Agios Pharmaceuticals

Agios Pharmaceuticals specializes in developing first-in-class therapies that modulate cellular metabolism, with a primary focus on rare hematologic diseases. Its lead product mitapivat (PYRUKYND) was approved by the FDA in 2022 as the first disease-modifying therapy for adults with pyruvate kinase deficiency. The company has filed regulatory submissions in multiple regions—including the U.S., EU, Saudi Arabia, and UAE—for mitapivat's use in thalassemia following positive Phase 3 results. A pivotal Phase 3 trial ("RISE UP") of mitapivat in sickle cell disease completed enrollment with topline results expected by late 2025. Additional pipeline assets include tebipivat—a next-generation PK activator—and AG‑181 targeting phenylketonuria.