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Agios Pharmaceuticals (AGIO) Q2 FY26 earnings call: guidance and Q&A

Earnings call· Jun 2026 (Q2 FY26)· Health Care· Jul 30, 2026

Source: earnings call transcript

Executive summary

Agios Pharmaceuticals Q2 FY26 — Strong Commercial Performance and Pipeline Advancement

Agios Pharmaceuticals reported a strong second quarter, driven by robust commercial execution for AQVESME in thalassemia and significant pipeline progress, including the in-licensing of cevidoplenib and advancing Mitapivat for sickle cell disease with a November 1 PDUFA date. The company is strategically diversifying its rare hematology portfolio while maintaining a strong financial position, positioning it for long-term growth despite anticipated shifts in launch dynamics for AQVESME.

Highlights

5

  • Delivered $44.7 million in total net revenue, including $40.9 million in the U.S.

  • Achieved 442 cumulative AQVESME prescriptions from REMS-certified physicians as of June 30.

  • Strengthened pipeline with in-licensing of cevidoplenib for ITP, progressing towards Phase III.

  • Received FDA acceptance of sNDA for Mitapivat in sickle cell disease with Priority Review and a PDUFA goal date of November 1.

  • Ended the quarter with approximately $1 billion in cash, cash equivalents, and marketable securities.

Concerns

4

  • Net loss for Q2 FY26 was $100.7 million, compared to $112 million in Q2 FY25.

  • Anticipate natural moderation in new patient starts as adoption broadens beyond highly motivated patients, leading to quarter-to-quarter revenue variability.

  • Time to treatment initiation for AQVESME is trending towards the anticipated 10- to 12-week range as adoption expands into the non-transfusion-dependent (NTDT) population.

  • Sickle cell disease population has a higher Medicaid proportion, which will drive gross-to-net to a higher level (23% mandatory rebate) compared to PKD and thalassemia.

Guidance & targets

CategoryTargetConfidence
PK deficiency revenues
$45 million to $50 million
medium materiality
High
Full-year operating expenses (adjusted)
approximately flat versus 2025
medium materiality
High
Mitapivat sNDA PDUFA date for sickle cell disease
November 1
high materiality
High

Segment performance

SegmentRevenueYoYQoQMargin
Total Mitapivat
Total net revenue for the quarter.
$44.7 million———
Mitapivat U.S.
U.S. net revenue for Mitapivat.
$40.9 million———
Mitapivat ex-U.S.
Ex-U.S. net sales, reflecting demand in GCC and Europe.
$3.8 million———

Deals & partnerships

Oscotec In-licensing of cevidoplenib, a next-generation, highly selective oral SYK inhibitor for immune thrombocytopenia (ITP). $25 million upfront payment

Strengthened the pipeline and expanded reach within rare hematology.

Risks & headwinds

Natural moderation of new patient starts for AQVESME Ongoing, as launch matures

Not quantified, but described as 'naturally moderates as adoption broadens beyond the earliest wave of highly motivated patients and prescribers'.

Mitigation:Focus on expanding reach across the thalassemia community, expanding adoption in the non-transfusion-dependent segment, and adding new prescribers.

Quarter-to-quarter revenue variability for AQVESME Ongoing

Not quantified, but attributed to 'order timing, inventory movement and gross to net dynamics'.

Mitigation:Transitioning to revenue as primary measure of commercial performance after Q3 FY26, away from prescription counts.

Increased time to treatment initiation for non-transfusion-dependent (NTDT) patients Ongoing, as patient mix shifts

Trending towards anticipated 10- to 12-week range.

Mitigation:Acknowledged as a natural evolution of the launch; teams are prepared for this dynamic.

6-month clinical assessment point for AQVESME patients Second half of the first launch year

First cohort of patients approaching 6 months of treatment, a natural point for physicians to assess clinical response.

Mitigation:Building broader real-world understanding of how physicians and patients evaluate response and integrate Mitapivat into long-term care; confident in continuation rates based on clinical trials.

Higher Medicaid mix for sickle cell disease impacting gross-to-net Upon potential sickle cell disease launch

Mandatory rebate of 23%, which will drive the gross to net to a higher level compared to PKD and thalassemia.

Mitigation:Will provide specifics on pricing at the time of approval, driven by label and competitive environment; strong market access team leveraging thalassemia experience.

What to watch in Q3 FY26

Mitapivat sNDA approval for sickle cell disease

Q4 FY26
Current PDUFA goal date of November 1
Target FDA approval

Why it matters

Represents a significant expansion opportunity for the PK activation franchise and a potential next growth driver for the company.

We received FDA acceptance of our sNDA with priority review and were assigned a PDUFA goal date of November 1, bringing us one step closer to delivering a first-in-class medicine in an area of significant unmet need.

Find out whether AGIO delivers this. Inve re-checks your case every morning.

Q&A highlights

Inquired if ENERGIZE studies are a good barometer for time on treatment, if time to treatment changed QoQ, and if repeat prescriptions are being seen.

Management stated that initial time to treatment was faster than anticipated (10-12 weeks) due to highly motivated patients, but is now trending towards the 10-12 week range as NTDT adoption broadens. They expect continuation rates to be in line with ENERGIZE study data and are seeing repeat prescriptions.

“Currently, what we see from the market is a very kind of strong feedback and a positive feedback from the community. So we expect continuation rates to be in line with the ENERGIZE study.”

asked by Alec Stranahan · answered by Tsveta Milanova

2 min read 5 chapters

Detailed narrative

AQVESME Launch Momentum

Agios reported strong commercial performance for AQVESME in thalassemia, with $44.7 million in total net revenue, including $40.9 million from the U.S. and 442 cumulative prescriptions from REMS-certified physicians as of June 30. The launch is progressing beyond initial highly motivated patients into a broader non-transfusion-dependent (NTDT) population, with payer coverage strengthening to approximately 75% of thalassemia lives.

Mitapivat in Sickle Cell Disease

The company is advancing Mitapivat towards a potential new indication in sickle cell disease, with the FDA accepting its sNDA for priority review and assigning a PDUFA goal date of November 1. A Phase III confirmatory trial, REIGNITE, has dosed its first patient. Management is actively preparing for a potential launch, targeting approximately 25,000 actively treated patients, leveraging existing thalassemia launch capabilities.

Pipeline Diversification and Advancement

Agios further diversified its pipeline by in-licensing cevidoplenib, an oral SYK inhibitor for immune thrombocytopenia (ITP), which is progressing towards Phase III. Additionally, AG-236, an siRNA TMPRSS6 inhibitor for polycythemia vera, is advancing into a seamless Phase II/III program, with Phase II initiations planned for H2 2026. AG-181 for phenylketonuria is also progressing, with Phase Ib proof-of-mechanism data expected in H2 2026.

Financial Strength and Capital Allocation

The quarter ended with approximately $1 billion in cash, cash equivalents, and marketable securities, providing financial flexibility for commercial execution and pipeline advancement. The company emphasizes disciplined capital allocation, focusing investments on opportunities with the greatest potential to create long-term value for patients and shareholders.

EHA Data Presentations

At EHA in June, Agios presented broad data for Mitapivat in both thalassemia and sickle cell disease. The RISE UP Phase III study in sickle cell demonstrated hemoglobin responses and clinically meaningful improvements in pain crisis endpoints and fatigue, with new data showing reductions in transfusion burden. Open-label extension data for thalassemia showed 60% of patients achieved hemoglobin response, including NTDT patients with high baseline hemoglobin levels.

Summary of the company's earnings call; see how call summaries are prepared. Not investment advice.