Agios Pharmaceuticals Inc AGIO

Overview

Agios Pharmaceuticals, Inc. is a prominent biopharmaceutical company based in the United States, specifically headquartered in Cambridge, Massachusetts. Founded in 2007, Agios is at the forefront of discovering and developing innovative medicines targeting cellular metabolism. The company's research and development efforts are primarily focused on creating treatments for diseases with significant unmet medical needs, emphasizing hemolytic anemias, myelodysplastic syndromes, phenylketonuria, and polycythemia vera among others. Through its dedication to scientific breakthroughs, Agios Pharmaceuticals aims to transform the therapeutic landscape for patients across these disease areas.

Products and Services

• PYRUKYND (mitapivat)
  

  An activator of both wild-type and mutant forms of the pyruvate kinase (PK) enzyme, PYRUKYND is Agios Pharmaceuticals' leading product. It represents a pioneering approach in the treatment of hemolytic anemias, offering significant hope for patients with this group of disorders by targeting the underlying metabolic dysfunction.

• AG-946
  

  Another PK activator in the company's pipeline, AG-946, is currently under development for its potential application in the treatment of lower-risk myelodysplastic syndrome and hemolytic anemias. This exploration signifies the company's ongoing efforts to expand the therapeutic applications of its PK activation technology to broader patient populations.

• AG-181
  

  AG-181 is a phenylalanine hydroxylase stabilizer aimed at treating phenylketonuria, a genetic disorder that increases the levels of phenylalanine in the blood. By stabilizing the enzyme involved in metabolizing phenylalanine, AG-181 holds the promise of managing and potentially reducing the complications associated with this condition.

• siRNA for Polycythemia Vera
  

  In the preclinical development stage, Agios is exploring the use of small interfering RNA (siRNA) technology for the treatment of polycythemia vera, a rare blood disorder characterized by the overproduction of red blood cells. This innovative approach aims to address the disease at its genetic roots, offering a novel avenue for potentially effective therapy.