Agios shares rose after strong earnings and Aqvesme's commercial launch.
Mitapivat faces competition from Novo Nordisk's etavopivat in the treatment of sickle cell disease.
Key pipeline updates and catalysts are expected in 2026, especially for tebapivat.
Shares in Agios Pharmaceuticals (NASDAQ: AGIO) rose by 11.2% this week, putting the stock into positive territory for the year. The move comes after a well-received first-quarter earnings report that showed strong progress in Aqvesme's U.S. commercial launch. Management also provided an update on the progress of its pipeline, including mitapivat for sickle cell disease (SCD) and tebapivat for Lower-Risk Myelodysplastic Syndromes (MDS) and SCD.
The pharmaceutical company already has mitapivat approved to treat hemolytic anemia in adults with Pyruvate Kinase (PK) deficiency under the brand name Pyrukynd. It also has mitapivat, which was more recently approved in the U.S. to treat adult thalassemia under the brand name Aqvesme, and the addition of Aqvesme sales (the first quarter of full sales) helped sales grow to $20.7 million from just $8.7 million in the same quarter of 2025. Management expects "very strong demand" for Aqvesme in the coming quarters.
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As readers already know, the company recently suffered a setback when a rival developer of a pyruvate kinase-R (PKR) activator, Novo Nordisk, reported top-line results from a trial indicating that its drug, etavopivat, was superior to mitapivat in treating SCD. In a nutshell, etavopivat achieved both of its primary endpoints in SCD, but mitapivat failed to meet one of them: reducing the annualized rate of pain crisis events.
It's unclear what the market potential for mitapivat (SCD) will be. Still, management confirmed it was aligned with the Food and Drug Administration (FDA) "on a path toward U.S. accelerated approval for mitapivat in sickle cell disease" with filing expected in the second quarter.
Image source: Getty Images.
That said, investors probably have higher hopes for its next-generation PKR, tebapivat, which management confirmed will have Phase 2 top-line results available in MDS in the first half of 2026, and then SCD in the second half of 2026. All told, a good start for Aqvesme, progress on mitapivat approval in SCD, and the hope of some positive news on tebapivat make for an exciting year for the company.
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Lee Samaha has no position in any of the stocks mentioned. The Motley Fool recommends Novo Nordisk. The Motley Fool has a disclosure policy.