend
AQVESME thalassemia launch44 prescriptions at end of January242 prescriptions by March 31, concentrated in motivated TDT and NTDT patients—
Mitapivat in sickle cell diseasepre-sNDA meeting completedaligned on Accelerated Approval path; sNDA filing planned Q2—
Prescription-to-initiation timing—faster than expected in Q1; expected to normalize to ~10-12 weeks—
Tebapivat next-gen PK activator—Phase IIb low-risk MDS data in 1H; Phase II sickle cell data in 2H 2026—
Pipeline market opportunity—pipeline represents greater than $10 billion potential 2030 market opportunity—

Q&A Summary

Are you seeing similar Q2 demand cadence to Q1, and is REMS-certified prescriber count increasing?
Management stuck to Q1 dynamics: 242 prescriptions from REMS-certified physicians by March 31, driven by highly engaged TDT and motivated NTDT patients; they cautioned against treating Q1 as a run rate but expect continued strong demand.
How do you view the mitapivat sickle cell opportunity given competitor data, and how does tebapivat fit?
They see sickle cell as supporting multiple treatment options and will maximize mitapivat's opportunity assuming a label, anchored by strong hemoglobin-responder RISE UP data and over 1,000 patient-years of experience; tebapivat is positioned for potential best-in-class pending Phase II data.
How many of the 242 scripts were trial patients transitioning, and how do they translate to revenue?
Open-label extensions in the THAL program continue but the U.S. proportion is small; PKD was guided to ~$45-50M for the year with Q1 in line, and the rest coming from thalassemia, with a lag from prescription to therapy initiation.
When will you get FDA feedback on the confirmatory study design, and will you share it?
Engagements with FDA have progressed well, enabling a Q2 filing; more confirmatory-trial detail will come before posting on clinicaltrials.gov and at/around EHA, with the trial prioritizing operational feasibility and probability of success.
Do you expect a REMS for sickle cell disease, and what do you want to see from tebapivat Phase II?
They have optionality with Endari and AQVESME as REMS options; the tebapivat Phase II is a dose-finding study looking for a dose to advance plus depth and breadth of hemoglobin response.
Given 44 scripts at end of January and 242 by March 31, can you reconcile the acceleration with the run-rate caution?
The 242 reflect the totality of the quarter with demand generated throughout January before REMS was operational; they declined to extrapolate the two data points but expect strong ongoing demand.
Have you lost any of the 242 patients, and what is the conversion rate?
Conversion from prescription to treatment was faster than expected and REMS is not a hurdle to initiation; they are confident in high conversion of early motivated patients, typical of rare disease launches.
Are the extra ~200 scripts patients written before January whose physicians only later became REMS-certified, and have you actually seen fill times slow?
The 242 reflect full-quarter launch preparation; motivated patients converted faster than 10-12 weeks in some cases, and the 10-12 week average is a forward expectation as adoption reaches NTDT patients with less frequent clinical engagement.
What treating physicians and settings are driving prescriptions?
Prescriptions come from both academic and community settings but the majority from community hematologist-oncologists across the country, consistent with where thalassemia patients are managed, with breadth expected to keep growing.
What are the implications of Novo's/HIBISCUS results for Agios and for driving tebapivat in sickle cell?
They have not seen full HIBISCUS results; they see clear room for multiple players given disease severity, will maximize mitapivat with strong RISE UP anti-hemolytic and hemoglobin-responder data, and remain pleased with FDA progress enabling the Q2 filing.

More on Agios Pharmaceuticals, Inc.

Reported 2026-04-29 · figures from the Agios Pharmaceuticals, Inc. Q1 2026 earnings call.

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