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Agios Advances Sickle Cell Treatment, Completes Enrolment For Phase 3 RISE UP Study Of Mitapivat

Agios completes enrolment for its Phase 3 RISE UP trial, testing mitapivat in sickle cell patients.

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  • Oct 24, 2024

  • Simantini Singh Deo

Agios Advances Sickle Cell Treatment, Completes Enrolment For Phase 3 RISE UP Study Of Mitapivat

Agios Pharmaceuticals, a chief in cellular metabolism and pyruvate kinase (PK) activation therapies for rare diseases, has completed patient enrolment for its Phase 3 RISE UP trial. This global, double-blind, placebo-controlled study evaluates the safety and effectiveness of mitapivat, an oral PK activator, in patients aged 16 and older with sickle cell disease. Agios anticipates releasing the topline results from the 52-week trial by late 2025.


Sickle cell disease is a lifelong genetic disorder caused by hemoglobin mutations, resulting in sickle-shaped red blood cells. These cells struggle to move through blood vessels, often leading to blocked blood flow, anemia, pain, organ damage, and even premature death. In people with sickle cell disease, red blood cells require more energy in the form of adenosine triphosphate (ATP) and have higher levels of 2,3-diphosphoglycerate (2,3-DPG), which increases the risk of sickling. Mitapivat enhances red blood cell function by boosting ATP levels and lowering 2,3-DPG concentrations.


Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios, said, “There is a critical need for novel regimens that elevate the standard of care for patients who have sickle cell disease, a burdensome and debilitating disease,”


She also commented, “We sincerely thank the patients, study investigators and patient advocates for their instrumental support and partnership in helping us complete the Phase 3 RISE UP study enrolment. With enrolment now complete, we look forward to completing the trial and sharing topline results with the community in late 2025.”


The RISE UP trial consisted of over 200 patients from across the globe and aims to answer two critical endpoints: an increase of at least 1g/dL in hemoglobin values from Week 24 to Week 52 and a decrease in painful crises caused by sickle cell disease. Such endpoints are significant because anemia and pain are the defining features of sickle cell disease and impact these patients' quality of life.


Positive Phase 2 trial results, presented at the 2023 American Society of Hematology Annual Meeting and Exposition, paved the way to further Agios’ more extensive Phase 3 study into the risks and benefits of mitapivat for people living with sickle cell disease.

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