TD Cowen analyst Marc Frahm has identified Agios as the firm’s top pick within the biotech sector, pointing to the company’s lead therapy mitapivat and an expanding development portfolio. The recommendation follows a series of data disclosures from Agios at the European Hematology Association 2026 conference that the analyst said reinforced confidence in mitapivat across several indications and highlighted early-stage progress for other assets.
Several of the presentations centered on mitapivat. New analyses from the Phase 3 RISE-UP study in sickle cell disease showed an average improvement in hemoglobin of 0.77 g/dL for mitapivat-treated patients versus 0.03 g/dL for placebo. The company also reported transfusion-related benefits: a 41% relative reduction in the proportion of patients requiring transfusions and a 56% reduction in red blood cell units transfused per patient, metrics that the company noted surpass historical outcomes observed with hydroxyurea.
To confirm efficacy, Agios plans a Phase 3 confirmatory trial named REIGNITE. The study is expected to enroll about 159 patients and uses a primary endpoint defined as transfusion-free status from weeks 4 through 52. The trial is reported to be powered at roughly 98% for that primary endpoint.
Additional Phase 3 information came from the ENERGIZE program in non-transfusion dependent thalassemia. Data from an open-label extension showed a sustained and deepening hemoglobin response, with the mean duration of hemoglobin response rising to approximately 43.6 weeks - more than double earlier measures. Among patients who continued mitapivat into the open-label extension, around 60% achieved hemoglobin increases of at least 1 g/dL.
On the commercial front, the company’s Aqvesme launch is tracking ahead of internal expectations. Agios reported roughly 242 prescriptions by the end of the first quarter, which the company says implies about $82 million in annualized demand. That figure compares with fiscal year 2026 consensus revenue of about $63 million.
Beyond mitapivat and Aqvesme, Agios shared interim Phase 1 data for AG-236, an siRNA therapeutic targeting TMPRSS6 in polycythemia vera. The interim results included dose-dependent induction of hepcidin and pharmacodynamic activity persisting beyond approximately 57 days. The company said these findings support the potential for extended dosing intervals, possibly out to every 24 weeks.
TD Cowen’s designation of Agios as its top biotech pick is driven by the combined clinical and early commercial signals described at the conference. The analyst emphasized mitapivat’s profile in thalassemia and expressed confidence that the company can maintain the strong early launch momentum it has demonstrated.
Contextual note - The updates presented at the conference span confirmatory trials, open-label extension data, commercial launch metrics, and interim Phase 1 pharmacodynamics, reflecting activity across both late-stage and early-stage programs within Agios’ pipeline.