Agios Pharmaceuticals

Agios Pharmaceuticals — A to Z Product Catalog

Marketed Product

• Pyrukynd®
  • Generic / Type: Mitapivat (oral small‑molecule pyruvate kinase activator)
  • Indication: Treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency
  • Dose/Form: Oral tablets, dosing titrated (common regimen: start 5 mg twice daily, escalate to 20 mg or 50 mg twice daily per hemoglobin response and tolerability)

---

Clinical-Stage Pipeline (All Indications Investigational)

• **Mitapivat** (beyond PK deficiency)
  • Type: Pyruvate kinase activator (oral)
  • Indications Under Investigation:

  • Non‑transfusion‑dependent and transfusion‑dependent alpha- and beta-thalassemia (adult and pediatric populations)

  • Sickle cell disease (adult and pediatric)
    • Dose/Form: Oral dosing similar to PK deficiency trials (titrated per regimen)

• Tebapivat
  • Type: Pyruvate kinase activator (oral, investigational)
  • Indications Under Investigation:

  • Lower‑risk myelodysplastic syndromes (MDS)

  • Sickle cell disease
    • Dose/Form: Oral dosing in early clinical development

• AG-181
  • Type: Phenylalanine hydroxylase stabilizer (small molecule)
  • Indication Under Investigation: Phenylketonuria (PKU)
  • Dose/Form: Preclinical stage

• AG-236
  • Type: siRNA targeting TMPRSS6
  • Indication: Polycythemia vera
  • Dose/Form: Preclinical development

• Other PKR/PKM2 Modulators
  • Type: Additional pyruvate kinase activators
  • Indications: Broad rare metabolic diseases involving red blood cells
  • Dose/Form: Early discovery/preclinical

•