Agios Pharmaceuticals (NASDAQ:AGIO) reported record third-quarter product revenue as its thalassemia drug AQVESME continued its U.S. launch, while the company prepares for a delayed regulatory decision on the drug’s use in sickle cell disease.
Total mitapivat net revenue rose to $55.4 million from $12.9 million a year earlier. The company’s net loss narrowed to $65 million from $103.4 million, Chief Financial Officer Cecilia Jones said on the earnings call.
Chief Executive Officer Brian Goff said the company is targeting non-GAAP profitability in 2028 and expects savings next year from a reduction of more than 100 research and development roles during 2026.
AQVESME launch drives revenue growth
U.S. net revenue was $48.7 million, up 20% from the previous quarter, driven by AQVESME demand across the thalassemia population, Chief Commercial Officer Tsveta Milanova said. Revenue outside the U.S. was $6.6 million, up 74% sequentially, supported by European early access programs following approval for thalassemia in the second quarter.
Agios added 177 prescriptions with completed patient-support start forms from REMS-certified physicians during the quarter, bringing the total to 619 since launch. Another 48 prescriptions had start forms in process at quarter-end. Milanova said all of those prescriptions were for thalassemia, and that the company would stop reporting the metric after this quarter.
The 619 completed forms represent roughly 15% of the company’s initial target population of about 4,000 U.S. thalassemia patients. Approximately 75% of patients remained on therapy at about six months, Milanova said.
Earlier quarters benefited from pent-up demand, while adoption is now broadening into community practices and among patients who are not transfusion-dependent, where treatment decisions can take longer, she said. Asked about the pace of new prescriptions, Milanova described the 177 recorded in the third quarter as a more appropriate run rate as Agios continues to penetrate those markets.
Sickle cell decision moved to February
The Food and Drug Administration extended its review of mitapivat for sickle cell disease by three months, to a Feb. 1, 2027, decision goal date, after classifying the company’s response to a request for additional information as a major amendment.
Goff said the agency requested information to evaluate whether a Risk Evaluation and Mitigation Strategy, or REMS, is necessary. Agios responded with approximately nine months of additional safety data from the RISE UP trial’s open-label extension, he said.
Goff said there continued to be no cases of liver injury in sickle cell patients similar in pattern or severity to those seen in thalassemia. The company is in labeling discussions with the FDA, including the elements of a potential REMS. Chief Medical Officer Sarah Gheuens said the requirements could range from educational measures to more extensive restrictions, but declined to specify what was under discussion.
Milanova said the company views the delay as a change in timing rather than in the commercial opportunity. If approved, Agios plans to focus initially on about 25,000 U.S. patients who are actively treated or in need of therapy, prioritizing those with low or declining hemoglobin. Its sales, medical and patient-support teams are hired and trained, she said.
Pipeline data and spending plans
Gheuens said Agios plans to present additional RISE UP data at the American Society of Hematology annual meeting in December. In the open-label extension, 62.7% of patients achieved a hemoglobin improvement, including 39.7% of those who had not initially responded. The company also reported kidney and cardiac signals from post-hoc analyses; Gheuens said these suggested potential organ-health benefits.
Agios expects to begin a phase II/III program for AG-236 in polycythemia vera by year-end. In a phase I healthy-volunteer study, the drug’s effects on measures including hemoglobin and hematocrit lasted through month six after a single dose, Gheuens said. The planned phase II portion will test doses and dosing frequencies in patients.
The company also plans a phase III trial of cevidoplenib for immune thrombocytopenia in the first half of 2028 and expects proof-of-mechanism data for AG-181 in phenylketonuria before year-end. Goff said Agios retains the option to keep AG-181 in its portfolio while exploring ways to unlock value from non-core assets.
Third-quarter research and development expense fell to $73.5 million from $86.8 million, while selling, general and administrative expense rose to $56.5 million from $41.3 million as Agios supported the thalassemia launch and prepared for a potential sickle cell launch. The company ended the quarter with $907 million in cash, cash equivalents and marketable securities.
Jones said it was too early to provide 2027 guidance. The 2028 non-GAAP profitability target assumes a potential sickle cell launch in 2027 and disciplined operating expense management, she said.
About Agios Pharmaceuticals (NASDAQ:AGIO)
Agios Pharmaceuticals, Inc is a biopharmaceutical company focused on discovering and developing medicines for rare diseases involving cellular metabolism. The company’s research is centered on metabolic pathways that influence red blood cell production and function, with the goal of addressing conditions that have limited treatment options.
Agios’ principal commercial product is Pyrukynd (mitapivat), an oral activator of pyruvate kinase. Pyrukynd is approved in the United States and other markets for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency, a rare inherited blood disorder.
