Agios Pharmaceuticals, Inc.
Clinical trials sponsored by Agios Pharmaceuticals, Inc., explained in plain language.
Biotech focused on cellular metabolism to treat cancer and rare genetic diseases.
Not investment advice. Company information is shown for context only. Clinical trials frequently fail.
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Upcoming readouts
Trials from this sponsor expected to report results within the next 12 months.
- Global registry launches to unlock secrets of rare blood disorder Results expected May 2027 (estimated)
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New PKU pill enters early human testing
Disease control Recruiting nowThis early-stage trial is testing a new drug called AG-181 in 20 adults with phenylketonuria (PKU), a genetic condition that makes it hard to break down an amino acid called phenylalanine. The main goal is to check if the drug is safe and tolerable. Researchers will also measure …
Phase 1 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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New pill could help kids with thalassemia fight anemia without transfusions
Disease control Recruiting nowThis Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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New pill may cut blood transfusions for kids with thalassemia
Disease control Recruiting nowThis study tests a drug called mitapivat in children aged 1 to 17 with alpha- or beta-thalassemia who need regular blood transfusions. The goal is to see if mitapivat can reduce the number of transfusions needed compared to a placebo. The study involves 54 participants and lasts …
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Could a pill cut transfusions for sickle cell patients?
Disease control Recruiting nowThis Phase 3 trial tests whether the drug mitapivat can reduce the need for blood transfusions in people with sickle cell disease. About 159 participants aged 12 and older will receive either mitapivat or a placebo daily for a year. The main goal is to see if more people can avoi…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC