Agios Pharmaceuticals NASDAQ: AGIO reported first-quarter 2026 worldwide mitapivat net revenues of $20.7 million and highlighted early traction from the U.S. launch of AQVESME in thalassemia, alongside progress toward a potential U.S. Accelerated Approval filing for mitapivat in sickle cell disease.
First-quarter results and expense trends
Chief Financial Officer Cecilia Jones said the company delivered $20.7 million in worldwide mitapivat net revenues in the quarter, including $18.8 million in U.S. sales and $1.9 million outside the U.S. Jones said the U.S. performance was “driven by the recent launch of AQVESME in thalassemia,” while ex-U.S. results reflected “expected quarterly fluctuations.”
Agios reported first-quarter R&D expense of $81 million, which Jones said increased by roughly $8 million from the prior year due to “workforce-related expenses supporting pipeline advancement efforts” and higher mitapivat process development costs. SG&A expense was $48 million, up about $7 million year-over-year, driven by launch-related activities for AQVESME in thalassemia and higher stock-based compensation.
The company ended the quarter with “over $1 billion in cash equivalents and marketable securities,” which Jones said positions Agios to remain disciplined while investing in its portfolio and pipeline. Chief Executive Officer Brian Goff reiterated the company expects 2026 operating expenses to be “approximately flat versus 2025,” and Jones said that outlook includes the planned mitapivat confirmatory clinical trial in sickle cell disease.
AQVESME U.S. launch: early prescription volume and launch dynamics
Agios framed the first quarter as the start of its U.S. commercial launch of AQVESME in thalassemia, with the REMS “fully operational as of the end of January,” according to Goff. The company reported 242 prescriptions written as of March 31 by REMS-certified physicians, up from 44 prescriptions as of the end of January, which Goff said reflected “solid execution as the launch continues to broaden.”
Chief Commercial Officer Tsveta Milanova said early adoption was concentrated among “highly engaged patients,” including transfusion-dependent and “motivated non-transfusion-dependent” patients, which she described as consistent with early launch dynamics. She also said the time from prescription to initiation in the first quarter was shorter than expected, attributing that to patient motivation, physician readiness, and “effective REMS coordination.”
However, Milanova and management repeatedly cautioned that early prescription volume should not be viewed as a steady run rate. Milanova said, “I wouldn’t take Q1 to be the run rate for upcoming quarters,” and noted that as adoption broadens into patients with less frequent healthcare interactions, the company expects initiation timelines to average about 10 to 12 weeks in coming quarters.
In response to analyst questions about prescriber mix, Milanova said prescriptions came from both academic and community settings, with the “majority” coming from community-based hematologist-oncologists, which she said matched expectations because most thalassemia patients are managed in the community. She added that early prescribing showed “healthy geographic breadth” across the U.S.
On payer dynamics, Milanova said the company had not seen payer access as a hurdle so far and noted it can take “about 6 months on average to get the payer policies in place.” She added that payers have been receptive to AQVESME’s profile. In discussing conversion from prescription to treatment initiation, management said early conversion has been strong, with Goff saying Agios feels “very confident with the high conversion” among early patients.
Regulatory path: mitapivat sNDA in sickle cell disease
Goff said that following a pre-sNDA meeting with the FDA in the first quarter, Agios now plans to submit an sNDA for mitapivat in sickle cell disease in the second quarter under the Accelerated Approval pathway. Chief Medical Officer and Head of R&D Sarah Gheuens said that after the pre-sNDA meeting, Agios had a series of informal and formal engagements to gain “official alignment on the confirmatory clinical trial required under this pathway,” and the company is “pleased with the progression” of those discussions.
Gheuens said the company expects to share additional data from the phase III RISE UP trial at an upcoming medical congress, including analyses that informed selection of the confirmatory trial’s primary endpoint. She also said the confirmatory study design process has emphasized “operational feasibility,” including enrollment timelines and time to completion, while aiming to maximize the probability of success and potentially enhance the product label if full approval is achieved.
Asked about the specific confirmatory endpoint, Gheuens said it is informed by the RISE UP dataset and that the company plans to provide more details “ahead of our posting on clinicaltrials.gov,” alongside data presentations “at or around EHA.”
On the potential commercial opportunity in sickle cell disease, Milanova said Agios sees “a very significant commercial opportunity” for both mitapivat and tebapivat. She cited feedback from the caregiver community around the need for an anti-hemolytic agent and pointed to the company’s “strength of our hemoglobin responder data.” Gheuens said RISE UP data shared with physicians and patient communities has been “very well received,” and described sickle cell disease as “multidimensional,” noting Agios has seen impacts including on hospitalizations, fatigue, and quality of life in its dataset.
Pipeline updates and upcoming catalysts
Beyond commercial execution and regulatory work, Agios outlined multiple pipeline milestones for 2026. Gheuens said the company plans to initiate two pediatric mitapivat trials in thalassemia: ENERGIZE Kids T in transfusion-dependent patients and ENERGIZE Kids in non-transfusion-dependent patients.
Agios also highlighted tebapivat, its next-generation pyruvate kinase activator, with Gheuens describing it as structurally differentiated with “potent dual activation of PKR and PKM2” and pharmacokinetic properties supporting once-daily dosing without a taper. She cited early clinical observations in sickle cell disease including a long half-life of roughly 87 to 93 hours and a mean hemoglobin increase of 1.9 g/dL at a 5 mg once-daily dose, along with durable pharmacodynamic effects for up to four weeks after the last dose.
In lower-risk myelodysplastic syndromes (MDS), Gheuens said early clinical signals included transfusion independence in a low transfusion burden cohort, but Agios observed 50% lower drug exposure versus healthy volunteers, prompting evaluation at higher doses. She said an ongoing phase II-B study is evaluating 10 mg, 15 mg, and 20 mg doses, with eight consecutive weeks of transfusion independence as the primary endpoint.
On timing, management reiterated expectations for phase II-B top-line data for tebapivat in lower-risk MDS in the first half of 2026, followed by top-line phase II data for tebapivat in sickle cell disease in the second half of 2026.
Goff also previewed additional catalysts, including phase I healthy volunteer top-line data for AG-236 in the first half of the year and phase I-B proof-of-mechanism data for AG-181 in phenylketonuria in the second half.
In closing remarks, Goff said the first quarter reflected execution across Agios’ priorities, including the early AQVESME launch, advancement toward the mitapivat sickle cell sNDA filing, and a “disciplined financial approach.” He added the company plans to provide updates at a planned investor event during the 2026 EHA Congress.
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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