Agios Pharmaceuticals, Inc. Q1 FY2026 earnings call

• Strategic priorities: 2026 strategic priorities are designed to drive near-term execution and long-term value creation. U.S. commercial launch of Acvesme in thalassemia is off to a strong start with 242 prescriptions by REM-certified physicians as of March 31st. - U.S. commercial launch: Acvesme U.S. commercial launch in thalassemia is off to a strong start, with early progress reflecting solid execution. The commercial team has rare disease capabilities. - FDA submission: Plan to submit an SNDA for mitopivet in sickle cell disease in the second quarter under U.S. accelerated approval pathway. - Pipeline: Advancing a robust pipeline anchored by PK Activation franchise. Plan to initiate two pediatric metapifat trials in thalassemia. Looking ahead to second-quarter readouts including Phase IIb top-line data for tepipivet in low-risk MDS. - Capital allocation: Priorities are to maximize U.S. commercial launch of Acvesme and thalassemia, manage operating expenses aligned with long-term value creation, and diversify pipeline.

In the first quarter, net revenues were $20.7 million, a 138% year-over-year growth. U.S. net revenues were $18.8 million driven by the launch of Acvesme in thalassemia, with 242 prescriptions written by REM-certified physicians as of March 31st. Outside the U.S., net revenue was $1.9 million. R&D expense was $81 million, an increase of ~$8 million from prior year due to workforce-related and meta-pivot process development expenses. SG&A spend was $48 million, up ~$7 million from prior year due to U.S. commercial launch activities and stock compensation expense.

• 2026 operating expenses expected to be approximately flat versus 2025. - Plan to submit an SNDA for mitopivet in sickle cell disease in the second quarter under U.S. accelerated approval pathway. - Anticipate phase 2b top-line data for tepipivet in low-risk MDS in the first half of the year, phase two top line data for tepipivet in sickle cell disease in the second half of 2026, phase one healthy volunteer top line data for AG236, phase 1B proof of mechanism data for AG181 in fetal ketonuria.

Q: Samantha Semenkow from Citi asked about the demand of Exvesme in the second quarter and increase in REM-certified healthcare professionals.

A: Sveta said early uptake is driven by highly engaged and motivated patients, not to take Q1 as run rate for upcoming quarters but expect strong demand as team executes.

Q: Alec Stranahan from Bank of America asked about covering costs of therapy for patients transitioning from clinical study to commercial drugs and when to expect feedback on confirmatory study design for SMDA.

A: Sarah commented on open-label extensions for thalassemia patients and that teams are making progress towards deliverables.

Q: Emily Bodner from HC Wainwright asked about REMS for sickle cell disease and expectations for Phase II sickle cell data for Tempipivac.

A: Sarah said teams have optionality with N-parakind and Agvesme, and looking for dose and hemoglobin response in Phase II.

Q: Eric Schmidt from Cantor asked about confirming acceleration in prescriptions from February to March and square with comments on not extrapolating run rate.

A: Sveta said 242 prescriptions reflect whole quarter's work, demand generated throughout quarter, and not to extrapolate run rate but expect strong demand.

Q: Mark Fromm from TD Calvin asked about whether prescriptions before January were by non-REM certified physicians and caution around trajectory slowing.

A: Sveta said 242 prescriptions reflect whole quarter's work, prescriptions from healthy geographic breadth and community prescribers, and expect average initiation timelines of 10-12 weeks going forward.

Q: Salveen Richter from Goldman Sachs asked about treating physicians and treatment settings for ECVESMI launch.

A: Sveta said majority of prescriptions coming from community setting, with prescriptions from academic and community settings, and will focus on breadth.

Q: Romero from JP Morgan asked about implications of Novo's recent results on Agios and tebapivet in sickle cell disease.

A: Sarah said see clear commercial opportunity in sickle cell disease with mitopivac and look to maximize commercial opportunity, and pleased with progression of engagements with FDA for mitopivac filing.

Q: Greg Renza with Truist asked about early signals of patient persistence with Agvesme and how to think about opportunity for early uptake with mid-pivot in sickle cell disease assuming accelerated approval.

A: Sveta said early in launch, monitor patient persistence, and Sveta and team preparing commercially for potential opportunity with accelerated approval pathway, and confirmatory trial prioritizes operational feasibility and probability of success.