Agios develops oral, disease‑modifying therapies that target metabolic enzymes to treat rare hemolytic anemias and phenylketonuria. By leveraging its expertise in cellular metabolism, the company creates PK activators and PAH stabilizers that improve red blood cell durability and normalize phenylalanine levels, offering long‑term benefits for patients and their clinicians.
Funding
$256M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Patients with rare hemolytic anemias and related metabolic disorders have few effective treatment options, leading to chronic symptoms, reduced red blood cell lifespan, and limited quality of life.
Solution
Agios leverages its deep expertise in cellular metabolism to develop oral, disease‑modifying therapies that target key metabolic enzymes such as pyruvate kinase (PK) and phenylalanine hydroxylase (PAH). By activating PK, its candidates increase ATP production, lower 2,3‑DPG levels, and improve red blood cell durability, addressing the underlying cause of hemolytic anemia. The company also pursues PAH stabilizers to normalize phenylalanine levels in phenylketonuria, enabling patients to increase natural protein intake. These therapies are designed to provide long‑term benefits rather than merely palliative relief, and are advanced through a structured pipeline from discovery to regulatory submission.
Target Audience
Primary customers are patients with rare hemolytic anemias (including PK deficiency, sickle cell disease, thalassemia) and phenylketonuria, as well as the hematologists, geneticists, and specialty clinics that treat these conditions.
Features
- PK activator drugs (e.g., mitapivat, tebapivat) that enhance red blood cell energy metabolism and lifespan
- Oral administration for convenient, patient‑friendly dosing
- Disease‑modifying mechanism targeting the metabolic root cause of hemolytic anemia and PKU
- Integrated preclinical and clinical development framework leveraging 15 years of metabolism research
- Collaborative partnership model with patient groups, healthcare providers, and research institutions to inform development
- Pipeline includes additional candidates such as PAH stabilizers and siRNA therapies for related rare diseases