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    AGIO
    Earnings call· Jun 2026(Q2 FY26)

    AGIOS PHARMACEUTICALS Q2 FY26 earnings call AGIO

    Jul 30, 2026 Source

    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

    3
    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

    3
    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———

    Operational metrics

    9
    Cost of sales
    $3 million
    Q2 FY26

    Cost of sales for the quarter.

    Research and Development Expense
    $100.8 millionvs $91.9 million in Q2 FY25
    Q2 FY26

    Primarily due to in-process R&D increase, including a $25 million upfront payment for cevidoplenib in-licensing.

    Selling, General and Administrative Expense
    $51.5 millionvs $45.9 million in Q2 FY25
    Q2 FY26

    Reflecting an increase in commercial-related activities for AQVESME launch.

    Net Loss
    $100.7 millionvs $112 million in Q2 FY25
    Q2 FY26

    Net loss for the second quarter.

    Cash, Cash Equivalents and Marketable Securities
    $1 billion
    Q2 FY26

    Ended the quarter with strong financial flexibility.

    AQVESME Gross to Net
    10% to 20%
    Ongoing

    Expected range with quarter-to-quarter variability.

    AQVESME Time to Treatment Initiation
    10 to 12 weekstrending towards
    Launch maturity

    Anticipated range as adoption broadens across the NTDT population.

    Sickle Cell Disease Target Patient Population
    25,000
    Initial focus

    Approximately 25,000 patients actively treated or in need of therapy today.

    Sickle Cell Disease Medicaid Rebate
    23%
    Ongoing

    Mandatory rebate for higher Medicaid proportion in sickle cell disease population, driving higher gross-to-net.

    Industry KPIs

    5
    MetricValueDetails
    Launch access metrics75%%
    Pipeline read out calendarAG-181 Phase Ib data
    Regulatory approvals filingsMitapivat sNDA for sickle cell disease
    Prescription volume new starts442prescriptions
    Clinical trial efficacy safety dataRISE UP Phase III (sickle cell)

    Deals & partnerships

    1
    OscotecIn-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

    5
    Natural moderation of new patient starts for AQVESMEOngoing, 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 AQVESMEOngoing

    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) patientsOngoing, 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 patientsSecond 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-netUpon 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

    5

    Mitapivat sNDA approval for sickle cell disease

    Q4 FY26
    CurrentPDUFA goal date of November 1
    TargetFDA 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.

    Q&A highlights

    6

    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 read5 chapters

    Detailed Narrative

    01

    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.

    02

    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.

    03

    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.

    04

    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.

    05

    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.

    AI-generated summary of the company’s earnings call. Not investment advice.