Agios Pharmaceuticals Q2 Earnings Call Highlights

Key Points

• Mitapivat revenue reached $44.7 million in Q2 2026, while Agios reported a $100.7 million net loss and ended June with approximately $1 billion in cash and marketable securities. The company maintained its 2026 U.S. pyruvate kinase deficiency revenue outlook of $45 million to $50 million.
• AQVESME’s U.S. thalassemia launch continued to expand, with 442 cumulative prescriptions and payer coverage for roughly 75% of patient lives. Agios plans to stop reporting prescription counts after Q3 and use revenue as its primary commercial metric.
• The FDA set Nov. 1 as the decision date for mitapivat in sickle cell disease, though Agios does not expect a material 2026 revenue contribution. The company is also advancing pipeline programs in immune thrombocytopenia, polycythemia vera and phenylketonuria.

Agios Pharmaceuticals NASDAQ: AGIO reported second-quarter 2026 mitapivat net revenue of $44.7 million, including $40.9 million in the United States and $3.8 million outside the U.S., as the company continued the U.S. thalassemia launch of AQVESME and prepared for a potential sickle cell disease approval later this year.

The company recorded a net loss of $100.7 million for the quarter, compared with a $112 million loss in the prior-year period. Agios ended June with approximately $1 billion in cash equivalents and marketable securities.

Chief Executive Officer Brian Goff said the company is pursuing several growth drivers, including the thalassemia launch, a potential expansion of mitapivat into sickle cell disease, and pipeline programs in rare hematology and other rare diseases.

Revenue, expenses and outlook

Cost of sales totaled $3 million during the quarter. Research and development expense rose to $100.8 million from $91.9 million a year earlier, primarily reflecting a $15 million increase in process research and development tied to the company’s agreement with Oscotec, including a $25 million upfront payment associated with the cevidoplenib licensing transaction.

Selling, general and administrative expense increased to $61.5 million from $45.9 million in the second quarter of 2025, which Chief Financial Officer Cecilia Jones attributed to commercial activities associated with the February launch of AQVESME.

Agios maintained its outlook for approximately $45 million to $50 million in 2026 U.S. revenue from pyruvate kinase deficiency. The company expects full-year operating expenses to be approximately flat compared with 2025, excluding the $25 million cevidoplenib upfront payment. Its expense outlook includes investment to prepare for a possible sickle cell disease launch.

Thalassemia launch progresses

Agios reported 442 cumulative AQVESME prescriptions from REMS-certified physicians as of June 30, including 200 additional prescriptions during the second quarter. The metric represents unique prescriptions for patients with completed START forms from REMS-certified physicians.

Chief Commercial Officer Tsveta Milanova said second-quarter revenue included about $5 million in one-time benefits related to thalassemia stocking, along with modest favorable gross-to-net dynamics. The company continues to expect gross-to-net adjustments in a 10% to 20% range, though it cautioned that results can vary by quarter.

Approximately 75% of thalassemia patient lives are now covered under payer policies, according to Milanova. She said the mix of prescriptions is increasingly shifting toward non-transfusion-dependent thalassemia patients, who may have less frequent interactions with health-care providers and more deliberate treatment discussions.

Agios said time from demand to treatment initiation has begun moving toward its anticipated 10- to 12-week range as adoption broadens. The company expects it to move within that range as it further penetrates the non-transfusion-dependent population.

The company plans to stop reporting prescriptions from REMS-certified physicians after the third quarter and instead use revenue as its primary commercial performance measure. Milanova said revenue better incorporates new patient starts, treatment initiation timing, refills and persistence on therapy as the launch matures.

Management said the earliest AQVESME patients are approaching six months of treatment, a point at which physicians may assess clinical benefit and determine whether therapy should continue. In response to analyst questions, Chief Medical Officer and Head of Research and Development Sarah Gheuens said more than 90% of patients in the ENERGIZE study continued treatment into its open-label extension, while noting that real-world persistence is still in the early stages of assessment.

Sickle cell decision expected Nov. 1

The Food and Drug Administration accepted Agios’ supplemental new drug application for mitapivat in sickle cell disease and granted priority review, setting a Prescription Drug User Fee Act goal date of Nov. 1.

Agios recently dosed the first patient in REIGNITE, its Phase III confirmatory trial in sickle cell disease. Gheuens said data presented at the European Hematology Association meeting showed hemoglobin responses in the RISE UP Phase III study, with responders experiencing improvements in sickle cell pain crisis-related endpoints and fatigue. The company also presented data on reductions in transfusion burden and red blood cell units transfused.

Agios is initially focusing its potential sickle cell launch on roughly 25,000 patients who are actively treated or need therapy. Management said sickle cell disease has a higher Medicaid mix than its existing commercial markets, which would lead to higher gross-to-net adjustments because of mandatory Medicaid rebates.

Gheuens said the company had not disclosed which brand name would be used for sickle cell disease if approved. She added that hepatocellular injury was not observed among sickle cell trial participants and that a REMS program may not be warranted, though the company said it is prepared to launch with or without one. Agios does not expect sickle cell disease to materially contribute to 2026 revenue given the November decision date.

Pipeline additions and development plans

During the quarter, Agios in-licensed cevidoplenib, a selective oral Syk inhibitor being developed for immune thrombocytopenia. The company said it intends to engage with the FDA in coming months to align on advancement to Phase III. Gheuens said available Phase II data showed dose-dependent activity, no dose-limiting toxicity through Phase II and evidence of durable platelet responses.

Agios also plans to initiate the Phase II portion of an operationally seamless Phase II/III program for AG-236 in polycythemia vera during the second half of 2026. The program is designed to identify an optimal dose and advance efficiently into a registrational stage.

Elsewhere, the company expects Phase Ib proof-of-mechanism data for AG-181 in phenylketonuria during the second half of the year. Goff said Agios is prioritizing investments in programs it believes can support long-term growth across rare disease markets that management estimates could exceed $10 billion in 2030.

About Agios Pharmaceuticals (NASDAQ:AGIO)

Agios Pharmaceuticals, Inc is a biopharmaceutical company founded in 2008 as a spin-out from research at Dana-Farber Cancer Institute and the Broad Institute. Headquartered in Cambridge, Massachusetts, Agios focuses on understanding and targeting cellular metabolism to develop novel therapies for cancer and rare genetic diseases. The company's scientific platform integrates genomic discovery, metabolic profiling and precision medicine approaches to identify and advance small-molecule candidates that correct or exploit metabolic dysfunction.

Agios's lead products are IDH (isocitrate dehydrogenase) inhibitors that target specific cancer mutations.

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