CAMBRIDGE, Mass. - Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) presented detailed results from the 52-week double-blind period of its RISE UP Phase 3 trial of mitapivat in patients aged 16 years or older with sickle cell disease today at the 31st European Hematology Association Congress in Stockholm, Sweden, according to a press release statement.
The trial demonstrated that 40.6% of patients in the mitapivat arm achieved hemoglobin response, defined as a 1.0 g/dL or greater increase from baseline in average hemoglobin from Week 24 through Week 52, compared with 2.9% in the placebo arm. Among hemoglobin responders, the mean change from baseline in average hemoglobin concentration was 1.6 g/dL.The positive trial data comes as Agios shares trade at $29.46, with the stock showing an 8% gain year-to-date. According to InvestingPro analysis, the stock appears undervalued relative to its Fair Value.
New analyses showed patients in the mitapivat arm had a 41.1% relative reduction in the proportion of patients requiring blood transfusions compared with placebo (23.9% versus 40.6%) and a 55.9% relative reduction in average red blood cell units transfused per patient (0.70 units versus 1.59 units).
Patients in the mitapivat arm who achieved hemoglobin response experienced a 26% reduction in the annualized rate of sickle cell pain crises (2.20 versus 2.98 for non-responders) and 34% fewer related hospitalizations (1.16 versus 1.76 for non-responders). These patients also reported greater improvements in fatigue scores than non-responders, exceeding the predefined 4.1-point threshold for clinical meaningfulness.
The trial did not meet its primary endpoint for reduction in the annualized rate of sickle cell pain crises compared with placebo, and there was no overall difference between mitapivat and placebo for the key secondary endpoint measuring patient-reported fatigue.
Mitapivat was well-tolerated, with 97.1% of patients in the mitapivat arm reporting treatment-emergent adverse events compared with 98.6% in the placebo arm. No treatment-related deaths occurred during the trial.
The global RISE UP trial randomized 207 participants 2:1 to receive oral mitapivat (100 mg) twice daily or matched-placebo. Of the 176 participants who completed the double-blind period, 174 transitioned into a 216-week open-label extension period.
In May 2026, Agios submitted a supplemental New Drug Application to the U.S. Food and Drug Administration for accelerated approval of mitapivat in sickle cell disease.The company, valued at $1.75 billion, posted 78% revenue growth in the last twelve months as of Q1 2026. Analysts have set price targets ranging from $28 to $59, with a consensus recommendation leaning positive. An InvestingPro tip notes that while the company holds more cash than debt on its balance sheet, it is quickly burning through cash—typical for biotech firms advancing clinical programs. For deeper insights into Agios’s financial health and growth prospects, investors can access the comprehensive Pro Research Report, available for this and 1,400+ other US equities on InvestingPro.
In other recent news, Agios Pharmaceuticals Inc. has entered into a licensing agreement with Oscotec to secure exclusive global rights to cevidoplenib, a spleen tyrosine kinase inhibitor aimed at treating immune thrombocytopenia. Oscotec will receive $25 million upfront and could earn up to $140 million in development and regulatory milestones for up to three indications in the U.S. and Europe. Additionally, Oscotec will receive commercial milestone payments and royalties on future net sales. In a significant development, Agios has decided to halt the development of tebapivat for lower-risk myelodysplastic syndromes after a Phase 2b trial did not meet the company’s advancement criteria. The trial involved 65 patients and aimed to achieve transfusion independence over a 24-week period. Meanwhile, the European Commission has approved mitapivat for treating anemia in adults with thalassemia, and Avanzanite Bioscience will commercialize the drug in Europe under an agreement with Agios. Lastly, Agios will present data from its RISE UP Phase 3 trial of mitapivat in sickle cell disease at the European Hematology Association Congress.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.
The fastest way to find out is with our Fair Value calculator. We use a mix of 17 proven industry valuation models for maximum accuracy. Get the bottom line for AGIO plus thousands of other stocks and find your next hidden gem with massive upside.